ABSTRACT
Approximately 90% of confirmed cancer cases annually are reported in low to middle-income countries. In Honduras, the incidence of pediatric cancer has been steadily increasing, accompanied by a higher cancer mortality rate attributed to diagnostic errors, limited access to healthcare, and management challenges. Diagnostic pitfalls, such as failure to recognize signs of malignancy, inadequate assessment of persistent symptoms, and misinterpretation of diagnostic tests, significantly impede effective cancer care. This retrospective case study collected data from 68 pediatric patients diagnosed with Acute Lymphoblastic Leukemia (ALL) at the Honduran Social Security Institute in Tegucigalpa between January 2015 and December 2022. Data retrieval encompassed demographic features, clinical characteristics, and laboratory findings. We used SPSS Statistics version 29.0.2.0 to perform all statistical analysis. The cohort comprised patients of equal gender distribution, with 42.6% (N: 29) belonging to the age group of 1 to 4 years. The hospital diagnosed an average of 8.5 cases each year. Fever was the most prevalent symptom, affecting 80.9% of patients (N: 55). Hemoglobin levels were below 10 mg/dL in 67.6% of patients, with 33.8% exhibiting levels below 7 mg/dL (N: 23) and equal proportion falling within the 7-10 mg/dL range (N: 23). Platelet levels were below 150,000/µL, with 48.5% experiencing severe thrombocytopenia (platelet levels <50,000/µL). Additionally, most patients presented phosphorus levels exceeding 4.5 mg/dl (N: 33, 48.5%), along with elevated LDH levels surpassing 500 U/l (N: 34, P: 50%). The presence of persistent fever should trigger suspicion of cancer, necessitating thorough assessment. Implementing guidelines outlining common symptoms and referral protocols could significantly reduce mortality in Honduran children with ALL.
Aproximadamente el 90% de los casos confirmados de cáncer anualmente se reportan en países de ingresos bajos a medios. En Honduras, la incidencia de cáncer pediátrico ha aumentado, con una mayor mortalidad atribuida a errores de diagnóstico, acceso limitado a la atención médica y desafíos en el manejo. Los errores diagnósticos, como no reconocer signos de malignidad, evaluación inadecuada de síntomas persistentes e interpretación errónea de pruebas diagnósticas, dificultan la atención efectiva del cáncer. Este estudio retrospectivo comprende una muestra de 68 pacientes pediátricos diagnosticados con Leucemia Linfoblástica Aguda (LLA) en el Instituto Hondureño de Seguridad Social en Tegucigalpa entre enero de 2015 y diciembre de 2022. La recopilación de datos incluyó características demográficas, clínicas y hallazgos de laboratorio; con análisis estadístico utilizando SPSS Statistics versión 29.0.2.0. La cohorte comprendía pacientes de igual distribución por género, con el 42.6% (N: 29) perteneciendo al grupo de edad de 1 a 4 años. El hospital diagnosticó un promedio de 8.5 casos cada año. La fiebre fue el síntoma más prevalente, afectando al 80.9% de los pacientes (N: 55). Los niveles de hemoglobina fueron inferiores a 10 mg/dL en el 67.6% de los pacientes, con el 33.8% por debajo de 7 mg/dL (N: 23) y una proporción igual dentro del rango de 7-10 mg/dL (N: 23). Los niveles de plaquetas fueron inferiores a 150,000/µL, con el 48.5% experimentando trombocitopenia severa (niveles de plaquetas <50,000/µL). Además, la mayoría de los pacientes presentaron niveles de fósforo superiores a 4.5 mg/dL (N: 33, 48.5%) y niveles elevados de LDH que superaban los 500 U/L (N: 34, P: 50%). La presencia de fiebre persistente debe despertar sospechas de cáncer, requiriendo una evaluación exhaustiva. La implementación de guías que delineen síntomas comunes y protocolos de referencia podría reducir significativamente la mortalidad en niños hondureños con LLA.
Subject(s)
Humans , Male , Female , Infant , Child, Preschool , Child , Adolescent , Pediatrics/methods , Precursor Cell Lymphoblastic Leukemia-Lymphoma/immunology , Precursor Cell Lymphoblastic Leukemia-Lymphoma/therapy , Population Characteristics , Retrospective Studies , Clinical Laboratory Techniques , Antineoplastic Agents, Immunological/therapeutic use , Honduras , Immunotherapy/methodsABSTRACT
Introducción. La leucemia es el principal tipo de cáncer infantil, con una tasa de incidencia para 2022 de 5.5 por cada 100,000 menores. La hipercalcemia maligna es una de sus manifestaciones paraneoplásica grave y poco frecuente (incidencia global del 0.4 1.3%) en la población pediátrica. Objetivo. Presentar un caso de leucemia linfoblástica aguda que debutó con hipercalcemia severa. Descripción del caso. Se trata de una preescolar femenina de 3 años que inició con cuadro clínico caracterizado por dolores óseos y limitación funcional, los hemogramas iniciales no mostraron alteración de líneas celulares, las radiografías evidenciaron osteopenia generalizada, acompañado de hipercalcemia severa, paratohormona inhibida e hipercalciuria secundaria, que fueron manejados con hidratación endovenosa, diurético, esteroide y ácido zolendrónico. Así mismo, presentó desequilibrios electrolíticos que requirieron reposición de potasio y fósforo con adecuada respuesta. Se realizaron estudios de médula ósea, confirmándose el diagnóstico de leucemia linfoblástica aguda, recibió quimioterapia protocolo ALLIC 2009 con enfermedad refractaria al final de la fase de inducción, y finalmente trasplante haploidéntico de médula que fue exitoso. Discusión. La hipercalcemia maligna es una de las urgencias oncológicas endocrinológica con una incidencia baja, que es más frecuente en la población adulta, por lo que no es la primera impresión diagnóstica para considerar en pediatría, lo que lleva a retrasos en el diagnóstico etiológico y en pronóstico. Conclusión. La hipercalcemia acompañada de lesiones osteolíticas difusas puede ser la primera y única manifestación en la población infantil con diagnóstico de leucemia linfoblástica aguda, reconocerla permitirá llevar al inicio oportuno de tratamiento, impactando en sobrevida.
Introduction. Leukaemia is the leading childhood cancer, with a 2022 incidence rate of 5.5 per 100,000 children. In the pediatric population, Hypercalcaemia maligna is one of its rare and severe paraneoplastic manifestations (overall incidence 0.4 - 1.3%). Objective. To present a case of acute lymphoblastic leukemia that debuted with severe hypercalcemia. Case description. It is about a 3-year-old female preschooler who started with a clinical picture characterized by bone pain and functional limitation; initial haemograms showed no alteration of cell lines, radiographs showed generalized osteopenia, accompanied by severe hypercalcemia, inhibited parathyroid hormone and secondary hypercalciuria, which were managed with intravenous hydration, diuretic, steroid and zoledronic acid. She also presented electrolyte imbalances that required potassium and phosphorus replacement with adequate response. Bone marrow studies were performed, confirming the diagnosis of acute lymphoblastic leukemia; she received ALLIC 2009 protocol chemotherapy with refractory disease at the end of the induction phase, and finally, a haploidentical bone marrow transplant, which was successful. Discussion. Hypercalcemia of malignancy is one of the endocrinological oncological emergencies with a low incidence, which is more frequent in the adult population. So, it is not the first diagnostic impression to be considered in pediatrics, leading to delays in aetiological diagnosis and prognosis. Conclusion. Hypercalcaemia accompanied by diffuse osteolytic lesions may be the first and only manifestation in the pediatric population with a diagnosis of acute lymphoblastic leukemia. Recognizing it will lead to the timely initiation of treatment, with an impact on survival
Introdução. A leucemia é o principal tipo de câncer infantil, com uma taxa de incidência de 5.5 por 100,000 crianças em 2022. A hipercalcemia maligna é uma das suas manifestações paraneoplásicas graves e raras (incidência global de 0.4 1.3%) na população pediátrica. Objetivo. Apresentar um caso de leucemia linfoblástica aguda que teve início com hipercalcemia severa. Descrição do caso. Trata-se de uma criança pré-escolar do sexo feminino, de 3 anos, que apresentou um quadro clínico caracterizado por dores ósseas e limitação funcional. Os hemogramas iniciais não mostraram alterações nas linhas celulares, e as radiografias evidenciaram osteopenia generalizada, acompanhada de hipercalcemia severa, com paratormônio inibido e hipercalciúria secundária. O manejo incluiu hidratação endovenosa, diurético, esteroide e ácido zoledrônico. Da mesma forma, a paciente apresentou desequilíbrios eletrolíticos que exigiram reposição de potássio e fósforo, com resposta adequada. Foram realizados estudos de medula óssea, confirmando o diagnóstico de leucemia linfoblástica aguda. Ela recebeu quimioterapia seguindo o protocolo ALLIC 2009, mas apresentou doença refratária ao final da fase de indução, e, por fim, foi submetida a um transplante haploidêntico de medula, que foi bem-sucedido. Discussão. A hipercalcemia maligna é uma das urgências oncológicas endocrinológicas com baixa incidência, sendo mais comum na população adulta. Por isso, não é a primeira impressão diagnóstica a ser considerada em pediatria, o que pode resultar em atrasos no diagnóstico etiológico e no prognóstico da condição. Conclusão. A hipercalcemia acompanhada de lesões osteolíticas difusas pode ser a primeira e única manifestação na população infantil com diagnóstico de leucemia linfoblástica aguda. O reconhecimento precoce permitirá o início oportuno do tratamento, impactando positivamente na sobr evida.
Subject(s)
Hypercalcemia , Bone Marrow , Case Reports , Hematologic Neoplasms , Precursor Cell Lymphoblastic Leukemia-LymphomaSubject(s)
Humans , Child, Preschool , Child , Adolescent , Neoplasm, Residual/therapy , Precursor Cell Lymphoblastic Leukemia-Lymphoma/therapy , Arabinonucleosides/therapeutic use , Antibodies, Bispecific/therapeutic use , Hematopoietic Stem Cell Transplantation , Cell- and Tissue-Based Therapy , Clofarabine/therapeutic use , Inotuzumab OzogamicinABSTRACT
Introducción: desde 2002, el Grupo Argentino para el Tratamiento de la Leucemia Aguda (GATLA) implementa protocolos del grupo Berlín-Frankfurt-Münster (BFM) como tratamiento estándar de las recaídas de la leucemia linfoblástica aguda (LLA). En 2010, el BFM generó el protocolo IntReALL 10, que en la Argentina se implementó con las limitaciones propias de la región. Población y métodos: 180 pacientes menores de 18 años fueron tratados entre 2010 y 2015 por una LLA recaída de alto riesgo en la Argentina siguiendo un protocolo de recaída del BFM que comparó en forma abierta el tratamiento estándar con una terapéutica innovadora (experimental); esta incluyó el fármaco clofarabina. Se evaluaron 171 pacientes, de los cuales 78 pacientes fueron aleatorizados en forma centralizada (ensayo clínico) y 93 fueron asignados a una de las ramas según el criterio del grupo tratante (cohorte prospectiva). La cohorte donde la asignación del tratamiento no fue aleatorizada fue analizada realizando un ajuste por sexo, edad y por la presencia o no de síndrome de Down, cromosoma Philadelphia e inmunofenotipo T. Resultados: los pacientes que recibieron el tratamiento experimental tuvieron peores resultados (el doble de mortalidad a cinco años) que los que recibieron tratamiento estándar. Esta diferencia alcanzó significancia estadística tanto en el ensayo clínico (p=0,001) como en la cohorte prospectiva (p=0,0009). Conclusiones: nuestros resultados avalan continuar con la rama estándar de los protocolos tipo BFM para el tratamiento de las recaídas de la LLA y fueron concordantes con las conclusiones del grupo ALLIC-REC. (AU)
Introduction: since 2002, the Grupo Argentino para el Tratamiento de la Leucemia Aguda (GATLA) has been implementing protocols from the Berlin-Frankfurt-Münster (BFM) group as the standard treatment for relapses of acute lymphoblastic leukemia (ALL). In 2010, BFM developed the IntReALL 10 protocol, implemented in Argentina with the inherent limitations of the region. Population and Methods: we treated a total of 180 patients under 18 years of age between 2010 and 2015 for high-risk relapsed acute lymphoblastic leukemia (ALL) in Argentina following a BFM relapse protocol. This protocol openly compared standard treatment with an innovative (experimental) therapeutic approach that included Clofarabine. Out of these, 171 patients were assessable, with 78 patients being centrally randomized in a clinical trial, and 93 were assigned to one of the arms based on the treating group's criteria (prospective cohort). The cohort where the treatment assignment had not been randomized, was analyzed with adjustments for gender, age, and the presence or absence of Down Syndrome, Philadelphia Chromosome, and T-cell immunophenotype. Results: patients who received the experimental treatment had worse outcomes (double the five-year mortality) compared to those who received the standard treatment. This difference reached statistical significance in the clinical trial (p=0.001) and the prospective cohort (p=0.0009). Conclusions: our results support the continuation of the standard arm in BFM-type protocols for relapsed ALL treatment and were consistent with the conclusions of the ALLIC-REC group. (AU)
Subject(s)
Humans , Male , Female , Infant, Newborn , Infant , Child, Preschool , Child , Adolescent , Neoplasm, Residual/drug therapy , Precursor Cell Lymphoblastic Leukemia-Lymphoma/drug therapy , Clofarabine/administration & dosage , Argentina/epidemiology , Asparaginase/administration & dosage , Vincristine/administration & dosage , Dexamethasone/administration & dosage , Survival Analysis , Clinical Protocols , Methotrexate/administration & dosage , Treatment Outcome , Neoplasm, Residual/mortality , Neoplasm, Residual/epidemiology , Cyclophosphamide/administration & dosage , Cytarabine/administration & dosage , Etoposide/administration & dosage , Precursor Cell Lymphoblastic Leukemia-Lymphoma/mortality , Precursor Cell Lymphoblastic Leukemia-Lymphoma/epidemiologyABSTRACT
Chimeric antigen receptor (CAR)-modified T-cell therapy has achieved remarkable success in the treatment of acute lymphoblastic leukemia (ALL). Measurable/minimal residual disease (MRD) monitoring plays a significant role in the prognostication and management of patients undergoing CAR-T-cell therapy. Common MRD detection methods include flow cytometry (FCM), polymerase chain reaction (PCR), and next-generation sequencing (NGS), and each method has advantages and limitations. It has been well documented that MRD positivity predicts a poor prognosis and even disease relapse. Thus, how to perform prognostic evaluations, stratify risk based on MRD status, and apply MRD monitoring to guide individual therapeutic decisions have important implications in clinical practice. This review assesses the common and novel MRD assessment methods. In addition, we emphasize the critical role of MRD as a prognostic biomarker and summarize the latest studies regarding MRD-directed combination therapy with CAR-T-cell therapy and allogeneic hematopoietic stem cell transplantation (allo-HSCT), as well as other therapeutic strategies to improve treatment effect. Furthermore, this review discusses current challenges and strategies for MRD detection in the setting of disease relapse after targeted therapy.
Subject(s)
Humans , Receptors, Chimeric Antigen/therapeutic use , Neoplasm, Residual , Transplantation, Homologous/methods , Transplantation Conditioning/methods , Hematopoietic Stem Cell Transplantation/methods , Recurrence , Precursor Cell Lymphoblastic Leukemia-Lymphoma/therapyABSTRACT
Objective: To analyze the clinical characteristics and prognosis of patients with infant acute lymphoblastic leukemia (IALL). Methods: A retrospective cohort study.Clinical data, treatment and prognosis of 28 cases of IALL who have been treated at Beijing Children's Hospital, Capital Medical University and Baoding Children's Hospital from October 2013 to May 2023 were analyzed retrospectively. Based on the results of fluorescence in situ hybridization (FISH), all patients were divided into KMT2A gene rearrangement (KMT2A-R) positive group and KMT2A-R negative group. The prognosis of two groups were compared. Kaplan-Meier method and Log-Rank test were used to analyze the survival of the patients. Results: Among 28 cases of IALL, there were 10 males and 18 females, with the onset age of 10.9 (9.4,11.8) months. In terms of immune classification, 25 cases were B-ALL (89%), while the remaining 3 cases were T-ALL (11%). Most infant B-ALL showed pro-B lymphocyte phenotype (16/25,64%). A total of 22 cases (79%) obtained chromosome karyotype results, of which 7 were normal karyotypes, no complex karyotypes and 15 were abnormal karyotypes were found. Among abnormal karyotypes, there were 4 cases of t (9; 11), 2 cases of t (4; 11), 2 cases of t (11; 19), 1 case of t (1; 11) and 6 cases of other abnormal karyotypes. A total of 19 cases (68%) were positive for KMT2A-R detected by FISH. The KMT2A fusion gene was detected by real-time PCR in 16 cases (57%). A total of 24 patients completed standardized induction chemotherapy and were able to undergo efficacy evaluation, 23 cases (96%) achieved complete remission through induction chemotherapy, 4 cases (17%) died of relapse. The 5-year event free survival rate (EFS) was (46±13)%, and the 5-year overall survival rate (OS) was (73±10)%.The survival time was 31.3 (3.3, 62.5) months. There was no significant statistical difference in 5-year EFS ((46±14)% vs. (61±18)%) and 5-year OS ((64±13)% vs. (86±13)%) between the KMT2A-R positive group (15 cases) and the KMT2A-R negative group (9 cases) (χ2=1.88, 1.47, P=0.170, 0.224). Conclusions: Most IALL patients were accompanied by KMT2A-R. They had poor tolerance to traditional chemotherapy, the relapse rate during treatment was high and the prognosis was poor.
Subject(s)
Male , Child , Infant , Female , Humans , Retrospective Studies , In Situ Hybridization, Fluorescence , Prognosis , Precursor Cell Lymphoblastic Leukemia-Lymphoma/therapy , Abnormal Karyotype , RecurrenceABSTRACT
ABSTRACT Introduction: B cell acute lymphoblastic leukemia-lymphoma (B-ALL) accounts for approximately 75% of ALL cases and is observed in children and adults. Recent advances in disease diagnosis, stratification and prognostication have led to a better characterization of different subgroups of ALL. Notwithstanding the significant improvement in the complete remission rate of B-ALL, patients with minimal residual disease (MRD) and relapsed/refractory (R/R) settings suffer from poor outcomes. Hypothesis: However, novel therapies, such as agents targeting tyrosine kinases or the CD20 molecule, combination therapies and improved supportive care, have changed the treatment landscape of B-ALL. Method and results: Meanwhile, blinatumomab has been FDA-approved for MRD-positive or R/R B-ALL patients. Blinatumomab is a bispecific T cell engager containing the CD3 and CD19 that recognize domains redirecting cytotoxic T cells to lyse B cells. Promising outcomes, including long-term overall survival and improved MRD-negative response rates, have been reported in patients who received this drug. Adding blinatumomab to new ALL regimens seems promising for achieving better outcomes in poor prognosis B-ALL patients. Nevertheless, the neurotoxicity and cytokine release syndrome are the two major adverse events following the blinatumomab therapy. Conclusion: This review summarizes the function and effectiveness of blinatumomab in R/R and MRD positive B-ALL patients. Furthermore, blinatumomab's positive and negative aspects as a novel therapy for B-ALL patients have been briefly discussed.
Subject(s)
Humans , Male , Female , Neoplasm, Residual , Precursor Cell Lymphoblastic Leukemia-Lymphoma , LymphomaABSTRACT
Resumo Introdução O câncer tem impacto na vida das crianças e seus familiares. As Histórias em Quadrinhos podem ser uma estratégia de fortalecer o vínculo e a comunicação entre profissional/paciente/família. Objetivo Desenvolver e validar um material instrucional/educativo, no formato de Histórias em Quadrinhos, voltada para crianças hospitalizadas com leucemia linfóide aguda. Metodologia Estudo metodológico desenvolvido em nove etapas: elaboração do projeto de pesquisa; definição e seleção do conteúdo; adaptação da linguagem; inclusão de ilustrações; construção de um material piloto; validação do material; layout; impressão final e disponibilização. A validação ocorreu com 10 especialistas entre março e maio de 2022, utilizando-se o Instrumento de Validação de Conteúdo Educativo em Saúde. Resultados Foram elaboradas 5 Histórias em Quadrinhos, com 6 personagens principais, sendo necessárias 63 horas de trabalho. Elas foram divididas por temáticas (distúrbios gastrointestinais; cistite hemorrágica; problemas relacionados a autoestima e autoimagem; risco de infecção e dor óssea) que obtiveram Índice de Validade de Conteúdo global satisfatório entre 0,78 e 0,87. Conclusões e implicações para a prática As histórias em quadrinhos podem ser utilizadas como fonte atrativa e confiável de informações sobre a doença, servindo como apoio às informações durante a internação hospitalar e o preparo para alta.
Resumen Introducción El cáncer tiene un impacto en la vida de los niños y sus familias. Los cómics pueden ser una estrategia para fortalecer el vínculo y la comunicación entre profesional/paciente/familia. Objetivo Desarrollar y validar un material didáctico/educativo, en formato de Historietas, dirigido a niños hospitalizados con leucemia linfocítica aguda. Metodología Estudio metodológico desarrollado en nueve etapas: elaboración del proyecto de investigación; definición y selección de contenidos; adaptación lingüística; inclusión de ilustraciones; construcción de un material piloto; validación del material; disposición; impresión final y disponibilidad. La validación se realizó con 10 especialistas entre marzo y mayo de 2022, utilizando el Instrumento de Validación de Contenido de Educación en Salud. Resultados Se crearon 5 Comics, con 6 personajes principales, requiriendo 63 horas de trabajo. Fueron divididos por temas (trastornos gastrointestinales; cistitis hemorrágica; problemas relacionados con la autoestima y la autoimagen; riesgo de infección y dolor óseo) que obtuvieron un Índice de Validez de Contenido global satisfactorio entre 0,78 y 0,87. Conclusiones e implicaciones para la práctica Los cómics pueden ser utilizados como una fuente atractiva y confiable de información sobre la enfermedad, apoyando información durante la hospitalización y preparación para el alta.
Abstract Introduction Cancer has an impact on the lives of children and their families. Comics can be a strategy to strengthen the bond and communication between professional/patient/family. Objective To develop and validate an instructional/educational material, in the format of Comics, aimed at children hospitalized with acute lymphocytic leukemia. Methodology Methodological study developed in nine stages: preparation of the research project; content definition and selection; language adaptation; inclusion of illustrations; construction of a pilot material; validation of the material; layout; final printing and availability. Validation took place with 10 specialists between March and May 2022, using the Health Education Content Validation Instrument. Results 5 Comics were created, with 6 main characters, requiring 63 hours of work. They were divided by themes (gastrointestinal disorders; hemorrhagic cystitis; problems related to self-esteem and self-image; risk of infection and bone pain) that obtained a satisfactory global Content Validity Index between 0.78 and 0.87. Conclusions and implications for practice Comics can be used as an attractive and reliable source of information about the disease, supporting information during hospitalization and preparation for discharge.
Subject(s)
Humans , Male , Female , Child , Adult , Middle Aged , Child Health , Precursor Cell Lymphoblastic Leukemia-Lymphoma , Graphic Novels as Topic , Play and Playthings , NauseaABSTRACT
Introducción: La leucemia congénita constituye una entidad rara que se diagnostica entre el momento del nacimiento y hasta las 4 o 6 semanas de vida, es de evolución rápida y mal pronóstico. Se caracteriza por la presencia de hepatomegalia, esplenomegalia y lesiones hemorrágicas o infiltrativas en la piel de un recién nacido. El diagnóstico definitivo se realiza a través del estudio de aspirado de médula ósea, para poder determinar el fenotipo de la leucemia. El trastorno mieloproliferativo transitorio y las infecciones congénitas constituyen los principales diagnósticos diferenciales. El tratamiento se basa en regímenes de quimioterapia de agentes múltiples que pueden lograr la remisión del cuadro, pero los índices de recaídas continúan siendo altos. Objetivo: Describir dos presentaciones de casos poco frecuentes de leucemia aguda congénita. Caso clínico: Se presentan dos casos clínicos de recién nacidos a término con leucemia aguda congénita, el primero con fenotipo mixto y el segundo con fenotipo mieloide, se comenta el diagnóstico, tratamiento y pronóstico. Conclusiones: Se presentan dos casos de leucemia aguda congénita, esta enfermedad rara y de mal pronóstico, es poco publicada en la literatura. El presente reporte resalta la importancia de un examen físico exhaustivo que oriente la sospecha diagnóstica. Actualmente no se han definido claramente los factores pronósticos de esta enfermedad, por lo que se recomienda investigar factores asociados y establecer lineamientos para un diagnóstico temprano y tratamiento oportuno(AU)
Introduction: Congenital leukemia is a rare entity diagnosed between the time of birth and up to 4 to 6 weeks of life; it is of rapid evolution and poor prognosis. It is characterized by the presence of hepatomegaly, splenomegaly and hemorrhagic or infiltrative lesions on a newborn's skin. The definitive diagnosis is made by a bone marrow aspirate study, in order to determine the phenotype of the leukemia. Transient myeloproliferative disorder and congenital infections are the main differential diagnoses. Treatment is based on multi-agent chemotherapy regimens that may achieve remission, but relapse rates remain high. Objective: To describe two rare case presentations of acute congenital leukemia. Case report: Two clinical cases of term newborns with congenital acute leukemia are presented: the first with mixed phenotype and the second with myeloid phenotype. Diagnosis, treatment and prognosis are discussed. Conclusions: Two cases of acute congenital leukemia are presented, a rare disease with poor prognosis and about which little has been published in the literature. The present report highlights the importance of a thorough physical examination to guide the diagnostic suspicion. Currently, the prognostic factors of this disease have not been clearly defined, a reason why investigating associated factors is recommended, as well as establishing guidelines for early diagnosis and timely treatment(AU)
Subject(s)
Humans , Male , Female , Infant, Newborn , Leukemia, Biphenotypic, Acute/diagnosis , Leukemia, Myeloid, Acute/diagnosis , Leukemia/drug therapy , Precursor Cell Lymphoblastic Leukemia-Lymphoma/diagnosis , PeruABSTRACT
Introdução: Pacientes pediátricos com leucemia linfoblástica aguda (LLA), considerado o câncer infantil mais comum, demandam cuidado complexo, que inclui uma terapêutica com múltiplas etapas e de difícil compreensão pela população em geral. Nesse contexto, o déficit de conhecimento em saúde foi identificado como uma das causas da diminuição da autoeficácia em saúde, podendo impactar adversamente o tratamento das doenças. Portanto, torna-se imperativa a adoção de estratégias que possam efetivamente auxiliar os pacientes e seus cuidadores para aprimorar seus conhecimentos sobre o processo terapêutico da LLA. Objetivo: Descrever a elaboração e o contexto de utilização de um material educativo direcionado aos cuidadores e pacientes pediátricos diagnosticados com LLA, submetidos ao protocolo de tratamento ALL IC-BFM 2009. Método: Estudo descritivo da elaboração de cartilhas abordando detalhes sobre cada um dos medicamentos a serem administrados durante o referido protocolo. A confecção das cartilhas foi conduzida por uma farmacêutica, revisada por uma médica oncologista pediátrica e um segundo farmacêutico. Realizou-se também a análise da legibilidade textual por meio do software A LT®. Resultados: Foram confeccionadas sete cartilhas, cada uma relacionada a uma fase do protocolo de LLA. As cartilhas apresentaram alta legibilidade, com um texto considerado simples, contendo em média 21,5% de palavras complexas. Conclusão: Acredita-se que o material elaborado pode apoiar o uso apropriado de medicamentos para crianças em tratamento de LLA, podendo ser aprimorado ou adaptado para novos cenários e realidades.
Introduction: Pediatric patients with Acute Lymphoblastic Leukemia (ALL), the most common childhood cancer, require complex care, including multi-step therapy that may be challenging for the general population to comprehend. In this context, the lack of health literacy was identified as one of the causes of decreased health self-efficacy, with potential negative impacts on the treatment of diseases. Therefore, it is imperative to adopt strategies that can effectively assist patients and their caregivers in enhancing their knowledge about the therapeutic process of ALL. Objective: To describe the development and context of use of an educational material aimed at caregivers and pediatric patients diagnosed with ALL, undergoing the ALL IC-BFM 2009 treatment protocol. Method: Descriptive study on the preparation of booklets covering details about each of the medications to be administered during the mentioned protocol. The creation of the booklets was conducted by a pharmacist, reviewed by a pediatric oncologist, and further assessed by a second pharmacist. An analysis of textual readability was also conducted using the ALT© software. Results: Seven booklets were created, each corresponding to a phase of the ALL protocol. The booklets exhibited high readability, with text deemed simple, containing an average of 21.5% of complex words. Conclusion: It is believed that the material created can support the proper use of medications for children undergoing ALL treatment and can be enhanced or adapted to new scenarios and realities.
Introducción: Pacientes pediátricos con Leucemia Linfoblástica Aguda (LLA), el cáncer infantil más común, requieren cuidados complejos, que incluye una terapia de múltiples pasos difícil de entender para la población general. En este contexto, la falta de conocimiento en salud fue identificada como una de las causas de la disminución de la autoeficacia en salud, lo que podría impactar negativamente en el tratamiento de las enfermedades. Por lo tanto, es imperativo adoptar estrategias que puedan ayudar eficazmente a los pacientes y sus cuidadores a mejorar sus conocimientos sobre el proceso terapéutico de la LLA. Objetivo: Describir la elaboración y contexto de uso de un material educativo dirigido a cuidadores y pacientes pediátricos diagnosticados con LLA, sometidos al protocolo de tratamiento ALL IC-BFM 2009. Método: Estudio descriptivo sobre la elaboración de folletos que detallan cada uno de los medicamentos a ser administrado durante dicho protocolo. La creación de los folletos fue realizada por una farmacéutica, revisada por una oncóloga pediatra y un segundo farmacéutico. Además, se llevó a cabo un análisis de legibilidad textual mediante el softwareA LT©. Resultados: Se crearon siete folletos, cada uno asociado a una fase del protocolo LLA. Los folletos mostraron alta legibilidad, con un texto considerado simple y que contenía un promedio de 21,5% de palabras complejas. Conclusión: Se cree que el material creado puede respaldar el uso adecuado de medicamentos para niños en tratamiento contra la LLA y puede ser mejorado o adaptado a nuevos escenarios y realidades.
Subject(s)
Humans , Male , Female , Pediatrics/education , Professional-Patient Relations , Health Education , Patient Education Handout , Precursor Cell Lymphoblastic Leukemia-LymphomaABSTRACT
ABSTRACT Introduction: The treatment of acute lymphoblastic leukemia (ALL) has evolved in recent decades, reaching an overall survival rate close to 90%. Currently, approximately 4% of patients with ALL die from secondary complications of chemotherapy. Among these complications, the most frequent is febrile neutropenia (FN). The treatment of acute myeloid leukemias (AMLs) is even more aggressive, being consequently related to a considerable amount of treatment-related toxicity with a high risk of severe infection and death. Method: In order to reduce the infection-related risks in these groups of patients, systemic antibacterial prophylaxis has emerged as a possible approach. Results: Antibiotic prophylaxis during neutropenia periods in those undergoing chemotherapy have .already been proven in adults with acute leukemias (ALs). Among the possible available therapeutic options for bacterial prophylaxis in children with cancer, fluoroquinolones emerged with the most amount of evidence. Within this class, levofloxacin became the best choice. Conclusion: Therefore, the use of levofloxacin seems to be indicated in very specific situations: in children who are known to be neutropenic for a long time, secondary to intensive chemotherapy; in children with AL undergoing chemotherapy to induce remission; or in children undergoing hematopoietic stem cell transplantation (HSCT). This article aims to describe recent evidence focusing on antibiotic prophylaxis in children with ALs.
Subject(s)
Precursor Cell Lymphoblastic Leukemia-Lymphoma , PediatricsABSTRACT
ABSTRACT Introduction: Improving survival of Acute Lymphoblastic Leukemia (ALL) in adult patients has been a challenge. Despite intensive chemotherapy treatment, overall survival is poor. However, several studies demonstrate that young adult patients have better survival when treated with pediatric-based intensive regimens. Considering these results, We decided to treat newly diagnosed ALL patients according to age and risk factors. The goal of this study was to describe the results of this intensive chemotherapy treatment approach for ALL adult patients diagnosed at our institution. Methods: Fifty-eight ALL patients, diagnosed from 2004 to 2013, were included in the analysis. Patients were assigned to either the St. Jude Total Therapy XIIIB high-risk arm (St Jude) or the CALGB 8811 (CALGB). The Kaplan-Meier survival curve was used for the survival analyses and the Cox proportional hazard regression, for multivariable analysis. Results: The overall survival was 22.9% at 10 years. The St. Jude improved survival, compared to the CALGB (p = 0.007), with 32.6% vs. 7.4% survival rate at 10 years. However, no survival benefit was found for patients younger than 20 years old (p = 0.32). The multivariable analysis demonstrated that undetectable minimal residual disease (MRD) and hematopoietic stem cell transplantation (HSCT) had beneficial impact on survival (p = 0.0007 and p = 0.004, respectively). Conclusion: ALL is a disease of poor prognosis for adults. The joint effort to standardize treatment and seek solutions is the way to start improving this scenario.
Subject(s)
Precursor Cell Lymphoblastic Leukemia-LymphomaABSTRACT
La enfermedad fúngica invasora (EFI) es una de las principales causas de morbimortalidad en los pacientes pediátricos inmunocom- prometidos. Los hongos que con mayor frecuencia causan EFI en este grupo de pacientes corresponden a especies de Candida y Aspergillus. Sin embargo, en los últimos años se ha descrito un aumento de patógenos no clásicos, tales como Fusarium, Scedosporium, Mucorales, Cryptococcus, Trichosporon, entre otros. Se presenta un caso de EFI por Trichosporon asahii en un preescolar con una leucemia linfo- blástica aguda en quimioterapia de inducción. Además, se presenta una revisión actualizada de la literatura especializada, con énfasis en la importancia del diagnóstico precoz y el tratamiento antifúngico específico.
Invasive fungal disease (IFD) is one of the leading causes of morbidity and death among immunosuppressed pediatric patients. The fungi that most frequently cause IFD in this group of patients correspond to Candida and Aspergillus species, however, in recent years an increase in non-classical pathogens, such as Fusarium, Scedosporium, Mucorales, Cryptococcus, Trichosporon, among others. A case of invasive fungal disease caused by Trichosporon asahii is presented in a preschool patient with acute lymphoblastic leukemia in induction stage. This review highlights the importance of active search for pathogens in immunosuppressed patients, and proposes a specific treatment.
Subject(s)
Humans , Male , Child, Preschool , Precursor Cell Lymphoblastic Leukemia-Lymphoma/complications , Trichosporonosis/complications , Invasive Fungal Infections , Trichosporon/isolation & purification , Trichosporonosis/diagnosis , Trichosporonosis/microbiology , Trichosporonosis/drug therapy , Antifungal Agents/therapeutic useABSTRACT
Resumen Caso de un masculino de 53 años, con enfermedad por virus de inmunodeficiencia humana y con leucemia linfoblástica crónica, del que se aisló el microorganismo Listeria monocytogenes a partir de líquido cefalorraquídeo y hemocultivos. La condición leucémica junto con el síndrome de inmunodeficiencia fueron agravantes del cuadro clínico, ocasionando el deceso del paciente. Se analiza el cuadro clínico y las condiciones de fondo que favorecieron el curso de la infección bacteriana, así como las pruebas de laboratorio que permitieron encontrar el agente causal de la bacteriemia y meningitis.
Abstract Listeria monocytogenes was isolated from cerebrospinal fluid and blood cultures from a 54-year-old human immunodeficiency virus-positive male with lymphoblastic leukemia. The leukemic condition and its immunodeficient syndrome aggravated the clinical picture that led to the death of the patient. The clinical picture and the underlying conditions that favored the course of the bacterial infection are analyzed, and the laboratory tests that made it possible to find the underlying causal agent.
Subject(s)
Humans , Male , Middle Aged , HIV , Precursor Cell Lymphoblastic Leukemia-Lymphoma/diagnosis , Costa RicaABSTRACT
ANTECEDENTES: La obesidad se ha asociado con estado proinflamatorio de bajo grado que se ha relacionado con el desarrollo del cáncer en general incluyendo el hematológico. OBJETIVOS: El presente trabajo tiene el objetivo de identificar la asociación del diagnóstico de obesidad acorde al índice de masa corporal (IMC) con indicadores pronóstico de pacientes adultos con Leucemia Linfoblástica Aguda (LAL). PACIENTES Y MÉTODO: Se trata de un estudio observacional, retrospectivo que incluyó pacientes hospitalizados con diagnóstico de LAL de linaje de células B. Se estimó el IMC con base al peso y talla registrado al ingreso de los pacientes. Se determinó el riesgo de recaídas, recaídas a médula ósea y supervivencia. Se utilizó el método de Kaplan-Meier mediante el test log-Rank en el análisis estadístico. RESULTADOS: Se incluyeron 128 pacientes. El peso y el IMC no mostraron una asociación significativa con el riesgo de recaídas. La frecuencia de recaída a médula ósea fue del 43,8%. La obesidad no impactó con la supervivencia global (p = 0,640) ni en la supervivencia libre de enfermedad (p = 0,527). La presencia de obesidad no se comportó como una variable de riesgo de recaída (p = 0,873). El IMC con punto de corte de 30 kg/m2 no se comportó como un factor de riesgo de recaída (OR 1.078). Conclusión: La obesidad no es un factor de riesgo independiente para el pronóstico de los pacientes adultos portadores de Leucemia Linfoblástica Aguda de linaje B.
BACKGROUND: Obesity has been associated with a low-grade proinflammatory state, and it has been related to the development of cancer in general, including hematologic cancer. AIM: The present work aimed to identify the association of the diagnosis of obesity according to the body mass index (BMI) with prognostic factors of adult patients with Acute Lymphoblastic Leukemia (ALL). PATIENTS AND METHOD: This observational, retrospective study included hospitalized patients diagnosed with ALL of the B-cell lineages. BMI was estimated based on the weight and height registered on clinical records at the admission of the patients. The relapse risk and bone marrow relapse were determined, and the survival rate was measured. The statistical analysis included the Kaplan-Meier method using the log-Rank test. Results: This study included 128 clinical records of patients. Weight had no significant association with relapse risk. The frequency of bone marrow relapse was 43.8%. Obesity did not impact overall survival (p = 0.640) or disease-free survival (p = 0.527). The presence of obesity does not behave as a relapse risk variable (p = 0.873). BMI with a 30 kg/m2 cut-off point did not influence relapse risk (OR 1.078). CONCLUSION: Obesity is not an independent risk factor for the prognosis of adult patients with Acute Lymphoblastic Leukemia B-lineage.
Subject(s)
Humans , Male , Female , Adolescent , Adult , Middle Aged , Aged , Young Adult , Body Mass Index , Precursor Cell Lymphoblastic Leukemia-Lymphoma/complications , Precursor Cell Lymphoblastic Leukemia-Lymphoma/mortality , Obesity/complications , Prognosis , Recurrence , Retrospective Studies , Risk Factors , Disease-Free Survival , Kaplan-Meier EstimateABSTRACT
ntrodução:O câncer infantojuvenil corresponde a um grupo de várias doenças que têm em comum a proliferação descontrolada de células anormais e que pode ocorrer em qualquer local do organismo. Objetivo:Identificar os tipos de neoplasias mais frequentes na infância e adolescência e analisar o perfil clínico-epidemiológicodos pacientes. Metodologia:Estudo de transversal exploratório, de natureza aplicada com análise documental, realizado no Centro de Oncohematologia Pediátrica do Hospital Universitário Oswaldo Cruz, Recife, Pernambuco.Foram incluídos crianças e adolescentes diagnosticados com neoplasia e tratados por terapia antineoplásica.Os critérios de exclusão foram crianças e adolescentes normorreativas e/ou com doenças sistêmicas; prontuários ilegíveis ou com falta de informações clínicas.Resultados:Identificou-se que 54,21% dos pacientes eram dosexo feminino, seguido por 44,86% do sexo masculino.A faixa etária prevalente no estudo foi o de crianças de 5 a 14 anos (54,21%), ainda sobre o perfil dos pacientes, identificou-se que população autodeclarada como negra foi a mais prevalente representando 44,86% do total, seguido dos brancos com 43,93%. O diagnóstico que prevaleceu foi o de Leucemia Linfoide Aguda(23,36%), seguido pela Retinoblastoma (7,48%) e pela Rabdomiossarcoma embrionário (6,54%), e consequentemente o local da neoplasia primária que prevaleceu foi a Medula óssea (27,10%) seguido do olho (10,28%), deste total nota-se que o tratamento antineoplásico mais utilizado foi a quimioterapia (40,19%) seguido da quimioterapia associada à radioterapia(12,15%) e pela quimioterapia associada a cirurgia (10,28%). Conclusões:A leucemia linfoide aguda foi a neoplasia mais frequente na infância e adolescência, com prevalência na idade entre 5 e 14 anos, no sexo feminino e na etnia negra. A terapia antineoplásica mais utilizada foi a quimioterapia, seguida da associação entre quimioterapia e radioterapia (AU).
Introduction:Childhood cancer correspondsto a group of several diseases that have in common the uncontrolled proliferation of abnormal cells and that can occur anywhere in the body. Objective:Identify the most frequent types of neoplasms in childhood and adolescence and analyze the clinical-epidemiological profile of patients. Methodology:Exploratory cross-sectional study, applied in nature with document analysis, carried out at the Pediatric Oncohematology Center of Oswaldo Cruz University, Recife, Pernambuco. Children and adolescents diagnosed with neoplasia and treated with antineoplastic therapy were included. Exclusion criteria were normoreactive children and adolescents and/or with systemic diseases; illegible medical records or lacking clinical information. Results:It was identified that54.21% of the patients were female, followed by 44.86% male. The prevalent age group in the study was children from 5 to 14 years old (54.21%), still regarding the patients'profile , it was identified that the population self-declared as black was the most prevalent, representing 44.86% of the total, followed by of whites with 43.93%. The diagnosis that prevailed was Acute Lymphoid Leukemia (23.36%), followed by Retinoblastoma (7.48%) and Embryonic Rhabdomyosarcoma (6.54%), and consequently,the site of the primary neoplasm that prevailed was Bone marrow (27.10%) followed by the eye (10.28%), of this total it is noted that the most used anticancer treatment was chemotherapy (40.19%) followed by chemotherapy associated with radiotherapy (12.15% ) and chemotherapy associated with surgery (10.28%). Conclusions:Acute lymphoblastic leukemia was the most frequent neoplasm in childhood and adolescence, with a prevalence between 5 and 14 years of age, in females,and black ethnicity. The most used antineoplastic therapy was chemotherapy, followed by the association between chemotherapy and radiotherapy (AU).
ntroducción: El cáncer infantil corresponde a un grupo de varias enfermedades que tienen en común la proliferación descontrolada de células anormales y que pueden presentarse en cualquier parte del cuerpo. Objetivo: Identificar los tipos de neoplasias más frecuentes en la infancia y la adolescencia y analizar el perfil clínico-epidemiológico de los pacientes. Metodología: Estudio transversal exploratorio, aplicado en la naturaleza con análisis de documentos, realizado en el Centro de Oncohematología Pediátrica del Hospital Universitario Oswaldo Cruz, Recife, Pernambuco. Se incluyeron niños y adolescentes con diagnóstico de neoplasia y tratados con terapia antineoplásica. Los criterios de exclusión fueron niños y adolescentes normorreactivos y/o con enfermedades sistémicas; registros médicos ilegibles o carentes de información clínica. Resultados: Se identificó que el 54,21% de los pacientes eran del sexo femenino, seguido del 44,86% del masculino. El grupo etario prevalente en el estudio fueron los niños de 5 a 14 años (54,21%), en cuanto al perfil de los pacientes, se identificó que la población autodeclarada afrodescendiente fue la más prevalente, representando el 44,86% del total, seguido de los blancos con un 43,93%. El diagnóstico que predominó fue Leucemia Linfoide Aguda (23,36%), seguido de Retinoblastoma (7,48%) yRabdomiosarcoma Embrionario (6,54%), y en consecuencia el local de la neoplasia primaria que predominó fue Médula Ósea (27,10%) seguido de ocular (10,28%), de este total se destaca que el tratamiento anticancerígeno más utilizado fue la quimioterapia (40,19%) seguida de la quimioterapia asociada a radioterapia (12,15%) y la quimioterapia asociada a cirugía (10,28%). Conclusiones: La leucemia linfoblástica aguda fue la neoplasia más frecuente en la infancia y la adolescencia, con prevalencia entre los 5 y los 14 años, en el sexo femenino y en la etnia negra. La terapia antineoplásica más utilizada fue la quimioterapia, seguida de la asociación entre quimioterapia y radioterapia (AU).
Subject(s)
Humans , Male , Female , Child , Adolescent , Health Profile , Precursor Cell Lymphoblastic Leukemia-Lymphoma/pathology , Neoplasms/epidemiology , Antineoplastic Agents/therapeutic use , Medical Records , Cross-Sectional Studies/methods , Document Analysis , Hospitals, PediatricABSTRACT
El metotrexato es un fármaco análogo del ácido fólico ampliamente utilizado en el tratamiento de enfermedades autoinmunes, leucemias y linfomas. Su uso puede ocasionar la aparición de múltiples efectos adversos entre los que se encuentran aquellos relacionados con la presencia de toxicidad neurológica, que puede presentarse de forma aguda, subaguda o crónica. La neurotoxicidad subaguda es aquella que ocurre típicamente entre los 2 y los 14 días posteriores a la administración y puede manifestarse con una amplia gama de síntomas neurológicos. En la mayoría de los casos, no recurre con futuras exposiciones al medicamento. Presentamos tres casos de neurotoxicidad subaguda por metotrexato con manifestaciones clínicas diferentes en pacientes oncohematológicos que se internaron entre los años 2018 y 2020. Dos de ellos presentaron recurrencia frente a la nueva administración del fármaco y todos evidenciaron lesiones en resonancia magnética nuclear.
Methotrexate is a folic acid analogue widely used in the treatment of autoimmune diseases, leukemias, and lymphomas. Methotrexate use may cause multiple adverse effects, including those related to the presence of neurological toxicity, which may be acute, subacute, or chronic. Subacute neurotoxicity typically occurs between 2 and 14 days after administration and may present as a wide range of neurological symptoms. In most cases, it does not recur with future exposures to the drug. Here we describe 3 cases of subacute methotrexate neurotoxicity with different clinical manifestations in patients with oncohematological disease who were hospitalized between 2018 and 2020. Two of them showed recurrence with a new drug administration. Lesions were observed in the magnetic resonance imaging tests of all of them.
Subject(s)
Humans , Male , Child , Adolescent , Neurotoxicity Syndromes/etiology , Neurotoxicity Syndromes/pathology , Neurotoxicity Syndromes/drug therapy , Precursor Cell Lymphoblastic Leukemia-Lymphoma/drug therapy , Lymphoma , Magnetic Resonance Imaging , Methotrexate/adverse effects , Antimetabolites, Antineoplastic/adverse effectsABSTRACT
Introducción: La infiltración del nervio óptico como forma inicial de recaída de la leucemia linfoblástica aguda es rara, aunque altamente indicativa de que el sistema nervioso central está involucrado. Objetivo: Actualizar el conocimiento sobre infiltración del nervio óptico como forma inicial de recaída de la leucemia linfoblástica aguda. Métodos: Se realizó una revisión de las publicaciones más relevantes relacionadas con el tema durante los últimos años. La búsqueda y la localización de la información se apoyaron en la elección de palabras clave/descriptores que configuraron el perfil de búsqueda. Se utilizó el MeSH Database de PubMed. Se realizó una extensa revisión en Google Académico y otros megabuscadores de revisión sistemática mediante TripDatabase y Cochrane. Conclusiones: La infiltración directa de células tumorales y las alteraciones hematológicas propias de la enfermedad constituyen los mecanismos fundamentales de la fisiopatogenia. El edema del disco óptico es su signo oftalmoscópico más distintivo. La imagen por resonancia magnética de cráneo, el análisis citológico del fluido cerebroespinal y la biopsia de médula ósea negativas no deben excluir el diagnóstico. La terapia combinada que incluye la radiación localizada constituye una buena opción de tratamiento. Un número considerable de ojos recuperan su agudeza visual y muestran resolución del cuadro infiltrativo(AU)
Introduction: Optic nerve infiltration as an initial form of relapse of acute lymphoblastic leukemia is rare, although it is highly indicative of central nervous system involvement. Objective: To update the knowledge about optic nerve infiltration as an initial form of relapse of acute lymphoblastic leukemia. Methods: A review of the most relevant publications related to the subject during the last years was carried out. The search and localization of information was supported by the choice of keywords/descriptors that configured the search profile. The MeSH Database of PubMed was used. An extensive review was performed in Google Scholar and other systematic review mega search engines using TripDatabase and Cochrane. Conclusions: Direct tumor cell infiltration and disease-specific hematologic alterations are the fundamental mechanisms of pathophysiology. Optic disc edema is the most distinctive ophthalmoscopic sign. Negative cranial magnetic resonance imaging, cytologic analysis of cerebrospinal fluid and bone marrow biopsy should not exclude the diagnosis. Combination therapy including localized radiation is a good treatment option. A considerable number of eyes recover visual acuity and show resolution of the infiltrative picture(AU)
Subject(s)
Humans , Biopsy/methods , Magnetic Resonance Imaging/methods , Precursor Cell Lymphoblastic Leukemia-Lymphoma/physiopathology , Review Literature as Topic , Databases, BibliographicABSTRACT
Introdução: O tratamento da leucemia linfoblástica aguda (LLA) é relacionado a eventos adversos (EA) e mortalidade por toxicidade dos medicamentos utilizados. Protocolos com L-asparaginase (L-Asp) têm demonstrado melhor prognóstico, porém podem causar hipersensibilidade e desenvolvimento de anticorpos neutralizantes por ser produzida a partir da Escherichia coli. A conjugação de E. coli L-Asp com monometoxipolietilenoglicol resulta na PEG-Asp, com menor imunogenicidade e maior meia-vida. Objetivo: Avaliar eficácia e segurança da PEG-Asp, em comparação à L-Asp, no tratamento de LLA, e sua viabilidade econômica para subsidiar a tomada de decisão quanto à sua incorporação. Material e Métodos: Estudo descritivo de síntese de evidências e avaliação econômica. Busca de evidências foi realizada no MEDLINE, Cochrane Library, Embase, Epistemonikos, e agências de avaliação de tecnologias em saúde. Foram considerados elegíveis revisões sistemáticas, ensaios clínicos e estudos observacionais, publicados em inglês, espanhol e português, independente da data. Viabilidade econômica foi calculada a partir do custo do uso da PEG-Asp no protocolo GRAALL 2003 frente ao valor faturado via Autorização de Procedimentos de Alta Complexidade. Resultados: As evidências demonstraram eficácia semelhante entre PEG-Asp e L-Asp para maioria dos desfechos de interesse, com superioridade na prevenção de leucemia no sistema nervoso central em adultos e comodidade posológica. PEG-Asp demonstrou maior frequência de EA em adultos recém diagnosticados, e ausência de diferença na toxicidade e mortalidade nos recidivados. A incorporação da PEG-Asp se mostrou economicamente viável para pacientes adultos, e desvantajosa naqueles com 18 e 19 anos incompletos, considerando superfície corpórea média de 1,7m². Conclusão: Recomendou-se a incorporação de PEG-Asp para tratamento de LLA em pacientes acima de 18 anos e naqueles com 18 a 19 anos incompletos, deve-se avaliar a viabilidade econômica em função da superfície corpórea. Além do perfil de eficácia e segurança da PEG-Asp, não há medicamentos dessa classe terapêutica com registro para adultos na Agência Nacional de Vigilância Sanitária.
Introduction: Treatment of acute lymphoblastic leukemia (ALL) is associated with adverse events (AEs) and mortality due to toxicity of drugs used. Protocols with L-asparaginase (L-Asp) have shown improved prognosis, but can cause hypersensitivity and development of neutralizing antibodies, as L-Asp is produced from Escherichia coli. The conjugation of E. coli L-Asp with monomethoxypolyethylene glycol results in PEG-Asp, with lower immunogenicity and longer half-life. Objective: To evaluate the efficacy and safety of PEG-Asp, compared to L-Asp, in ALL treatment, and its economic viability in order to subsidize decision making regarding its incorporation. Material and Methods: Descriptive study of evidence synthesis and economic evaluation. Evidence was searched in MEDLINE, Cochrane Library, Embase, Epistemonikos, and Health technology assessment agencies. Systematic reviews, clinical trials and observational studies published in English, Spanish and Portuguese, regardless of date, were considered eligible. Economic viability was calculated based on the cost of using PEG-Asp in GRAALL - 2003 protocol compared to the amount billed via High-complexity Procedures Authorization. Results: Evidence showed similar efficacy between PEG-Asp and L-Asp for most of the outcomes of interest, with superiority in prevention of Central Nervous System leukemia in adults and in dosage convenience. PEG-Asp showed a higher frequency of AEs in newly diagnosed adults, and no difference in toxicity and mortality in relapsed adults. The incorporation of PEG-Asp proved to be economically viable for adult patients, and disadvantageous for patients between 18 and 19 years of age, considering a mean body surface area of 1.7m². Conclusion: Incorporation of PEG-Asp for the treatment of ALL in patients over 18 years was recommended, and in those aged 18 to 19 years incomplete, the economic viability should be assessed according to body surface area. In addition to the efficacy and safety profile of PEG-Asp, there are no drugs in this therapeutic class for adults registered within ANVISA.
Subject(s)
Precursor Cell Lymphoblastic Leukemia-Lymphoma , Technology Assessment, Biomedical , Costs and Cost Analysis , Health Management , Drug-Related Side Effects and Adverse Reactions , Evaluation of the Efficacy-Effectiveness of InterventionsABSTRACT
Abstract Introduction Hyperglycemia occurs in Acute Lymphoblastic Leukemia (ALL) due to chemotherapeutic agents and may be stress-induced. Given the potential impact of hyperglycemia on the clinical outcomes of ALL patients, we sought to determine the association of hyperglycemia with the development of infectious complications. Methods This is a retrospective cohort involving adult Filipino ALL patients admitted at a tertiary referral center. Patients were stratified according to blood glucose levels and infections were classified into microbiologically and clinically defined infections. Logistic regression was performed to determine whether hyperglycemia was associated with the development of infectious complications. Results Of the 174 patients admitted for ALL, only 76 patients (44%) underwent blood glucose monitoring and were thus included in this study. Hyperglycemia was observed in 64 patients (84.21%). Infectious complications were seen in 56 patients (73.68%), of whom 37 patients (48.68%) had microbiologically defined infections and 19 (25%) had clinically defined infections. The respiratory tract was the most common site of infection and gram-negative bacteria were the predominant isolates. Hyperglycemia significantly increased the likelihood of infectious complications, particularly at blood glucose levels ≥ 200 mg/dL. Conclusion Hyperglycemia is associated with an increased likelihood of infectious complications in Filipino ALL patients. With sepsis being one of the main causes of mortality in this population, our study provides compelling evidence for us to consider routine blood glucose monitoring in order to manage and potentially decrease the occurrence of infections in these patients.