Acute promyelocytic leukemia in childhood and adolescence: treatment results of a modified AIDA protocol at a Brazilian center

ABSTRACT Introduction: Acute promyelocytic leukemia currently presents an excellent chance of cure with protocols based on all-trans-retinoic acid (ATRA) and anthracycline or only differentiation agents. However, high early mortality rates continue to be reported Methods: Between 2000 and 2018, patients were enrolled and retrospectively analyzed by medical records. A modified AIDA protocol, with a 1-year shortening of the treatment duration, reduction in the number of drugs and a strategy to reduce early mortality by the postponement of the initiation of anthracyclines were employed. Overall and event-free survival rates and toxicity were analyzed Results: Thirty-two patients were enrolled, of whom 56% were female, with a median age of 12 years and 34% belonged to the high-risk group. Two patients had the hypogranular variant and three had another cytogenetic alteration, in addition to the t(15;17). The median start of the first anthracycline dose was 7 days. There were two early deaths (6%) due to central nervous system (CNS) bleeding. All patients achieved molecular remission after the consolidation phase. Two children relapsed and were rescued by arsenic trioxide and hematopoietic stem cell transplantation. The presence of disseminated intravascular coagulation (DIC) at diagnosis (p = 0.03) was the only factor with survival impact. The five-year event-free survival (EFS) was 84% and 5-year overall survival (OS) was 90% Conclusion: The survival results were comparable to those found in the AIDA protocol, with a low rate of early mortality in relation to the Brazilian reality.

Prevalence and outcomes of thrombotic and hemorrhagic complications in pediatric acute promyelocytic leukemia in a tertiary Brazilian center

Abstract Introduction: Little attention is given to thrombosis associated with pediatric acute promyelocytic leukemia (APL). This study describes the thrombotic and hemorrhagic manifestations of APL in pediatric patients and evaluates their hemostasis, based on coagulation tests. Methods: Inclusion criteria were age 0-18 years and APL diagnosis between April 2005 and November 2017. Patients who had received blood transfusion prior to coagulation tests were excluded. Baseline coagulation tests, hematologic counts, and hemorrhagic/thrombotic manifestations were evaluated. Results: Median age was 10.7 years (1-15 years). The initial coagulation tests revealed a median Hgb of 8.3 g/dL (4.7-12.9 g/dL), median leucocyte count of 10.9 × 109/L (1.1-95.8 × 109/L), median platelet count of 31.8 × 109/L (2.0-109.0 × 109/L), median activated partial thromboplastin time (aPTT) of 31.7 s (23.0-50.4 s), median aPTT ratio of 1.0 (0.78-1.6), median thromboplastin time (PT) of 17.5 s (13.8-27.7 s), median PT activity of 62% (25-95 %), and median fibrinogen of 157.7 mg/dL (60.0-281.0 mg/dL). Three patients (13%) had thrombosis. At diagnosis, 21 patients (91.3%) had bruising, one patient (4.3%) had splenic vein and artery thrombosis and one patient (4.3%) presented without thrombohemorrhagic manifestations. During treatment, two patients (8.6%) had thrombosis. Conclusion: Knowledge of thrombosis in pediatric APL is important to determine its risk factors and the best way to treat and prevent this complication.

Differences in children and adolescents with SARS-CoV-2 infection: a cohort study in a Brazilian tertiary referral hospital

OBJECTIVES: To compare demographic/clinical/laboratory/treatments and outcomes among children and adolescents with laboratory-confirmed coronavirus disease 2019 (COVID-19). METHODS: This was a cross-sectional study that included patients diagnosed with pediatric COVID-19 (aged <18 years) between April 11, 2020 and April 22, 2021. During this period, 102/5,951 (1.7%) of all admissions occurred in neonates, children, and adolescents. Furthermore, 3,962 severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2) detection samples were processed in patients aged <18 years, and laboratory-confirmed COVID-19 occurred in 155 (4%) inpatients and outpatients. Six/155 pediatric patients were excluded from the study. Therefore, the final group included 149 children and adolescents (n=97 inpatients and 52 outpatients) with positive SARS-CoV-2 results. RESULTS: The frequencies of sore throat, anosmia, dysgeusia, headache, myalgia, nausea, lymphopenia, pre-existing chronic conditions, immunosuppressive conditions, and autoimmune diseases were significantly reduced in children and adolescents (p<0.05). Likewise, the frequencies of enoxaparin use (p=0.037), current immunosuppressant use (p=0.008), vasoactive agents (p=0.045), arterial hypotension (p<0.001), and shock (p=0.024) were significantly lower in children than in adolescents. Logistic regression analysis showed that adolescents with laboratory-confirmed COVID-19 had increased odds ratios (ORs) for sore throat (OR 13.054; 95% confidence interval [CI] 2.750-61.977; p=0.001), nausea (OR 8.875; 95% CI 1.660-47.446; p=0.011), and lymphopenia (OR 3.575; 95% CI 1.355-9.430; p=0.010), but also had less hospitalizations (OR 0.355; 95% CI 0.138-0.916; p=0.032). The additional logistic regression analysis on patients with preexisting chronic conditions (n=108) showed that death as an outcome was significantly associated with pediatric severe acute respiratory syndrome (SARS) (OR 22.300; 95% CI 2.341-212.421; p=0.007) and multisystem inflammatory syndrome in children (MIS-C) (OR 11.261; 95% CI 1.189-106. 581; p=0.035). CONCLUSIONS: Half of the laboratory-confirmed COVID-19 cases occurred in adolescents. Individuals belonging to this age group had an acute systemic involvement of SARS-CoV-2 infection. Pediatric SARS and MIS-C were the most important factors associated with the mortality rate in pediatric chronic conditions with COVID-19.

An unusual abdominal wall mass in a child

ABSTRACT Abdominal tumors are one of the most common types of pediatric cancer. Therefore, they should always be included in the differential diagnosis of abdominal masses. Here, we present the case of a child whose initial hypothesis of diagnosis contemplated this possibility. Later, it was demonstrated that the abdominal mass found was secondary to a common parasitosis. A 2-year old, moderately malnourished and pale white boy was referred with a history of a rapidly growing, well-limited, middle abdominal mass. The mass was 10 by 3 cm, hard and poorly movable, apparently involving both abdominal rectus muscles. A complete resection was performed, revealing an abdominal wall abscess, with intense eosinophilic proliferation, secondary to a local and intense reaction to innumerous Ascaris lumbricoides eggs. Extra luminal infestations with Ascaris, that usually form peritoneal granulomas have been previously described. However, neither external trauma nor fistula, that could explain the superficial presence of the eggs, was found. This description reinforces the relevance of infectious diseases within the differential diagnosis of abdominal masses, particularly in areas with high prevalence of parasitic infestations.

Comparative study of quality of life of adult survivors of childhood acute lymphocytic leukemia and Wilms’ tumor / Estudo comparativo da qualidade de vida de adultos sobreviventes de leucemia linfocítica aguda e tumor de Wilms na infância

Abstract Objective To analyze and compare the health-related quality of life of adult survivors of acute lymphocytic leukemia and Wilms’ tumor amongst themselves and in relation to healthy participants. Methods Ninety participants aged above 18 years were selected and divided into three groups, each comprising 30 individuals. The Control Group was composed of physically healthy subjects, with no cancer history; and there were two experimental groups: those diagnosed as acute lymphocytic leukemia, and those as Wilms’ Tumor. Quality of life was assessed over the telephone, using the Medical Outcomes Study 36-Item Short Form Health Survey. Results Male survivors presented with better results as compared to female survivors and controls in the Vitality domain, for acute lymphocytic leukemia (p=0.042) and Wilms’ tumor (p=0.013). For acute lymphocytic leukemia survivors, in Social aspects (p=0.031), Mental health (p=0.041), and Emotional aspects (p=0.040), the latter also for survivors of Wilms’ tumor (p=0.040). The best results related to the Functional capacity domain were recorded for the experimental group that had a late diagnosis of acute lymphocytic leukemia. There were significant differences between groups except for the Social and Emotional domains for self-perceived health, with positive responses that characterized their health as good, very good, and excellent. Conclusion Survivors of acute lymphocytic leukemia showed no evidence of relevant impairment of health-related quality of life. The Medical Outcomes Study 36-Item Short Form Health Survey (via telephone) can be a resource to access and evaluate survivors.

Resumo Objetivo Analisar e comparar a qualidade de vida relacionada à saúde de sobreviventes adultos de leucemia linfocítica aguda e tumor de Wilms entre si, e em relação a participantes sadios. Métodos Foram selecionados noventa participantes, acima de 18 anos, os quais foram divididos em três grupos, sendo cada um com 30 sujeitos: Grupo Controle, que contou com indivíduos fisicamente saudáveis, sem histórico oncológico; grupo experimental formado por pacientes que tiveram diagnóstico de leucemia linfocítica aguda; e grupo experimental formado por pacientes que tiveram diagnóstico de Tumor de Wilms. A avaliação da qualidade de vida foi realizada por telefone e utilizou o Medical Outcomes Study 36-Item Short Form Health Survey. Resultados Os sobreviventes do sexo masculino apresentaram melhores resultados em relação aos do sexo feminino e controles no Aspecto vitalidade, para leucemia linfocítica aguda (p=0,042) e tumor de Wilms (p=0,013). Para os sobreviventes de leucemia linfocítica aguda nos Aspectos sociais (p=0,031), Saúde mental (p=0,041) e Aspectos emocionais (p=0,040), neste último também para as sobreviventes de Tumor de Wilms (p=0,040). Os melhores resultados relacionados ao domínio Capacidade funcional foram registrados para o grupo experimental de pacientes que tiveram diagnóstico tardio de leucemia linfocítica aguda. Observaram-se diferenças significativas entre os grupos, exceto para os domínios Aspectos sociais e emocionais para a percepção da própria saúde, que teve respostas de cunho positivo, que qualificavam a própria saúde como boa, muito boa e excelente. Conclusão O grupo experimental de pacientes que tiveram diagnóstico de leucemia linfocítica aguda não apresentou evidências de comprometimento relevante da qualidade de vida relacionada à saúde. O Medical Outcomes Study 36-Item Short Form Health Survey (via telefone) pode ser um recurso de acesso e avaliação de sobreviventes.

New concepts and outcomes for children with hepatoblastoma based on the experience of a tertiary center over the last 21 years

OBJECTIVE: The aim of this study was to summarize the experience of a tertiary center in treating hepatoblastoma for the last 21 years. PATIENTS AND METHODS: Fifty-eight cases were included. The tumor extent and prognosis were assessed using the PRETEXT system. The following data were analyzed: age at diagnosis, comorbidities, prematurity, treatment modalities, histopathological findings, surgical details and complications, treatment outcomes, chemotherapy schedules, side effects and complications. Treatment outcomes included the occurrence of local or distant recurrence, the duration of survival and the cause of death. The investigation methods were ultrasonography, CT scan, serum alpha-fetoprotein level measurement and needle biopsy. Chemotherapy was then planned, and the resectability of the tumor was reevaluated via another CT scan. RESULTS: The mean numbers of neoadjuvant cycles and postoperative cycles of chemotherapy were 6±2 and 1.5±1.7, respectively. All children except one were submitted for surgical resection, including 50 partial liver resections and 7 liver transplantations. Statistical comparisons demonstrated that long-term survival was associated with the absence of metastasis (p=0.04) and the type of surgery (resection resulted in a better outcome than transplantation) (p=0.009). No associations were found between vascular invasion, incomplete resection, histological subtype, multicentricity and survival. The overall 5-year survival rate of the operated cases was 87.7%. CONCLUSION: In conclusion, the experience of a Brazilian tertiary center in the management of hepatoblastoma in children demonstrates that long survival is associated with the absence of metastasis and the type of surgery. A multidisciplinary treatment involving chemotherapy, surgical resection and liver transplantation (including transplantations using tissue from living donors) led to good outcomes and survival indexes. .

Avaliação da qualidade de vida de sobreviventes de leucemia na infância / Evaluation of quality of life of survivors of childhood leukemia

O progressivo sucesso dos esquemas terapêuticos para neoplasias malignas de início na infância permitiu uma melhora significativa da sobrevida dos portadores nas últimas décadas. Contudo, sequelas tardias secundárias à toxicidade destes esquemas devem ser devidamente monitoradas. Dada a relevância da avaliação de aspectos da saúde física, saúde mental, bem como fatores emocionais e sociais dos sobreviventes, avaliou-se a qualidade de vida dos entrevistados por uma abordagem alternativa: o contato telefônico. Aplicou-se o questionário SF-36 (Medical Outcomes Study 36 – Item Short Form Health Survey) validado para a língua portuguesa, em trinta indivíduos, maiores de 18 anos e de ambos os sexos, sobreviventes de leucemia linfocítica aguda, sem terapia antineoplásica há pelo menos cinco anos, em acompanhamento médico no Ambulatório Fora de Terapia do ITACI (grupo experimental), e em trinta indivíduos sadios, pareados por sexo e idade ao grupo experimental (grupo controle). Os sobreviventes analisados apresentaram todos os aspectos do SF-36 superiores ao valor médio. Os resultados médios do total de pontos foram estatisticamente semelhantes ao do grupo controle. O SF-36 proporciona dados valiosos para que a equipe de saúde estabeleça condutas terapêuticas aos sobreviventes. A avaliação por telefone se mostrou importante recurso no acompanhamento ambulatorial desta população. Os sobreviventes de Leucemia Linfocítica Aguda avaliados apresentaram adequados escores de qualidade de vida na atualidade.

The progressive success of therapeutic plans for malignant neoplasias that started in childhood allowed an important increase in the survival rate of carriers during the last decades. However, late sequelae secondary to the toxicity of such plans shall be duly monitored. Considering the importance of evaluating the aspects of physical and mental health, as well as emotional and social factors of the survivors, the quality of life of the interviewed persons was evaluated by an alternative approach: telephone calls. The SF-36 questionnaire (Medical Outcomes Study 36 – Item Short Form Health Survey), validated for the Portuguese language, was applied to thirty persons with more than 18 years of age and of both sexes, survivors of acute lymphocytic leukemia, without antineoplastic therapy for at least five years, under medical attendance at the Ambulatory outside the ITACI therapy (experimental group), and also to thirty healthy persons, matched by sex and age to the experimental group (control group). Analyzed survivors scored higher in all aspects SF-36 than the average value. The average results of the total points were statistically similar to the control group. SF-36 provides valuable data for the health team to establish therapeutic procedures to survivors. Evaluation by phone became an important resource in the ambulatory follow-up of this population. Survivors of Acute Lymphocytic Leukemia are presently showing appropriate scores of quality of life.

Discrimination of acute lymphoblastic leukemia from systemic-onset juvenile idiopathic arthritis at disease onset

OBJECTIVE: To assess clinical and laboratory features that differentiate acute lymphoblastic leukemia from systemic juvenile idiopathic arthritis at disease onset. METHODS: Fifty-seven leukemia patients with musculoskeletal involvement, without blasts on peripheral blood and without glucocorticoid therapy at disease onset and 102 systemic juvenile idiopathic arthritis patients (International League of Associations for Rheumatology criteria) were retrospectively evaluated. The following features were examined: fever, rheumatoid rash, arthritis, limb pain, hepatomegaly, splenomegaly, pericarditis, myocarditis, pleuritis, weight loss, bleeding, anemia, leukopenia, neutropenia, thrombocytopenia, erythrocyte sedimentation rate, and lactic dehydrogenase levels. RESULTS: The median age at disease onset was significantly higher in leukemia patients than in those with systemic-onset juvenile idiopathic arthritis (5.8 vs. 3.8 years). In addition, the frequencies of limb pain, hepatomegaly, weight loss and hemorrhagic manifestations were significantly higher in leukemia patients than in systemic-onset juvenile idiopathic arthritis patients (70 percent vs. 1 percent, 54 percent vs. 32 percent, 30 percent vs. 8 percent, and 9 percent vs. 0 percent, respectively). Likewise, the frequencies of anemia, leukopenia, neutropenia, thrombocytopenia and high lactic dehydrogenase levels were statistically higher in leukemia patients than in patients with systemic-onset juvenile idiopathic arthritis (88 percent vs. 57 percent, 39 percent vs. 1 percent, 60 percent vs. 1 percent, 77 percent vs. 1 percent, and 56 percent vs. 14 percent, respectively). Remarkably, multivariate analysis revealed that limb pain (OR = 553; 95 percent CI =46.48-6580.42) and thrombocytopenia (OR = 754.13; 95 percent CI =64.57-8806.72) were significant independent variables that differentiated leukemia from systemic-onset juvenile idiopathic arthritis. The R2 of the Nagelkerke test was 0.91, and the Kaplan-Meier survival curves were similar for acute lymphoblastic leukemia patients with and without limb pain. CONCLUSION: Our study emphasizes the importance of investigating leukemia in patients presenting with musculoskeletal manifestations and, in particular, limb pain associated with thrombocytopenia.

Manifestações neoplásicas em rinofaringe / Neoplasic manifestation in rhinopharynx

As neoplasias primárias de nasofaringe são raras na infância e adolescência. Sintomas como obstrução nasal persistente, cefaleia, epistaxes recorrentes e abaulamento paranasal ou de palato são comuns à maioria delas e podem ser confundidos com outras doenças infecciosas ou alérgicas.As neoplasias mais frequentes na faixa etária pediátrica são os rabdomiossarcomas, os linfomas não Hodgkin, o estesioneuroblastoma, o carcinoma de nasofaringe e o angiofibroma juvenil. A biópsia ou ressecção da lesão quando possível, seguida de tratamento quimio e radioterápico, quando indicado, compõem o tratamento destas neoplasias.

Linfoma de Hodgkin na infância e adolescência: 15 anos de experiência com o protocolo DH-II-90 / Hodgkin's lymphoma in children and adolescents: 15 years of experience with the DH-II-90 protocol

O desafio do tratamento do linfoma de Hodgkin na infância reside na redução da toxicidade aguda e tardia sem afetar os bons resultados terapêuticos. Crianças e adolescentes portadores de linfoma de Hodgkin recém-diagnosticado foram tratados com o protocolo institucional DH-II-90. Os objetivo deste trabalho foram: 1)avaliar as taxas de sobrevida global (SG) e livre de eventos (SLE) do protocolo DH-II-90 aplicado a portadores de LH; 2)avaliar as taxas de SG e SLE conforme estádio, idade, tumor "bulky", massa mediastinal, sintomas B, dose de radioterapia e 3)descrever os efeitos tardios. Sessenta e oito pacientes portadores de LH recém-diagnosticado, com idade entre 0 e 21 anos (idade mediana 9 anos, 20F:48M), foram tratados com quimioterapia (baixo risco:ABVD; alto risco:ABVD+MOP/COP) e radioterapia. O estadiamento foi distribuído desta forma: nove (13,2 por cento) estádio I A; 29 (42,6 por cento) II A; cinco (7,4 por cento) II B; nove (13,2 por cento) III A; dez (14,7 por cento) III B; dois (2,9 por cento) IV A e quatro (5,9 por cento) IV B. A SG em dez anos foi de 96,1 por cento ± 3,8 para o grupo de baixo risco e 93,3 por cento ± 4,5 para o de alto risco (p:0,402). A SLE foi de 88,9 por cento ± 5,2 em dez anos para o de alto risco e 86,5 por cento ± 6,3 para o de baixo risco (p: 0,969). A presença de massa mediastinal e doses de radioterapia maiores que 2100 cGy (p= 0,020 e p= 0,014, respectivamente) apresentam impacto negativo na SLE e a doença estádio I tem impacto positivo na SLE. Disfunção e carcinoma de tireoide são os efeitos tardios mais frequentes neste grupo de doentes. O protocolo DH-II-90 obteve resultados terapêuticos favoráveis, porém as taxas de complicações tardias, embora aceitáveis, demandam revisão do programa terapêutico.

The challenge of new protocols for Hodgkin's lymphoma (HL) treatment is to decrease the toxicity without impairing the results. The DH-II-90 protocol was designed to treat children and adolescents with HL. The objectives of this work were: 1) to assess the overall and event free survival of patients with newly diagnosed HL treated with the DH-II-90 protocol, 2) to assess the overall and event free survival by stage, age, presence of bulky disease, mediastinal mass, B symptoms, dose and type of radiotherapy, and 3) to describe late effects, data collected from the patients' charts. Sixty-eight patients with HL, from 0 to 21 years of age (median age 9 yr, 20F:48M) were treated with ABVD and involved-field radiotherapy for low-risk patients, and ABVD plus MOP or COP and extended field radiotherapy for high-risk patients. Stage distribution was: nine (13.2 percent) stage I A; 29 (42.6 percent) II A; five (7.4 percent) II B; nine (13.2 percent) III A; ten (14.7 percent) III B; two (2.9 percent) IV A and four (5.9 percent) IV B. The 10-year overall survival was 96.1 percent ± 3.8 percent for the low-risk group and 93.3 percent ± 4.5 percent for the high-risk group (p= 0.402). The 10-year event free survival was 88.9 percent ± 5.2 percent for high-risk and 86.5 percent ± 6.3 percent for low-risk patients (p= 0.969). The presence of mediastinal mass and more than 2100 cGy radiation doses had negative impact on event free survival (p= 0.020 and p= 0.014, respectively).Thyroid gland dysfunction was frequently observed and there were two cases of thyroid carcinoma. The DH-II-90 protocol is effective, but, due to the late effects presented by this group of patients, further modifications of the therapy schedule are required.

Retinoblastoma: a three-year-study at a Brazilian medical school hospital

OBJECTIVE: To present the characteristics and treatment outcomes of patients with retinoblastoma. METHODS: A retrospective case series was conducted to review the records of all new patients diagnosed with retinoblastoma between 2003 and 2005. Eyes with early disease, or advanced disease with potential vision were treated with chemotherapy (carboplatin and etoposide) in conjunction with early local therapy (laser or cryo). Radiotherapy was used in cases where the disease did not respond to the above protocols or in recurrent cases. Eyes in the late stage of disease with no potential vision in the initial examination or eyes and where conservative treatment had failed were enucleated. RESULTS: In total, we reviewed 28 new cases of retinoblastoma, 15 of which were unilateral and 13 of which were bilateral (46 percent). These data correspond to a mean of 9.3 new cases per year (0.77 case/ month). The mean age at diagnosis was 33.8 months for unilateral cases, and 19.15 months for bilateral cases (p=0.015). Leucocoria was the major presenting symptom (75 percent). All but one patient with unilateral disease had the affected eye enucleated due to advanced disease (mean follow-up: 39.91 months). Among the 13 bilateral cases, 13 eyes (50 percent) were enucleated, 11 eyes (42.4 percent) were saved with chemotherapy in conjunction with local therapy and 2 eyes (7.6 percent) were saved using external beam radiotherapy (mean follow-up: 41.91 months). In unilateral and bilateral disease, pathology data revealed choroid involvement in 50 percent and 30 percent, respectively, and optic nerve invasion in 92 percent and 50 percent, respectively. CONCLUSION: In this population, retinoblastoma was diagnosed too late and most eyes were consequently enucleated. In cases with bilateral disease, half of the eyes were preserved.

Registry of Hospital das Clínicas of the University of São Paulo Medical School: first official solid organ and tissue transplantation report - 2008

OBJECTIVE: The aim of this study was to report a single center experience of organ and tissue transplantation INTRODUCTION: This is the first report of organ and tissue transplantation at the Hospital das Clínicas of the University of Sao Paulo Medical School. METHODS: We collected data from each type of organ transplantation from 2002 to 2007. The data collected were patient characteristics and actuarial survival Kaplan-Meier curves at 30 days, one year, and five years RESULTS: There were a total of 3,321 transplants at our institution and the 5-year survival curve ranged from 53 percent to 88 percent. CONCLUSION: This report shows that solid organ and tissue transplants are feasible within the institution and allow us to expect that the quality of transplantation will improve in the future.

Comprometimento musculoesquelético como primeira manifestação de neoplasias / Musculoskeletal involvement as a first manifestation of neoplasm disease

OBJETIVO: Identificar pacientes portadores de neoplasias encaminhados ao ambulatório de reumatologia pediátrica por apresentarem sintomas musculoesqueléticos como manifestação inicial, bem como apontar os achados clínicos, laboratoriais e radiológicos que contribuíram para o esclarecimento diagnóstico. MÉTODOS: Foi realizada análise retrospectiva dos prontuários dos pacientes com diagnóstico final de neoplasia, que foram avaliados na Unidade de Reumatologia do Instituto da Criança - FMUSP entre janeiro de 1983 e dezembro de 2006. Foram registrados os dados referentes às queixas musculoesqueléticas, exame físico, provas laboratoriais e procedimentos radiológicos e invasivos realizados. RESULTADOS: Dos 4876 pacientes, 25 (0,5 por cento) crianças foram estudadas, sendo que 52 por cento apresentavam leucemia linfóide aguda - LLA e 24 por cento neuroblastoma. Vinte crianças (80 por cento) apresentaram artrite e/ou artralgia no início da doença. Todos os pacientes apresentavam sintomas constitucionais, sendo febre o mais prevalente (22 casos - 88 por cento). O hemograma inicial estava alterado em 16 pacientes (64 por cento), mas presença de blastos no sangue periférico foi observada em apenas dois pacientes. Onze pacientes demonstraram alterações evolutivas nos hemogramas seriados. Radiografias simples se mostraram alteradas em 11/14 pacientes, ultra-som em 12/18, cintilografia em 5/5, tomografia em 7/9 e ressonância em 3/3. O mielograma estava alterado em 18/22 pacientes, três deles apenas na repetição do exame. CONCLUSÃO: Queixas musculoesqueléticas são manifestações iniciais freqüentes das neoplasias, em especial da LLA, que devem ser consideradas no diagnóstico diferencial das doenças reumatológicas. Os hemogramas podem ser inicialmente normais, sendo necessário seu seguimento evolutivo. Exames de imagem e punção de medula óssea demonstraram ser fundamentais no diagnóstico.

OBJECTIVE: To describe the clinical and laboratory findings that contributed for the diagnosis of neoplasia in patients with musculoskeletal symptoms at presentation. METHODS: Retrospective analysis of medical records from patients with final diagnosis of neoplasia attended at the "Unidade de Reumatologia do Instituto da Criança - FMUSP" between January 1983 and December 2006. Data on musculoskeletal complaints, clinical examination, laboratory tests, radiological studies and diagnostic procedures were obtained. RESULTS: From 4876 patients, 25 (0.5 percent) children were studied (52 percent with acute lymphoid leukemia and 24 percent with neuroblastoma). Twenty children (80 percent) presented arthritis and/or arthralgia at onset of the disease. All patients presented systemic symptoms, such as fever (22 cases - 88 percent). The initial blood cell count was abnormal in 16 patients (64 percent), showing anemia and thrombocytopenia (12 and 5 cases, respectively). Blast cells were present in only two patients and eleven patients developed blood cell count abnormalities during follow-up. X-ray studies showed abnormalities in 11/14 patients, ultrasound in 12/18, scintigraphy in 5/5, CT in 7/9 and MRI in 3/3. Bone marrow smear was abnormal in 18/22 patients, but in three of them the abnormalities were not detected by the first test. CONCLUSION: Musculoskeletal symptoms are common at the onset of neoplasia, especially for acute lymphoid leukemia, and this possibility should be considered in the differential diagnosis of rheumatic diseases. Laboratory tests may be normal at the onset of the disease, therefore serial exams should be performed. For a correct diagnosis. radiological studies and bone marrow aspiration have proven to be essential.

Piomiosite tropical ao diagnóstico de LLA: relato de caso / Tropical pyomyositis at diagnosis of ALL: case report

Os autores apresentam o caso de um jovem de 14 anos com história de dor em membros e febre. A investigação radiológica mostrou presença de abscessos compatíveis com o diagnóstico de piomiosite...

The authors report the case of 14 ys.o boy complaining of limbs pain and fever. The disclosure of abscesses at radiology work-up fulfilled the diagnosis S.aureus was isolated from right forearm effusion. Otherside the usual blood count featured in an infectious condition like that, leucopenia...

A prevalência de perdas auditivas em crianças e adolescentes com câncer / The prevalence of hearing loss in children and adolescents with cancer

O tratamento do câncer infantil provoca diversos efeitos colaterais, como a ototoxicidade, que é capaz de lesar estruturas da orelha interna e pode levar à perda auditiva. OBJETIVO: Estimar a prevalência de perda auditiva em crianças e adolescentes com câncer, utilizando três classificações: American Speech-Language-Hearing Association (ASHA), Pediatric Oncology Group Toxicity (POGT) e Perda Auditiva Bilateral (PAB). Forma de Estudo: Transversal. MATERIAL E MÉTODO: Analisou-se 94 pacientes atendidos entre 2003 e 2004. Os indivíduos foram submetidos à inspeção visual do meato acústico externo e avaliação audiológica. Para caracterização da amostra utilizou-se a estatística descritiva e para a análise da concordância da perda auditiva nas três classificações foi utilizada a estatística Kappa. RESULTADOS: Houve prevalência de perda auditiva de 42,5 por cento pela ASHA, 40,4 por cento pela POGT e 12,8 por cento pela PAB. A concordância para POGT e PAB, e para PAB e ASHA foi fraca (respectivamente, k=0,36 e k=0,33). A concordância entre ASHA e POGT foi quase perfeita (k=0,96). CONCLUSÕES: A perda de audição é um efeito colateral importante nos pacientes com câncer. A monitorização auditiva é fundamental, pois possibilita detecção precoce e revisão do tratamento. Recomenda-se adotar uma classificação que contemple perdas auditivas leves, como proposta pela ASHA.

The treatment of cancer in children has several side effects, including ototoxicity. Inner ear structures may be affected and hearing loss may ensue. AIM: To estimate the prevalence of hearing loss in patients with cancer using the American Speech-Language-Hearing Association (ASHA), the Pediatric Oncology Group Toxicity (POGT), and the Bilateral Hearing Loss (PAB) criteria. Study design: a prospective study. MATERIAL AND METHODS: 94 patients admitted between 2003 and 2004 were analyzed. Visual inspection of the external auditory meatus and an audiologic evaluation were done. Descriptive statistics was used to characterize the sample, and Kappa statistics was used to investigate concordance of hearing loss in the three types of classification. RESULTS: The prevalence of hearing loss was 42.5 percent using ASHA, 40.4 percent using POGT, and 12.8 percent using PAB. The concordance of hearing loss was weak for POGT and PAB (k=0.36) and for PAB and ASHA (k=0.33). The concordance between ASHA and POGT was almost perfect (k=0.96). CONCLUSIONS: Hearing loss is an important side effect of the treatment of cancer in children. Periodic audiology monitoring is recommended to detect early hearing loss and to revise the treatment if necessary. Adoption of a classification system that detects mild hearing loss (ASHA) is recommended.

Perspectivas para a leucemia mielóide aguda na infância após a observação de um grupo de pacientes tratados convencionalmente / Pediatric acute myeloid leukemia outcomes after conventional treatment

O tratamento atual das leucemias mielóides agudas (LMA) obedece principalmente aos seguintes critérios: precisa caracterização clínica e biológica, adequada e agressiva quimioterapia, intensa terapia de suporte e possível indicação precoce de transplante de medula óssea. Neste artigo, envolvendo crianças tratadas entre 1986 e 1996, são apresentados os resultados passíveis de obtenção com tratamento quimioterápico convencional e discutidas as bases de implantação de um programa terapêutico moderno e de maior eficácia. Quarenta e três pacientes com LMA "de novo", idades entre 6 meses e 14,5 anos, foram tratados conforme programa terapêutico que incluiu indução e consolidação com quatro drogas: DAUNO, ARA-C, VCR e DEXA e 60 semanas de manutenção com os seguintes ciclos administrados seqüencialmente: ARA-C & ASP, CICLO & ETO, DAUNO (6-TG) & ARA-C & VCR & DEXA e 6-TG & ARA-C & VCR & DEXA. Profilaxia de envolvimento de SNC com ARA-C por via intratecal. Nenhuma criança foi submetida a transplante de medula óssea em primeira instância. 38/43 (84 por cento) crianças alcançaram remissão completa e a sobrevida livre de eventos estimada para três anos foi de 24 ± 7 por cento. A importância da discriminação das LMA em grupos de risco, que levem em consideração particularmente os achados citogenéticos, além de novas modalidades de abordagem terapêutica, é discutida, destacando-se a importância atual dos transplantes alogênicos de medula óssea.

Modern treatment of acute myeloid leukemias follows well defined guidelines: precise clinical and biological characterization, adequate aggressive chemotherapy, intensive supportive care and eventual early indication of bone marrow transplantation. In this report we present the best results obtained with conventional chemotherapy delivered to a group of pediatric patients between 1986 and 1996 and discuss the strategies for contemporary and more efficient regimens. Forty-three patients with "de novo" acute myeloid leukemias with ages between 6 months and 14.5 years, were treated with a 4-drug induction and consolidation program: AUNO, ARA-C, VCR and DEXA, followed by 60 weeks of maintenance with sequential cycles of ARAC & ASP, CICLO & ETO, DAUNO (6-TG) & ARA-C & VCR & DEXA and 6-TG & ARA-C & VCR & DEXA. CNS prophylaxis with intrathecal ARA-C was undertaken. No child underwent bone marrow transplantation in first remission. A total of 38/43 (84 percent) children achieved complete remission and event-free survival at 3 years was estimated at 24 ± 7 percent. The importance of discriminating AML in risk groups with particular attention paid to cytogenetic criteria and new modalities of treatment are discussed. The current relevance of bone marrow transplantations is emphasized.

O diagnóstico tardio de rabdomiossarcoma / Late diagnosis of pediatric rhabdomyosarcoma

Objetivo: avaliar o período de tempo decorrido desde o início da sintomatologia até o diagnóstico de rabdomiossarcoma em crianças e a possível implicação na evolução e prognóstico...

Objective: to evaluate the period lenght since the beginning of symptoms until the rhabidomyosarcoma in a reference study was conducted with 163 patients aged under 16-years-old admited for treatment...

Alterações do desenvolvimento dentofacial em pacientes da oncopediatria / Dentofacial development alterations in oncopediatric patients

No tratamento do câncer infantil, uma das principais armas utilizadas é a quimioterapia, quer isoladamente ou associada à cirurgia e à radioterapia. Como conseqüência, algumas complicações orais podem ser observadas, dentre elas uma série de seqüelas em função dos efeitos tardios da químio e da radioterapia. A odontogênese e o crescimento facial, comumente, são afetados durante o tratamento oncológico e, de acordo com a idade do paciente, surgirão malformações dentárias e hipodesenvolvimento dos ossos maxilares. Os autores relatam alterações dentofaciais em crianças que foram submetidas a químio e/ou radioterapia na época do desenvolvimento e crescimento dentocraniofacial, através de revisão da literatura dos últimos 10 anos e da apresentação de um caso clínico

Growth and puberty after treatment for acute lymphoblastic leukemia

Nos últimos 20 anos, após o tratamento de pacientes portadores de leucemia linfoblástica aguda, com quimioterapia e radioterapia, houve melhora na taxa de sobrevivência e cura em torno de 70 por cento. Crianças portadoras da doença foram envolvidas em protocolos de tratamento internacionais que visavam melhorar a sobrevida e minimizar os graves e irreversíveis efeitos tardios. A nossa unidade utiliza o protocolo internacional GBTLI LLA-85 e 90, com as drogas metrotexate, citosina, arabinoside, dexametasona e radioterapia .Entretanto, estes tratamentos podem causar insuficiências gonadais e prejuízo no crescimento. PACIENTES E MÉTODO: Os autores analisaram 20 crianças fora de terapia a fim de determinar o papel das várias doses de radioterapia sobre alterações endocrinológicas. Foram divididos em três grupos baseados na profilaxia do sistema nervoso central: o grupo A foi submetido à quimioterapia, o grupo B à quimioterapia mias radioterapia (18Gy) e o grupo C à quimioterapia mais radioterapia (24 Gy). Foram avaliadas as concentrações séricas de LH, FSH, GH e testosterona. Os estudos de imagem incluiram idade óssea, ultrassonografia pélvica, escrotal e ressonância nuclear magnética do crânio. RESULTADOS: Houve diferenças significativas nas respostas do hormônio de crescimento e prejuízo na estatura final (Bayley-Pinneau) entre os dois grupos irradiados e o grupo que não foi irradiado, mas não houve diferenças quando se compararam as doses de radiação utilizadas (18 ou 24 Gy). A previsão da altura final (Bayley-Pinneau) foi menor (p= 0,0071) nos dois grupos tratados com radioterapia. Duas meninas apresentaram puberdade precoce e um menino teve atraso puberal associado a calcificação do epidídimo. CONCLUSÃO: A radioterapia é responsável por efeitos colaterais especialmente quanto ao crescimento e puberdade.