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1.
Article in Chinese | WPRIM | ID: wpr-928698

ABSTRACT

OBJECTIVE@#To evaluate the clinical effect of haploid allogeneic hematopoietic stem cell transplantation(haplo-HSCT) in the treatment of severe aplastic anemia (SAA), and to explore the efficacy different between post-transplant cyclophosphamide (PT/Cy) and standard-dose ATG.@*METHODS@#The clinical data of 38 patients with SAA in our hospital from January 2012 to December 2019 were collected and retrospectively analyzed. The efficacy was evaluated. The patients with haplo-HSCT were divided into low-dose ATG combined with PT/Cy group and standard-dose ATG group, and the blood cell hematopoietic reconstruction time, GVHD incidence, mortality and survival time of the patients in the two groups was compared.@*RESULTS@#Among the 32 patients, hematopoietic reconstitution were detected in 9375%(30/32) recipients. The median time of neutrophil and platelet engraftment was 15(10-22) days and 13(7-30) days, respectively. The incidence of GVHD was 21.89%, the incidence of infection was 93.75%, and the 2-year overall survival rate was 84.38%. The hematopoietic reconstitution time, incidence of GVHD, mortality rate and survival time were no statistical differences between the patients in the two groups(all P>0.05).@*CONCLUSION@#Haplo-HSCT is an effective method for the treatment of SAA,low-dose ATG combined with PT/Cy can lighten the economic burden on patients, it would be a feasible treatment plan for SAA with light side effect.


Subject(s)
Anemia, Aplastic/therapy , Cyclophosphamide , Graft vs Host Disease , Haploidy , Hematopoietic Stem Cell Transplantation , Humans , Retrospective Studies , Transplantation Conditioning
2.
Frontiers of Medicine ; (4): 718-727, 2021.
Article in English | WPRIM | ID: wpr-922504

ABSTRACT

Severe aplastic anemia II (SAA-II) progresses from non-severe aplastic anemia (NSAA). The unavailability of efficacious treatment has prompted the need for haploidentical bone marrow transplantation (haplo-BMT) in patients lacking a human leukocyte antigen (HLA)-matched donor. This study aimed to investigate the efficacy of haplo-BMT for patients with SAA-II. Twenty-two patients were included and followed up, and FLU/BU/CY/ATG was used as conditioning regimen. Among these patients, 21 were successfully engrafted, 19 of whom survived after haplo-BMT. Four patients experienced grade II-IV aGvHD, including two with grade III-IV aGvHD. Six patients experienced chronic GvHD, among whom four were mild and two were moderate. Twelve patients experienced infections during BMT. One was diagnosed with post-transplant lymphoproliferative disorder and one with probable EBV disease, and both recovered after rituximab infusion. Haplo-BMT achieved 3-year overall survival and disease-free survival rate of 86.4% ± 0.73% after a median follow-up of 42 months, indicating its effectiveness as a salvage therapy. These promising outcomes may support haplo-BMT as an alternative treatment strategy for patients with SAA-II lacking HLA-matched donors.


Subject(s)
Anemia, Aplastic/therapy , Bone Marrow Transplantation , Graft vs Host Disease , HLA Antigens , Hematopoietic Stem Cell Transplantation , Humans , Transplantation Conditioning
3.
Journal of Experimental Hematology ; (6): 1601-1605, 2021.
Article in Chinese | WPRIM | ID: wpr-922302

ABSTRACT

OBJECTIVE@#To analyze the clinical efficacy and safety of allogeneic hematopoietic stem cell transplantation (allo-HSCT) for paroxysmal nocturnal hemoglobinuria (PNH), and preliminarily explore the role of an improved post-transplantation cyclophosphamide (PTCy) based conditioning regimen in PNH patients receiving transplantation.@*METHODS@#Clinical related data of PNH sufferers receiving allo-HSCT in Department of Hematology, Tongji Hospital, Tongji Medical College, Huazhong University of Science and Technology were collected, and hematopoietic reconstitution, chimerism, PNH cloning, graft-versus-host disease (GVHD), infection, and survival were analyzed.@*RESULTS@#Totally five PNH patients receiving allo-HSCT were enrolled, including 1 case with classic PNH, 3 cases with aplastic anemia-PNH syndrome, 1 case with myelodysplastic syndrome, three of them (case 1-3) received the improved PTCy based conditioning regimen before HSCT. All sufferers engrafted successfully within 28 days, the median time of neutrophil and platelet engraftment was 11 days and 12 days, respectively, no patient occurred acute or chronic GVHD, after a median follow-up of 16 months, all recipients survived and completely eliminated PNH cloning.@*CONCLUSION@#Allo-HSCT can completely clear PNH cloning and restore hematopoietic function with controllable complications, and the improved PTCy based conditioning regimen is proved to be effective in PNH transplantation.


Subject(s)
Anemia, Aplastic/therapy , Graft vs Host Disease , Hematopoietic Stem Cell Transplantation , Hemoglobinuria, Paroxysmal/therapy , Humans , Transplantation Conditioning
4.
Article in English | WPRIM | ID: wpr-888493

ABSTRACT

Haploidentical hematopoietic stem cell transplantation is a recommended alternative therapy for children with severe aplastic anemia who lack a human leukocyte antigen (HLA)-identical sibling donor and do not respond well to immunosuppressive therapy; however, due to non-identical HLA, the patients may have donor-specific anti-HLA antibody, which may lead to a relatively high incidence rate of poor graft function. Compared with HLA-identical transplantation, conditioning regimen for haploidentical transplantation still needs to be explored. This article reviews the detection and treatment of donor-specific anti-HLA antibody, the selection of conditioning regimen, and the mechanism and treatment of poor graft function in haploidentical hematopoietic stem cell transplantation.


Subject(s)
Anemia, Aplastic/therapy , Child , Graft vs Host Disease , Hematopoietic Stem Cell Transplantation , Humans , Transplantation Conditioning , Transplantation, Homologous
5.
Rev. cuba. hematol. inmunol. hemoter ; 36(3): e1277, jul.-set. 2020. tab, graf
Article in Spanish | LILACS, CUMED | ID: biblio-1156444

ABSTRACT

Introducción: La aplasia medular adquirida grave es una enfermedad hematológica infrecuente caracterizada por una disminución o ausencia de precursores hematopoyéticos en la médula ósea, lo cual se expresa con distintos grados de citopenias. Varios factores, infecciosos o no, pueden incidir en su origen. Su manejo es complejo y puede incluir tratamiento inmunosupresor y trasplante de progenitores hematopoyéticos alogénico. Objetivo: Demostrar la utilidad de la realización del trasplante de progenitores hematopoyéticos alogénico haploidéntico en pacientes con aplasia medular grave. Caso clínico: Paciente masculino de 21 años de edad, con antecedentes de salud, que en octubre del 2018 debutó con íctero, pancitopenia, lesiones purpúrico hemorrágicas en piel y mucosas, en el curso de una hepatitis aguda seronegativa. La biopsia de médula ósea mostró aplasia medular severa. Se inició tratamiento inmunosupresor con globulina antitimocίtica, ciclosporina A y metilprednisolona. Al cabo de los 6 meses mantenía trombocitopenia severa con necesidades transfusionales y en octubre de 2019 se decide realizar trasplante de progenitores hematopoyéticos alogénico con donante haploidéntico y empleando como tratamiento acondicionante globulina antitimocίtica, fludarabina, ciclofosfamida y bajas dosis de irradiación corporal total. En evaluación clίnica de julio de 2020 (dίa + 280 del trasplante) el paciente estaba asintomático y con parámetros hematológicos normales. Conclusiones: Se demostró que el trasplante de progenitores hematopoyéticos alogénico haploidéntico es un proceder realizable y útil en pacientes con aplasia medular grave, lo cual corrobora el beneficio clínico que puede aportar su ejecución en pacientes con esta enfermedad(AU)


Introduction: Acquired severe marrow aplasia is a rare hematological disease characterized by decrease or absence of hematopoietic precursors in bone marrow, which is expressed with different degrees of cytopenias. Several factors, infectious or not, can influence its origin. Its management is complex and may include immunosuppressive treatment and allogeneic hematopoietic stem-cell transplantation. Objective: To demonstrate the usefulness of performing haploidentical allogeneic hematopoietic stem-cell transplantation in patients with severe medullary aplasia. Clinical case: A 21-year-old male patient, with medical history, who first presented, in October 2018, with icterus, pancytopenia, as well as purpuric hemorrhagic lesions on the skin and mucosa, in the course of acute seronegative hepatitis. The bone marrow biopsy showed severe marrow aplasia. Immunosuppressive treatment was started with antithymocytic globulin, cyclosporine A, and methylprednisolone. After six months, he maintained severe thrombocytopenia under transfusion requirements and, in October 2019, the decision was to perform allogeneic hematopoietic stem-cell transplantation with a haploidentical donor and using antithymocyte globulin, fludarabine, cyclophosphamide, and low doses of total body irradiation as conditioning treatment. In the clinical assessment carried out in July 2020 (day +280 after transplantation), the patient was asymptomatic and with normal hematological parameters. Conclusions: Transplantation of haploidentic allogeneic hematopoietic progenitors was shown to be a feasible and useful procedure in patients with severe marrow aplasia, which corroborates the clinical benefit that its execution can bring in patients with this disease(AU)


Subject(s)
Humans , Male , Young Adult , Tissue Donors/ethics , Methylprednisolone/therapeutic use , Whole-Body Irradiation/methods , Microscopy, Electron, Scanning Transmission/methods , Hematologic Diseases , Hematopoietic Stem Cell Transplantation/methods , Cuba , Transplantation, Haploidentical/methods , Anemia, Aplastic/therapy , Antilymphocyte Serum
7.
Rev. méd. Chile ; 146(3): 399-402, mar. 2018. graf
Article in Spanish | LILACS | ID: biblio-961407

ABSTRACT

Aplastic anemia (AA) or acquired aplastic anemia is an uncommon and potentially fatal disease. It is defined as reduction of at least two peripheral blood series, associated with persistent bone marrow hypocellularity. It's association with pregnancy is even more uncommon, and it may result in high mortality for the mother and the child. We report an adolescent female with aplastic anemia, which worsened during pregnancy. Her hemoglobin was 5.2 g/dl, her leukocytes were 1,833/ul and her platelets were 19,000/ul. She was initially treated with cyclosporine and horse antithymocyte globulin without hematologic improvement. At 36 weeks of pregnancy methylprednisolone and platelet transfusions were used and a cesarean section was performed. Six month later she continues to require red blood cell and platelet transfusions.


Subject(s)
Humans , Male , Female , Pregnancy , Infant, Newborn , Adolescent , Pregnancy Complications, Hematologic/therapy , Anemia, Aplastic/therapy , Severity of Illness Index , Pregnancy Outcome
8.
Ann. hepatol ; 16(2): 312-317, Mar.-Apr. 2017. tab, graf
Article in English | LILACS | ID: biblio-887238

ABSTRACT

ABSTRACT The addition of the new protease inhibitors (PIs) to peg-interferon (IFN) and ribavirin (RBV), approved for chronic hepatitis C, has clearly improved sustained virological response (SVR) rates although several adverse events have been reported with this regimens, including mild hematological toxicity. Moreover, severe pancytopenia and aplastic anemia during triple therapy with telaprevir has recently been described in seven patients. We report here two cases of severe agranulocytosis/aplastic anemia using boceprevir or simeprevir in interferon-based combination and 2 additional cases of severe myelosupression in IFN-free therapy with sofosbuvir and simeprevir plus RBV. Our observations suggest that PIs could have a sort of class-effect in developing severe hematologic toxicity or, at least, an additive interaction with other potentially myelotoxic agents such as IFN or RBV that are used in the classical regimens against HCV. Unfortunately, the mechanisms behind this phenomenon are currently unknown. In conclusion, given the lifethreatening character of these complications, close monitoring is mandatory in patients under PIs based therapy to promptly detect serious hematological toxicities and to carefully evaluate treatment discontinuation. Prospective studies assessing the usefulness of RBV in the era of new IFN-free combinations are needed.


Subject(s)
Humans , Protease Inhibitors/adverse effects , Bone Marrow Diseases/chemically induced , Bone Marrow Diseases/therapy , Proline/analogs & derivatives , Hepatitis C/drug therapy , Simeprevir/adverse effects , Anemia, Aplastic/chemically induced , Anemia, Aplastic/therapy , Severity of Illness Index , Bone Marrow Examination , Proline/adverse effects , Predictive Value of Tests , Risk Factors , Treatment Outcome , Hepatitis C/diagnosis , Fatal Outcome , Drug Therapy, Combination
9.
Med. Afr. noire (En ligne) ; 63(12): 607-612, 2016. tab
Article in French | AIM | ID: biblio-1266160

ABSTRACT

Introduction : L'Aplasie Médullaire (AM) est une insuffisance quantitative de l'hématopoïèse responsable d'une pancytopénie avec une moelle osseuse pauvre. Elle peut être constitutionnelle ou acquise (toxique, infectieuse, idiopathique). Si le traitement est bien codifié dans les pays riches avec l'amélioration du pronostic à long terme depuis plusieurs années, cette maladie demeure hautement mortelle dans les pays en voie de développement.Objectif : Etudier les caractéristiques cliniques, thérapeutiques et évolutives de l'AM chez l'enfant au CHU de Brazzaville.Matériels et méthodes : Il s'est agi d'une cohorte historique, réalisée sur la base des dossiers d'enfants hospitalisés pour une AM sur une période de 15 ans (2001- 2015). La classification de CAMITTA avait été utilisée.Résultats : 22 dossiers avaient été retenus. La moyenne d'âge était de 12,16 ans. Le sex-ratio garçon/fille était de 0,8. Les AM étaient toutes idiopathiques. Le syndrome anémique était le principal motif d'admission. Le taux moyen d'hémoglobine était de 4,3 g/dL, celui des polynucléaires neutrophiles de 0,340 Giga/l et celui des plaquettes de 13,2 Giga/l. Seize patients sur 22 (72,72%) souffraient de la forme sévère. Dans 90,90% des cas le traitement était symptomatique (concentrés érythrocytaires et plaquettaires, antibiothérapie) parfois associé à la corticothérapie ; 2 patients/22 (9,10%) ont reçu de la ciclosporine. Le taux de mortalité était de 94,73% après un suivi moyen de 9 semaines.Conclusion : L'AM de l'enfant au CHU de Brazzaville est essentiellement idiopathique et de pronostic redoutable


Subject(s)
Academic Medical Centers , Anemia, Aplastic/diagnosis , Anemia, Aplastic/therapy , Child , Congo , Disease Progression , Hematopoiesis
10.
Bogotá; IETS; dic. 2014. ilus.
Monography in Spanish | LILACS, BRISA | ID: biblio-847114

ABSTRACT

Introducción: La anemia aplásica es una enfermedad poco común que afecta a 2 de cada 1´000.000 de personas anualmente con igual distribución en hombres y mujeres. Se caracteriza por la sustitución del tejido hematopoyético de la medula ósea por grasa causando una pancitopenia periférica, con diferentes niveles de gravedad, originando un síndrome anémico, hemorragias e infecciones graves que pueden llevar a desenlaces fatales de no recibir tratamiento oportuno. La primera opción de tratamiento en pacientes no compatibles para trasplante de progenitores hematopoyeticos es la terapia inmunosupresora. La evidencia actual sugiere la efectividad del tratamiento con inmunoglobulina antitimocítica en los esquemas de tratamiento. Objetivo: Examinar los beneficios y riesgos del uso de la inmunoglobulina antitimocítica en pacientes con anemia aplásica no hereditaria severa a muy severa. Metodología: Se realizó una búsqueda sistemática de estudios clinicos incluyendo utilizando las bases de datos MEDLINE (In-Process & Other Non-Indexed Citations y Daily Update) EMBASE, Cochrane Database of Systematic Reviews, Database of Abstracts of Reviews of Effects ­ DARE, LILACS y la revisión de publicaciones obtenidas por otros medios comparando el uso de inmunoglobulina antitimocitica en pacientes con anemia aplasica no hereditaria, no candidatos a trasplante de prognitores hematopoyéticos con otros tratamientos inmunosupresores, considerando los resultados en cuanto a tasa de respuesta, sobrevida, recaída y transformación clonal. Resultados: Se obtuvieron 103 publicaciones, 24 fueron tamizadas para valoración de los criterios de inclusión. Tres cumplieron con estos y se evaluaron con las herramientas de la colaboración Cochrane. Todas las referencias reportaron alto riesgo de sesgo. Un ensayo clinico controlado no reportó diferencias significativas para ninguno de los desenlaces evaluados comparando CTX más CsA frente a ATGr más CsA. Uno de los estudios de cohorte reportó una efectividad de 77.8% y sobrevida a 5 años fue de 74.1%. La segunda cohorte reportó respuesta completa (17.7%), respuesta parcial (37.9%) y tasa de respuesta global (55.6%). La recaida fue de 3.2%, la transformacion clonal 0.8% y la sobrevida a 5 años fue 74.7%. Conclusiones: La evidencia sobre la efectividad y seguridad de la inmunoglobulina antitimocitica es limitada y de baja calidad. Con los hallazgos obtenidos en esta revision no es posible determinar la superioridad de esta tecnología frente a otras opciones de tratamiento disponibles.(AU)


Subject(s)
Humans , Anemia, Aplastic/therapy , Antifungal Agents/administration & dosage , Cyclophosphamide/administration & dosage , Cyclosporine/administration & dosage , Danazol/administration & dosage , Immunoglobulins/administration & dosage , Biomedical Technology , Colombia , Medication Adherence , Reproducibility of Results , Treatment Outcome
11.
J. pediatr. (Rio J.) ; 90(5): 523-527, Sep-Oct/2014. tab, graf
Article in English | LILACS | ID: lil-723162

ABSTRACT

Objective: To evaluate the outcome of children with severe acquired aplastic anemia treated with rabbit antithymocyte globulin and cyclosporine as first-line treatment at this institution. Methods: Retrospective analysis of 26 pediatric patients with aplastic anemia, treated between 1996 and 2011 with rabbit antithymocyte globulin plus cyclosporine. Results: The overall response rate at six months was 34.6% (9/26), and the cumulative incidence of relapse was 26.5% (95% confidence interval [CI]: 1.4%-66%) at 5 years. The cumulative incidence of clonal evolution after immunosuppressive therapy was 8.3% (95% CI: 0.001%-53.7%) at five years with both clonal evolutions in non-responders who acquired monosomy 7 karyotype. The overall survival at five years was 73.6% (95% CI: 49.2%-87.5%). Conclusions: The present results confirm the poor response rate with rabbit antithymocyte globulin as first therapy in pediatrics patients, similar to what has been reported for patients of all ages. This confirmation is problematic in Brazil, given the lack of horse antithymocyte globulin in many markets outside the United States. .


Objetivo: Avaliar o resultado de crianças com anemia aplástica grave adquirida tratadas com globulina antitimocítica de coelho e ciclosporina como tratamento inicial em nosso instituto. Métodos: Análise retrospectiva de 26 pacientes pediátricos com anemia aplástica tratados entre 1996 e 2011 com globulina antitimocítica de coelho e ciclosporina. Resultados: A taxa de resposta geral em seis meses foi de 34,6% (9/26), e a incidência acumulada de recorrência foi de 26,5% (intervalo de confiança [IC] de 95%,1,4%-66%) em cinco anos. A incidência acumulada de evolução clonal após a terapia imunossupressora foi de 8,3% (IC 95%, 0,001%-53,7%) em cinco anos, com ambas as evoluções clonais em pacientes sem resposta que adquiriram o cariótipo com monossomia 7. A sobrevida geral em cinco anos foi de 73,6% (IC 95%, 49,2%-87,5%). Conclusões: Nossos resultados confirmam a baixa taxa de resposta com globulina antitimocítica de coelho como terapia inicial em pacientes pediátricos, da mesma forma como relatado para pacientes de todas as idades. Essa confirmação é problemática em nosso país devido à falta de globulina antitimocítica de cavalo em muitos mercados fora dos Estados Unidos, incluindo o Brasil. .


Subject(s)
Adolescent , Animals , Child , Child, Preschool , Female , Humans , Infant , Male , Rabbits , Anemia, Aplastic/mortality , Antilymphocyte Serum/therapeutic use , Cyclosporine/therapeutic use , Immunosuppressive Agents/therapeutic use , Anemia, Aplastic/therapy , Brazil/epidemiology , Clonal Evolution , Follow-Up Studies , Recurrence , Retrospective Studies , Survival Rate , Treatment Outcome
12.
Article in English | WPRIM | ID: wpr-221307

ABSTRACT

Many Korean patients with transfusion-induced iron overload experience serious clinical sequelae, including organ damage, and require lifelong chelation therapy. However, due to a lack of compliance and/or unavailability of an appropriate chelator, most patients have not been treated effectively. Deferasirox (DFX), a once-daily oral iron chelator for both adult and pediatric patients with transfusion-induced iron overload, is now available in Korea. The effectiveness of deferasirox in reducing or maintaining body iron has been demonstrated in many studies of patients with a variety of transfusion-induced anemias such as myelodysplastic syndromes, aplastic anemia, and other chronic anemias. The recommended initial daily dose of DFX is 20 mg/kg body weight, taken on an empty stomach at least 30 min before food and serum ferritin levels should be maintained below 1000 ng/mL. To optimize the management of transfusion-induced iron overload, the Korean Society of Hematology Aplastic Anemia Working Party (KSHAAWP) reviewed the general consensus on iron overload and the Korean data on the clinical benefits of iron chelation therapy, and developed a Korean guideline for the treatment of iron overload.


Subject(s)
Anemia, Aplastic/therapy , Benzoates/therapeutic use , Blood Transfusion/adverse effects , Chelation Therapy/methods , Humans , Iron Chelating Agents/therapeutic use , Iron Overload/therapy , Myelodysplastic Syndromes/therapy , Pyridones/therapeutic use , Republic of Korea , Triazoles/therapeutic use
13.
Article in English | IMSEAR | ID: sea-140331

ABSTRACT

Background & objectives: Aplastic anaemia is a life threatening rare bone marrow failure disorder. The underlying haematopoietic cellular deficit leads to haemorrhage, infection and severe anaemia. The treatment of choice for this haematological condition is allogeneic bone marrow transplantation from fully matched HLA sibling. Though this procedure is curative in the majority of young patients with aplastic anaemia, extending this benefit to older patients or those lacking a family donor remains a major challenge. Herein, the safety and efficacy of infusing autologous retrodifferentiated haematopoietic stem cells (RHSC) into four patients with aplastic anaemia without the use of any pre- or post-conditioning regimen including immunosuppression is described. Methods: Un-mobilized, mononuclear cells were harvested from four patients with acquired aplastic anaemia by aphaeresis. Mononuclear cells of patients were cultured with purified monoclonal antibody against the monomorphic regions of the beta chain of MHC class II antigens (Clone CR3/43) for 3 h, to obtain autologous RHSC. Autologous RHSC were washed and infused into the four patients without the use of any pre- or post-conditioning regimen. Thereafter, the efficacy (engraftment) of autologous RHSC was assessed in these patients. Results: Following single infusion of the autologous RHSC, two of the four patients with aplastic anaemia become transfusion independent for more than seven years. Karyotyping and G-banding analysis prior and post-procedure in all patients remained the same. Interpretation & conclusions: The findings of this pilot study demonstrated the functional utility of reprogrammed fully differentiated adult cells into pluripotent stem cells with extensive repopulation potentials in a human setting and without any pre- or post-conditioning regimen, including immunosuppression. This autologous approach of stem cell creation may broaden the curative potentials of stem cell therapy to a wider population of patients with aplastic anaemia, including many patients suffering from other haematological and non-haematological disorders.


Subject(s)
Adult , Anemia, Aplastic/therapy , Induced Pluripotent Stem Cells , Adult Stem Cells , Stem Cell Transplantation , Leukocytes
14.
JCPSP-Journal of the College of Physicians and Surgeons Pakistan. 2011; 21 (4): 219-222
in English | IMEMR | ID: emr-110164

ABSTRACT

To determine the clinicohaematological features, treatment and outcome of children diagnosed with aplastic anemia at a single institution. Observational study. The Aga Khan University Hospital, Karachi, from January 1999 till December 2008. Medical records of children aged less than 15 years of age diagnosed with aplastic anemia were reviewed. Clinicohaematological features, treatment and its response to therapy and outcome were recorded. Results were described in percentages. Ninety patients were diagnosed to have aplastic anemia [AA]; 65 were male during the study period. Age ranged from 1 to 15 years. Fever in 65 patients [72.2%], pallor in 53 [58.8%], skin bleeding in 49 [54.4%] and epistaxis in 31[34.4%] were the most common and frequent presenting features. Congenital [Fanconi's] anemia was found in 15 [16.6%] and acquired idiopathic in 75 [83.4%] of patients. Very severe aplastic anemia [VSAA] was seen in 29 [32.2%], 26 [28.9%] had severe AA and 17 [18.9%] had moderate AA. Eight patients [8.9%] underwent haematopoietic stem cell transplantation [HSCT], 12 [13.3%] received immunosuppressive therapy [IST] and 70 patients [77.7%] received other and supportive therapy. Five [62.5%] patients showed complete response to HSCT and 3 [37.5%] failed to engraft. IST showed complete response in 3 [25%], partial response in 5 [41.6%] and no response in 4 [33.3%]. Twenty two patients [24.4%] expired either due to infection in 16 [72.7%, fungal in 6, bacterial in 10] and intracranial haemorrhage in 6 [27.3%] cases. Majority of cases with AA were acquired and idiopathic in etiology. VSAA and SAA were frequent. Response to HSCT and IST was sub-optimal


Subject(s)
Humans , Male , Female , Hematopoietic Stem Cell Transplantation , Anemia, Aplastic/therapy , Retrospective Studies , Treatment Outcome
15.
Braz. j. med. biol. res ; 42(3): 244-250, Mar. 2009. graf, tab
Article in English | LILACS | ID: lil-507344

ABSTRACT

Allogeneic hematopoietic stem cell transplantation (AHSCT) is the treatment of choice for young patients with severe aplastic anemia (SAA). The association of antithymocyte globulin (ATG) and cyclophosphamide (CY) is the most frequently used conditioning regimen for this disease. We performed this retrospective study in order to compare the outcomes of HLA-matched sibling donor AHSCT in 41 patients with SAA receiving cyclophosphamide plus ATG (ATG-CY, N = 17) or cyclophosphamide plus busulfan (BU-CY, N = 24). The substitution of BU for ATG was motivated by the high cost of ATG. There were no differences in the clinical features between the two groups, including age, gender, cytomegalovirus status, ABO match, interval between diagnosis and transplant, and number of total nucleated cells infused. No differences were observed in the time to neutrophil and platelet engraftment, or in the risk of veno-occlusive disease and hemorrhage. However, there was a higher risk of mucositis in the BU-CY group (71 vs 24 percent, P = 0.004). There were no differences in the incidence of neutrophil and platelet engraftment, acute and chronic graft-versus-host disease, and transplant-related mortality. There was a higher incidence of late rejection in the ATG-CY group (41 vs 4 percent, P = 0.009). Although the ATG-CY group had a longer follow-up (101 months) than the BU-CY group (67 months, P = 0.04), overall survival was similar between the groups (69 vs 58 percent, respectively, P = 0.32). We conclude that the association BU-CY is a feasible option to the conventional ATG-CY regimen in this population.


Subject(s)
Adolescent , Adult , Female , Humans , Male , Middle Aged , Young Adult , Anemia, Aplastic/therapy , Hematopoietic Stem Cell Transplantation , Immunosuppressive Agents/administration & dosage , Transplantation Conditioning/methods , Antilymphocyte Serum/administration & dosage , Busulfan/administration & dosage , Cyclophosphamide/administration & dosage , Drug Therapy, Combination , Graft Rejection/prevention & control , HLA Antigens/blood , Retrospective Studies , Severity of Illness Index , Treatment Outcome , Young Adult
16.
Rev. méd. Chile ; 135(11): 1421-1428, nov. 2007. ilus, tab, graf
Article in Spanish | LILACS | ID: lil-472841

ABSTRACT

Background: Severe acquired aplastic anemia (SAA) is an uncommon disease of childhood. Patients with SAA receive supportive care with transfusions and timely treatment of opportunistic infections, along with specific therapies, which may be allogenic stem cell transplantation (SCT) from a matched sibling or immunosupressive therapy (IT). Aim: To report the experience in the management of SAA. Patients and methods: Twenty five children with acquired SAA were treated from July 1992 to September 2005. Patients with full matched sibling donors received allogenic SCT after conditioning with a cyclophosphamide containing regimen. The other patients received immune suppression with cyclosporine plus methylprednisolone (n= 18) plus ATG (n=17). All received supportive care until recovery of hematopoietic function. Those who had severe opportunistic infections at diagnosis or did not respond to two cycles of ATG were evaluated for unrelated donor SCT. Results: Seven patients received sibling donor SCT and 18 IT, which was repeated in six. Three patients received mismatched related (1) or unrelated (2) SCT. Nineteen patients survived with a median follow up time of 4 years, 14 with full hematologic recovery. Six patients died: four due to infections after IT or SCT, one due to intracranial hemorrhage and one with secondary myelodysplasia 12 years after IT. Conclusions: Most children with SAA can be treated successfully with sibling donor SCT or IT. Patients without a histocompatible sibling who fail to respond to IS have a worse prognosis.


Subject(s)
Adolescent , Child , Child, Preschool , Female , Humans , Male , Anemia, Aplastic/therapy , Hematopoietic Stem Cell Transplantation , Immunosuppressive Agents/therapeutic use , Anemia, Aplastic/mortality , Combined Modality Therapy , Cyclophosphamide/therapeutic use , Cyclosporine/therapeutic use , Follow-Up Studies , Immunosuppressive Agents/adverse effects , Methylprednisolone/therapeutic use , Prognosis , Risk Factors , Severity of Illness Index , Transplantation, Homologous , Treatment Outcome
17.
Rev. méd. hered ; 18(1): 45-48, ene.-mar. 2007. ilus
Article in Spanish | LILACS, LIPECS | ID: lil-479950

ABSTRACT

Se presenta el caso de una paciente mujer de 31 años, con diagnóstico de anemia aplásica, quién refería disminución de agudeza visual central en el ojo derecho. Al examen del segmento anterior no había signos de sangrado. A la fundoscopía se encontraron hemorragias retinales en mancha en distinto cuadrante en cada ojo; hemorragia macular que comprometía la fovea en el ojo derecho y hemorragia preretinal con nivel en el cuadrante temporal inferior en el ojo izquierdo. Su evolución fue desfavorable, a la semana, presentó gran compromiso del sensorio, malestar general y al examen oftalmológico se encontraron hematomas subconjuntivales en ambos ojos como signo evolutivo de la severa plaquetopenia.(Rev Med Hered 2007;18:45-48).


Subject(s)
Humans , Female , Adult , Anemia, Aplastic/diagnosis , Anemia, Aplastic/etiology , Anemia, Aplastic/pathology , Anemia, Aplastic/therapy , Hemorrhage/diagnosis , Hemorrhage/therapy
19.
Diagnóstico (Perú) ; 44(2): 91-96, abr.-jun. 2005.
Article in Spanish | LILACS, LIPECS | ID: lil-423612
20.
JCPSP-Journal of the College of Physicians and Surgeons Pakistan. 2005; 15 (2): 117-118
in English | IMEMR | ID: emr-71498

ABSTRACT

This case report describes a patient with severe aplastic anaemia, who developed Guillain Barre Syndrome [GBS] 10 weeks after allogeneic haematopoietic stem cell transplantation [HSCT] from HLA-matched siblingíyounger sister. GBS was preceded by pneumonia, herpes labialis and oral candidiasis a week earlier. Treatment with ventilatory management, intravenous human immunoglobulin [IVIg] and antimicrobials resulted in smooth recovery in thirty-one days


Subject(s)
Humans , Male , Female , Guillain-Barre Syndrome/diagnosis , Guillain-Barre Syndrome/therapy , Anemia, Aplastic/therapy , Hematopoietic Stem Cell Transplantation
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