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1.
Bone Marrow Transplant ; 55(10): 2026-2034, 2020 10.
Artigo em Inglês | MEDLINE | ID: mdl-32286505

RESUMO

In 2010, we reported the outcome of hematopoietic stem cell transplantation (HSCT) in 11 children with Griscelli syndrome type 2 (GS2). We report here the update on this cohort to include 35 patients. Twenty-seven (77%) patients received conditioning regimen including busulfan, cyclophosphamide with etoposide. Eight (23%) were given busulfan, fludarabine. Thiotepa was added to busulfan and fludarabine regimen in two patients; one received haploidentical marrow and one unrelated cord blood. Posttransplant clinical events included veno-occlusive disease (n = 7), acute (n = 8), or chronic (n = 1) graft-versus-host disease II-IV. With a mortality rate of 37.1% (n = 13) and a median follow-up of 87.7 months of the survivors, 5-year cumulative probability of overall survival (OS) for our cohort of patients was 62.7% (±8.2%). Cumulative probability of 5-year OS was significantly better in those who did not have hemophagocytic lymphohistiocytosis (HLH) prior to HSCT (100% vs. 53.3 ± 9.5%, P value: 0.042). Of the 16 patients with neurologic involvement before HSCT, 8 survived and 3 presented sequelae. OS at 5-year was 50 ± 12.5% and 73.3 ± 10.2% (P value: 0.320) in patients with and without CNS involvement, respectively. In conclusion, HSCT in patients with GS2 is potentially curative with long-term disease-free survival. Early HSCT before the development of the accelerated phase is associated with a better outcome.


Assuntos
Doença Enxerto-Hospedeiro , Transplante de Células-Tronco Hematopoéticas , Linfo-Histiocitose Hemofagocítica , Piebaldismo , Doenças da Imunodeficiência Primária , Bussulfano , Criança , Humanos , Linfo-Histiocitose Hemofagocítica/terapia , Recidiva Local de Neoplasia , Piebaldismo/terapia , Doenças da Imunodeficiência Primária/terapia , Estudos Retrospectivos , Condicionamento Pré-Transplante , Vidarabina
2.
Bone Marrow Transplant ; 54(1): 63-67, 2019 01.
Artigo em Inglês | MEDLINE | ID: mdl-29884852

RESUMO

Hyper-IgM syndrome due to CD40 deficiency (HIGM3) is a rare disease with only a few reported cases of haematopoietic stem cell transplantation (HSCT). In retrospective study, we reviewed all patients with HIGM3 who underwent HSCT at King Faisal Specialist Hospital and Research Center, Riyadh, Saudi Arabia, between 2008 and 2013. Six patients were identified. Three male and three female patients from three families. The median age of diagnosis was 13 months (range, 1-28 months). All lacked CD40 expression on B cells by flow cytometry. The median time from diagnosis to transplantation was 8.5 months (range, 1-17 months). For all patients, the donors were HLA-identical siblings, with the exception of one patient for whom the donor was a sibling with one antigen mismatch. The conditioning regimen was busulfan and cyclophosphamide in five patients and busulfan, cyclophosphamide and antithymocyte globulin in one patient. For GVHD prophylaxis, cyclosporine and methotrexate was used. All patients engrafted. The survival rate was 100%, with a median follow-up of 54 months (range, 30-116 months). One patient developed acute GVHD. All patients showed complete immune recovery with positive CD40 expression on B cells and discontinued IVIG replacement. Our study shows that HSCT is potentially effective at curing the disease.


Assuntos
Antígenos CD40/deficiência , Doença Enxerto-Hospedeiro , Transplante de Células-Tronco Hematopoéticas , Síndrome de Imunodeficiência com Hiper-IgM , Irmãos , Condicionamento Pré-Transplante , Soro Antilinfocitário/administração & dosagem , Linfócitos B/imunologia , Linfócitos B/patologia , Bussulfano/administração & dosagem , Pré-Escolar , Ciclofosfamida/administração & dosagem , Intervalo Livre de Doença , Feminino , Doença Enxerto-Hospedeiro/imunologia , Doença Enxerto-Hospedeiro/mortalidade , Doença Enxerto-Hospedeiro/patologia , Doença Enxerto-Hospedeiro/prevenção & controle , Humanos , Síndrome de Imunodeficiência com Hiper-IgM/imunologia , Síndrome de Imunodeficiência com Hiper-IgM/mortalidade , Síndrome de Imunodeficiência com Hiper-IgM/patologia , Síndrome de Imunodeficiência com Hiper-IgM/terapia , Lactente , Recém-Nascido , Masculino , Estudos Retrospectivos , Taxa de Sobrevida
3.
Bone Marrow Transplant ; 52(1): 120-125, 2017 Jan.
Artigo em Inglês | MEDLINE | ID: mdl-27618684

RESUMO

To describe the hematopoietic stem cell transplantation (HSCT) activities for children in the Eastern Mediterranean (EM) region, data on transplants performed for children less than 18 years of age between 1984 and 2011 in eight EM countries (Egypt, Iran, Jordan, Lebanon, Oman, Pakistan, Saudi Arabia and Tunisia) were collected. A total of 5187 transplants were performed, of which 4513 (87%) were allogeneic and 674 (13%) were autologous. Overall, the indications for transplantation were malignant diseases in 1736 (38.5%) and non-malignant in 2777 (61.5%) patients. A myeloablative conditioning regimen was used in 88% of the allografts. Bone marrow (BM) was the most frequent source of stem cells (56.2%), although an increasing use of PBSC was observed in the last decade. The stem cell source of autologous HSCT has shifted over time from BM to PBSC, and 80.9% of autologous HSCTs were from PBSCs. The donors for allogeneic transplants were matched-related in 94.5% of the cases, and unrelated transplants, mainly cord blood (99%) in 239 (5.5%) cases. This is the first report to describe the pediatric HSCT activities in EM countries. Non-malignant disorders are the main indication for allogeneic transplantation. Frequency of alternate donor transplantation is low.


Assuntos
Transplante de Células-Tronco Hematopoéticas , Neoplasias/terapia , Condicionamento Pré-Transplante , Adolescente , Aloenxertos , Criança , Pré-Escolar , Feminino , Humanos , Lactente , Masculino , Região do Mediterrâneo/epidemiologia , Neoplasias/epidemiologia , Estudos Retrospectivos
4.
Bone Marrow Transplant ; 52(4): 588-591, 2017 Apr.
Artigo em Inglês | MEDLINE | ID: mdl-27892944

RESUMO

Veno-occlusive disease/sinusoidal obstruction syndrome (VOD/SOS) of the liver is a serious, early complication of haematopoietic stem cell transplantation (HSCT), severe and very severe forms of which are associated with a high mortality rate. A wide variety of patient, disease and treatment-related risk factors for VOD/SOS have been identified. Several bodies have published recommendations for the diagnosis, prevention and management of VOD/SOS following HSCT. A group of regional experts have developed a consensus statement on the diagnosis, prevention and management of VOD/SOS in the Middle East and North Africa region to help in the management of HSCT patients in the region. Risk factors of particular relevance in the region include iron overload in thalassaemia patients, some hereditary metabolic disorders due to consanguinity and infection with hepatitis virus B or C. Recommendations include diagnosis of VOD/SOS based on established clinical criteria, prophylaxis with defibrotide and/or ursodeoxycholic acid in patients at increased risk of VOD/SOS, and treatment with defibrotide for patients with severe/very severe VOD/SOS (and, if clinically indicated, in those with moderate or rapidly progressing VOD/SOS, as per the new European Society for Blood and Marrow Transplantation classification).


Assuntos
Transplante de Células-Tronco Hematopoéticas/efeitos adversos , Hepatopatia Veno-Oclusiva , África do Norte , Gerenciamento Clínico , Hepatopatia Veno-Oclusiva/diagnóstico , Hepatopatia Veno-Oclusiva/etiologia , Hepatopatia Veno-Oclusiva/prevenção & controle , Hepatopatia Veno-Oclusiva/terapia , Humanos , Oriente Médio , Polidesoxirribonucleotídeos/uso terapêutico , Fatores de Risco , Ácido Ursodesoxicólico/uso terapêutico
5.
Bone Marrow Transplant ; 51(4): 536-41, 2016 Apr.
Artigo em Inglês | MEDLINE | ID: mdl-26752139

RESUMO

Allogeneic hemopoietic stem cell transplantation (HSCT) is the only method currently available to cure transfusion-dependent thalassemia major that has been widely used worldwide. To verify transplantation distribution, demography, activity, policies and outcomes inside the European Group for Blood and Marrow Transplantation (EBMT), we performed a retrospective non-interventional study, extracting data from the EBMT hemoglobinopathy prospective registry database. We included 1493 consecutive patients with thalassemia major transplanted between 1 January 2000 and 31 December 2010. In total, 1359 (91%) transplants were performed on patients <18 years old, 1061 were from a human leukocyte Ag-identical sibling donor. After a median observation time of 2 years, the 2-year overall survival (OS) and event-free survival (EFS; that is, thalassemia-free survival) were 88 ± 1% and 81 ± 1%, respectively. Transplantation from a human leukocyte Ag-identical sibling offered the best results, with OS and EFS of 91 ± 1% and 83 ± 1%, respectively. No significant differences in survival were reported between countries. The threshold age for optimal transplant outcomes was around 14 years, with an OS of 90-96% and an EFS of 83-93% when transplants were performed before this age. Allogeneic HSCT for thalassemia is a curative approach that is employed internationally and produces excellent results.


Assuntos
Transplante de Células-Tronco Hematopoéticas , Sistema de Registros , Talassemia/terapia , Adolescente , Adulto , Intervalo Livre de Doença , Europa (Continente) , Feminino , Humanos , Masculino , Pessoa de Meia-Idade , Sociedades Médicas , Taxa de Sobrevida , Talassemia/mortalidade
6.
Bone Marrow Transplant ; 45(8): 1294-9, 2010 Aug.
Artigo em Inglês | MEDLINE | ID: mdl-20062095

RESUMO

In total, 11 consecutive pediatric patients with Griscelli syndrome (GS) type 2, who received allogeneic hematopoietic SCT (aHSCT) at our center between 1993 and 2007, were reviewed. The median age at transplantation was 8.2 months (range, 4-36.3 months) and the median time from diagnosis to transplantation was 3.7 months (range, 1.4-19.5 months). Seven patients developed an accelerated phase and were treated with chemotherapy before transplantation. At the time of transplantation, all patients were in clinical remission. The source of grafts was matched-related marrows in eight patients and partially mismatched unrelated cords in three patients. All patients were engrafted at a median time of 15 days (range, 12-36 days). Grade I-II acute GVHD and veno-occlusive disease occurred in three and one patient, respectively. A total of 10 patients are now alive and disease free at a median of 4.8 years post-HSCT. The post transplant course was complicated by CMV infection in four patients. One patient died in remission from septic shock, 6 months after transplantation. Chimerism studies at the last contact are available for nine patients: six patients have complete donor chimerism and three have stable mixed chimerism. Early aHSCT from matched-related donors or unrelated cord blood for children with GS is feasible.


Assuntos
Transplante de Células-Tronco Hematopoéticas/métodos , Pré-Escolar , Infecções por Citomegalovirus , Doença Enxerto-Hospedeiro , Transplante de Células-Tronco Hematopoéticas/efeitos adversos , Transplante de Células-Tronco Hematopoéticas/mortalidade , Histocompatibilidade , Humanos , Síndromes de Imunodeficiência/complicações , Síndromes de Imunodeficiência/terapia , Lactente , Linfo-Histiocitose Hemofagocítica , Piebaldismo/complicações , Piebaldismo/terapia , Doenças da Imunodeficiência Primária , Análise de Sobrevida , Resultado do Tratamento
7.
Bone Marrow Transplant ; 45(8): 1281-6, 2010 Aug.
Artigo em Inglês | MEDLINE | ID: mdl-20010867

RESUMO

In unrelated cord blood (UCB) transplantation, survival has been shown to correlate with the degree of HLA matching. Thus, to extend transplant access to different ethnic backgrounds, many western UCB transplantation banks now encourage donation from non-Caucasians. Until recently, Saudi Arabia did not have a national UCB bank. In this study we report our experience in UCB transplantation in children using units procured from western cord blood banks. A total of 97 children underwent unrelated UCB transplantation at King Faisal Specialist Hospital and Research Center (KFSHRC), of which 95 were of Arab ethnicity. A total of 30 patients had malignant disorders, 25 patients had non-malignant hematological disorders and 42 patients had inborn errors. Conditioning was according to disease, with six patients receiving reduced-intensity regimens. In all, 46 patients received one-Ag-mismatched units and 51 received two-Ag-mismatched units. Engraftment occurred in 93% of patients, the 100-TRM was 15%, acute GVHD developed in 20% of patients and chronic GVHD occurred in 9% of patients. The 5-year OS and EFS estimates were 52 and 43%, respectively. The search for UCB transplantation units for Saudi patients in western banks yielded reasonably compatible units for our patients; the results are consistent with published data. Our data are encouraging for UCB transplant programs in countries in which there are no national UCB transplantation banks.


Assuntos
Armazenamento de Sangue/métodos , Doadores de Sangue/provisão & distribuição , Transplante de Células-Tronco de Sangue do Cordão Umbilical/métodos , Etnicidade , Criança , Pré-Escolar , Transplante de Células-Tronco de Sangue do Cordão Umbilical/efeitos adversos , Transplante de Células-Tronco de Sangue do Cordão Umbilical/mortalidade , Feminino , Doenças Genéticas Inatas/terapia , Sobrevivência de Enxerto , Doença Enxerto-Hospedeiro/etiologia , Doenças Hematológicas/epidemiologia , Doenças Hematológicas/terapia , Teste de Histocompatibilidade , Humanos , Lactente , Cooperação Internacional , Masculino , Arábia Saudita , Imunologia de Transplantes
8.
Hemoglobin ; 33 Suppl 1: S183-7, 2009.
Artigo em Inglês | MEDLINE | ID: mdl-20001624

RESUMO

From January 1998-July 2006, 62 stem cell transplantation (SCT) were performed on 60 patients with beta-thalassemia from HLA-related match donors. The overall survival (OS) and event free survival (EFS) for all patients were 94 and 77%. The outcome of allogeneic SCT in our experience is satisfactory with OS 92% and EFS 77%. Transplantation at a young age and when the disease is mild offers the best outcome. More advanced disease is associated with higher rate of rejection and severe graft versus host disease.


Assuntos
Transplante de Células-Tronco Hematopoéticas/métodos , Hospitais Especializados/classificação , Talassemia beta/terapia , Rejeição de Enxerto/imunologia , Doença Enxerto-Hospedeiro/imunologia , Transplante de Células-Tronco Hematopoéticas/efeitos adversos , Transplante de Células-Tronco Hematopoéticas/mortalidade , Humanos , Estudos Retrospectivos , Fatores de Risco , Arábia Saudita , Análise de Sobrevida , Resultado do Tratamento , Talassemia beta/complicações , Talassemia beta/mortalidade
9.
Bone Marrow Transplant ; 41(3): 261-5, 2008 Feb.
Artigo em Inglês | MEDLINE | ID: mdl-17982499

RESUMO

In the literature, there is an abundance of promising data on the outcome of allogeneic stem cell transplantation (SCT) in patients with Fanconi anemia (FA); however, the data on the outcome of FA patients who present with myelodysplasia and/or abnormal clone are sketchy as the entity itself is a rare one, although, it is believed that the presence of any of these factors confers a worse prognosis on the outcome of the transplant. This is an update of our experience in 11 such patients who underwent SCT at King Faisal Specialist Hospital and Research Center; 10 from the matched and related donors and 1 from a partially matched unrelated cord blood unit; the conditioning was with the same regimen consisting of cyclophosphamide (total of 20 mg/kg), anti-thymocyte globulin (total dose 160 mg/kg of the equine product or 52 mg/kg of the rabbit product) and total-body irradiation at 450 cGy. Ten patients remain currently alive, well and with no evidence of disease, with a median follow-up of almost 4 years.


Assuntos
Anemia de Fanconi/terapia , Sobrevivência de Enxerto , Transplante de Células-Tronco Hematopoéticas , Adolescente , Criança , Anemia de Fanconi/complicações , Feminino , Seguimentos , Humanos , Masculino , Síndromes Mielodisplásicas/complicações , Arábia Saudita , Condicionamento Pré-Transplante , Transplante Homólogo
10.
Bone Marrow Transplant ; 33(8): 829-31, 2004 Apr.
Artigo em Inglês | MEDLINE | ID: mdl-14968137

RESUMO

Five patients with confirmed congenital amegakaryocytic thrombocytopenia (CAT) underwent stem cell transplantation (SCT) from HLA-matched related donors at King Faisal Specialist Hospital and Research Center (KFSHRC). The median age at SCT was 3.2 years (range, 0.4-5 years). Conditioning regimen consisted of busulfan (BU) 4 mg/kg p.o. for 4 days (total dose of 16 mg/kg), and cyclophosphamide (CY) 50 mg/kg once daily i.v. for 4 days (total dose of 200 mg/kg). Antithymocyte globulin (ATG) was given i.v. at a dose of 30 mg/kg for 4 days pre-SCT (total of 120 mg/kg); graft-versus-host disease (GVHD) prophylaxis was with cyclosporine and methotrexate. Four patients engrafted and are alive and transfusion independent with a median follow up time of 30 months (range, 16-45 months). One patient failed to engraft and underwent a second SCT 4 months later but died of respiratory failure. We conclude that the use of allogeneic SCT may be curative for such patients.


Assuntos
Transplante de Células-Tronco Hematopoéticas , Trombocitopenia/terapia , Pré-Escolar , Feminino , Sobrevivência de Enxerto , Doença Enxerto-Hospedeiro/etiologia , Transplante de Células-Tronco Hematopoéticas/efeitos adversos , Humanos , Lactente , Masculino , Megacariócitos/patologia , Trombocitopenia/congênito , Trombocitopenia/patologia , Transplante Homólogo
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