Ağır edinsel aplastik anemili çocuklarda kök hücre transplantasyonu: 15 olgunun değerlendirilmesi

Amaç: Ülkemiz koşullarında, çocukluk çağı edinsel aplastik anemisi tanısıyla kök hücre transplantasyon ünitelerine ulaşan olguların klinik ve prognostik özelliklerini ve yapılan kök hücre transplantasyonunun sonuçlarını belirlemektir. Gereç ve Yöntem: 15 olguya ağır aplastik anemi tanısı ile 16 kök hücre transplantasyonu uygulandı. Donör, 13 olguda HLA tam uygun kardeş (MSD), 1 olguda 1 antijen uygunsuz baba (MRD) ve 1 olguda HLA tam uygun akraba dışı verici (MUD) idi. Olguların ortanca yaşları 12 (3,6–15 yaş ) olup, aplastik anemi tanısından sonra geçen süre ortanca 6 ay ( 3–29 ay) idi. Kök hücre transplantasyonu öncesi bir olgu immunsupresif tedavi almıştı. Hazırlama rejiminde 12 olguda siklofosfamid 200mg/kg+Anti-timosit globulin 40 mg/kg kullanıldı, 3 olgunun 4 transplantasyonunda bunlara fludarabin eklendi. Kök hücre kaynağı 11 (%69) transplantasyonda periferik kan idi. Graft-versus-host hastalığı profilaksisinde siklosporin ve kısa süreli metotreksat kullanıldı. Bulgular: Lökosit ve trombosit engrafmanı, sırasıyla ortanca 17. (11-24) ve 18.(14-35) günlerde gerçekleşti. Akraba dışı vericiden KHT yapılan olguda gelişen engrafman yetersizliği nedeniyle, posttransplant 5. ayda aynı donörden 2. KHT yapıldı. Post-transplant erken dönemde 6 olguda bakteriyel infeksiyon, 2 olguda mantar infeksiyonu gelişti. Ortanca 3.6 yıllık (1.4-5.4 yıl) izlem süresi içinde bir olguda(%7) grade 2 akut Graft-versus-host hastalığı, bir olguda sınırlı kronik Graft-versus-host hastalığı, 1 olguda posttransplant lenfoproliferatif hastalık gelişti. Tüm olgular hastalıksız yaşamakta olup, 5-yıllık event-free survival (EFS) %94, overal survival (OS) ise %100 olarak bulundu. Sonuç: Ağır aplastik anemili çocuklarda kök hücre transplantasyonu etkilidir. Uygun kardeş vericisi olmayan olgularda, hızla araştırılacak aile içi ve dışı alternatif donörler, iyi destek bakım koşulları ve transplantasyon planlaması ile yüz güldürücü sonuçları ortaya çıkartabilmektedir.

Allogeneic hematopoietic stem cell transplantation in children with acquired severe aplastic anemia: Analysis of 15 patients

Aim: The data of allogeneic hematopoietic stem cell transplantation (HSCT) in children with acquired aplastic anemia between February 2006 and July 2010 were analyzed retrospectively. Materials and Methods: Fifteen patients with a median age of 12 years underwent transplantation including 13 matched sibling donors (MSD), 1 matched related donor (MRD) and 1 matched unrelated donor (MUD). One patient, who had been transplanted from an unrelated donor, received a second transplantation successfully after a primary graft failure from the same donor. The median time to transplantation from diagnosis was 6 months (range: 3-29 months). Conditioning regimens were ATG (40 mg/kg) and “cyclophosphamide” (200 mg/kg) in 12 patients and “fludarabine” (150 mg/m2) was added in 3 patients who received many transfusions. The stem cell source was peripheral blood in 11 (69%) transplantations. Cyclosporin and short term methotrexate were given for graft-versus-host disease (GVHD) prophylaxis in all patients. Results: The median duration of time to neutrophil and platelet engraftment was 17 and 18 days, respectively. Grade II acute GVHD developed in 1(7%) patient, and limited chronic GVHD developed in another patient (7%). Lymphoprolipherative disease appeared in a patient who received transplantation from a MUD. The overall 5-year survival rate was 100% and EFS was 94%. All recipients are alive without any graft failure in a median 3.6 years (range: 1.4-5.4 years). Conclusion: In our experience, HSCT from a MSD remains the first choice of treatment for pediatric patients with severe aplastic anemia. For patients without a MSD, transplantation from an unrelated or alternative related donor can also result in long-term survival.

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