Haploidentical transplantation for pediatric patients with acquired severe aplastic anemia

Abstract

Techniques for haploidentical hematopoietic stem cell transplantation (haplo-HSCT) to treat severe aplastic anemia (SAA) have recently improved, but no protocol has been evaluated in a large number of pediatric patients. Fifty-two children with SAA received haplo-HSCT in our center. The treatment protocol used G-CSF-primed bone marrow with G-CSF-mobilized PBSCs without in vitro T-cell depletion. The conditioning regimen included busulfan/cyclophosphamide and antithymocyte globulin. Fifty-one patients achieved primary engraftment; one child died of regimen-related toxicity on the day +1. Secondary graft failure occurred in three patients. The cumulative incidences of aGVHD grade II-IV and grade III-IV were 39.2±0.5 and 13.7±0.2%, respectively. The cumulative incidence of cGVHD was 34.2±0.5%. The 3-year overall and failure-free survival rates were 84.5±5.0 and 82.7±5.2%, respectively, with a median follow-up time of 744.5 days (100-3294) for surviving patients. The Eastern Cooperative Oncology Group score was the only predictor of overall and failure-free survival rates. Clinical outcomes were similar between the upfront and salvage group. This result suggests that both newly diagnosed and refractory pediatric SAA patients benefit from haplo-HSCT, especially when patients are in good general condition. Therefore, haplo-HSCT might be an alternative therapy for pediatric SAA patients without HLA-matched sibling donors.

Cotransplantation of haploidentical hematopoietic stem cells and allogeneic bone marrow‐derived mesenchymal stromal cells as a first‐line treatment in very severe aplastic anemia patients with refractory infections

“…In addition, recent advances in conditioning regimen, prophylaxis of graft-vs-host disease (GVHD), and supportive care have significantly enhanced therapeutic benefits of HRD-HSCT. More importantly, different centers in China including our center have reported the feasibility and promising outcomes of using HRD-HSCT in patients with acquired SAA, achieving long-term OS of 77.3%-86.1%, [5][6][7][8] which is comparable to outcomes of patients with SAA receiving HLA-identical sibling HSCTs. 8 Furthermore, a recent finding suggests that HRD-HSCT has similar efficacy in the treatment of pediatric SAA compared to IST.…”

Outcomes and healthcare utilization in children and young adults with aplastic anemia: A multiinstitutional analysis

“…This was reflected in racial distribution of the salvage transplant group in this study as well. Availability of alternate donors with good outcomes in AA is an important area for research and recent studies have shown promising results with haploidentical and combined Haplo‐cord transplants …”

“…As these patients tend to be sicker, the number of ICU visits was also significantly higher in this group. Moving toward more upfront trans- 32,33 and combined Haplo-cord transplants. 34,35 There are many limitations to this study that are inherent to administrative database studies.…”

Allogeneic Stem Cell Transplantation in Children with Acquired Aplastic Anemia

“…Bugün EAA için antitimosit globulin (ATG) ve siklosporin-A (CsA) içeren immünosupresif tedaviler (İST) ve hematopoetik kök hücre nakli (HKHN) standart tedaviler olarak kullanılmaktadır. Bu tedaviler sayesinde son dekadlarda AEAA prognozu belirgin şekilde iyileşmiştir (1,4). Özellikle allojenik HKHN çocukluk çağındaki AEAA için devrim niteliğinde bir küratif yaklaşım olmuştur.…”

“…İleri moleküler teknikler, gelişmiş hazırlama rejimleri ve graft versus host hastalığı (GVHH) profilaksi seçenekleri birleştiğinde, eğer insan lökosit antijen (human leucocyte antigen; HLA) tam uyumlu kardeş verici (TUKV) mevcutsa HKHN çocuklardaki edinsel AA için altın standart tedavi olarak kabul edilmektedir (4,5). Farklı ülkelerden gelen veriler, TUKV ile nakil olgularında uzun süreli genel sağ kalımın (GSK) %90'ın üzerinde olduğunu göstermektedir (4). HLA uyumlu aile donörü olmayan çocuklarda ise İST terapö-tik bir seçenek olarak kullanılmaktadır (6).…”