Sharma Anil, Rastogi Neha, Chatterjee Goutomi, Kapoor Rohit, Nivargi Sagar, Yadav Satya P
Pediatric Hematology Oncology and Bone Marrow Transplant Unit, Cancer Institute, Medanta The Medicity Hospital, Gurgaon, Haryana, India.
J Pediatr Hematol Oncol. 2021 Oct 1;43(7):e1033-e1036. doi: 10.1097/MPH.0000000000002030.
Haploidentical family donor is universally available and is fast emerging as an alternative donor choice for children with leukemia needing hematopoietic stem cell transplant (HSCT). Here we describe our experience of treating children with acute leukemia by haploidentical HSCT with posttransplant cyclophosphamide (PTCy).
We retrospectively analyzed the outcome data of 17 children with acute leukemia who underwent related haploidentical HSCT. Fifteen were in complete remission (CR) before HSCT: CR1-6, CR2-7, and CR3-2 and 2 were not in remission. Donors were mobilized with granulocyte colony stimulating factor. The conditioning was nonmyeloablative in 4 and myeloablative in 13. All received PTCy 50 mg/kg on days 3 and 4 as graft-versus-host disease (GVHD) prophylaxis along with tacrolimus or cyclosporine and mycophenolate mofetil. A median of 8.94 million of CD34+ cells/kg was infused.
All patients were engrafted for neutrophil and platelets, except 1 child with refractory acute myeloid leukemia disease who relapsed before engraftment. Five children relapsed (4 died and 1 child with CD20-positive leukemia is disease free after Rituximab therapy). There was 1 transplant-related mortality due to grade IV GVHD. Remaining 11 patients are in CR. Acute GVHD was seen in 4 patients. Of 4, 3 children later developed chronic GVHD and all are alive and disease free. Three of 4 children who received nonmyeloablative conditioning have relapsed. Overall survival is 70.5% and event-free survival is 64.7%. Median follow-up of all patients was 393 days.
Haploidentical HSCT with PTCy is a safe and effective therapy for children with acute leukemia. Myeloablative conditioning and chronic GVHD lead to improved disease-free survival.
单倍体相合的家族供体普遍可得，并且正迅速成为需要造血干细胞移植（HSCT）的白血病患儿的一种替代供体选择。在此，我们描述我们采用移植后环磷酰胺（PTCy）进行单倍体相合HSCT治疗急性白血病患儿的经验。
我们回顾性分析了17例接受相关单倍体相合HSCT的急性白血病患儿的结局数据。15例在HSCT前处于完全缓解（CR）状态：CR1期6例、CR2期7例、CR3期2例，2例未缓解。供体采用粒细胞集落刺激因子进行动员。4例采用非清髓性预处理，13例采用清髓性预处理。所有患儿在第3天和第4天均接受50mg/kg的PTCy以预防移植物抗宿主病（GVHD），同时联合使用他克莫司或环孢素以及霉酚酸酯。中位数为每千克体重输注894万个CD34+细胞。
除1例难治性急性髓系白血病患儿在植入前复发外，所有患者的中性粒细胞和血小板均实现植入。5例患儿复发（4例死亡，1例CD20阳性白血病患儿在接受利妥昔单抗治疗后无病生存）。有1例因IV级GVHD导致的移植相关死亡。其余11例患者处于CR状态。4例患者出现急性GVHD。其中4例中有3例患儿后来发展为慢性GVHD，且均存活且无病生存。接受非清髓性预处理的4例患儿中有3例复发。总生存率为70.5%，无事件生存率为64.7%。所有患者的中位随访时间为393天。
采用PTCy的单倍体相合HSCT是治疗急性白血病患儿的一种安全有效的疗法。清髓性预处理和慢性GVHD可提高无病生存率。
