소아 급성 골수구성 백혈병에서의 반일치 동종조혈모세포이식의 경험

소아 급성 골수구성 백혈병에서의 반일치 동종조혈모세포이식의 경험

Haploidentical Hematopoietic Stem Cell Transplantation using ex vivo T cell depletion in Pediatric Acute Myeloid Leukemia

(구연):
Release Date : 2017. 10. 26(목)
Sung Han Kang, Jae Won Yoo, Hyeri Kim, Kyung-Nam Ko, Jong Jin Seo , Ho Joon Im
Ulsan University Asan Medical Center Department of Pediatrics1
강성한, 유재원, 김혜리, 고경남, 서종진 , 임호준
울산의대 서울아산 어린이병원 소아청소년과1

Abstract

Currently, haploidentical hematopoietic cell transplantation (HHCT) is considered an established option for patients who have diseases curable with hematopoietic cell transplantation (HCT) but who lack a suitable donor. We report the results of a prospective study to evaluate the feasibility and efficacy of ex vivo T cell-depleted (TCD) HHCT for children with acute myeloid leumekia (AML). Twenty-nine patients with AML received TCD HHCT at Asan Medical Center Children’s Hospital between July 2008 and May 2017. Six received CD3-depleted HHCT (CD3-HHCT) and 23 received TCRαβ-depleted graft (TCRαβ-HHCT). Among 29 patients, 12 were in CR1, 7 in CR2 and 10 in non-CR. Six patients had relapsed after previous allogeneic HCT. Risk groups of patients were classified as high-risk (HR; patients with relapsed/refractory disease, or relapse after HCT) and low-risk (LR; patients in CR without any of the HR feature). Of the 29 patients, 16 were LR and 13 were HR. The median age at HHCT was 11 years (range, 1-19). All 29 patients achieved neutrophil engraftment at a median of 10 days (range, 9-17) post-HHCT. None experienced graft rejection. The cumulative incidence (CI) of acute GVHD grade II-IV and III-IV were 28.4% and 14.8%, respectively. Two of 26 evaluable patients developed extensive chronic GVHD with a CI of 8.6%. As of August 20, 2017, 19 of the 29 patients survived free of disease with a median follow-up of 12.1 months (range, 1.17-111.20 months). Causes of death of 9 patients were relapse (n=7), comorbid moyamoya disease (n=1) and disseminated tuberculosis (n=1), leading to treatment-related mortality (TRM) of 10.3%. The Kaplan-Meier estimated EFS and OS for all patients were 58.9% and 65.3%, respectively. The EFS for 10 patients with relapsed/refractory disease at the time of HHCT was worse than that for the remaining 19 patients (18.8% vs 76.7%, P=0.004). In addition, the outcome for 6 patients who had received previous allogeneic transplant was poor with EFS of 33.3%. According to risk groups, patients of LR had a significantly better survival than those of HR (86.2% vs 24.6%, P=0.001). In this study, HHCT using ex vivo T-cell depleted grafts showed a favorable outcome for pediatric patients with hematologic malignancy in CR. However, further study is warranted to improve the outcome for patients with relapsed and refractory malignant disease.

Keywords: haploidentical hematopoietic stem cell transplantation, ,