HAPLOIDENTICAL HEMATOPOIETIC STEM CELL TRANSPLANTATION USING REDUCED-INTENSITY CONDITIONING FOR PEDIATRIC PATIENTS WITH FAMILIAL HEMOPHAGOCYTIC LYMPHOHISTIOCYTOSIS
Author(s): ,
Chenguang Jia
Affiliations:
Hematology Oncology Center,Beijing Children’s Hospital,Beijing,China
,
Bin Wang
Affiliations:
Hematology Oncology Center,Beijing Children’s Hospital,Beijing,China
Maoquan Qin
Affiliations:
Hematology Oncology Center,Beijing Children’s Hospital,Beijing,China
EHA Library. Jia C. Jun 14, 2019; 266575; PF776
Chenguang Jia
Chenguang Jia
Contributions
×
Abstract

Abstract: PF776

Type: Poster Presentation

Presentation during EHA24: On Friday, June 14, 2019 from 17:30 - 19:00

Location: Poster area

Background
Allogeneic hematopoietic stem cell transplantation (HSCT) is considered to be the only curative treatment for familial hemophagocytic lymphohistiocytosis (FHLH). Treatment of pediatric FHLH with reduced-intensity conditioning (RIC)-based haploidentical (HID) HSCT has been rarely reported.

Aims
To investigate outcomes and adverse events in patients with FHLH who received HID HSCT.

Methods
We conducted a retrospective study of five patients, including three with mutations in PRF1 and two with XIAP deficiency. Four of the five donors were heterozygous for these mutations. The conditioning regimen included fludarabine, cyclophosphamide, and antithymocyte globulin, with or without low-dose irradiation. Unmanipulated mobilized bone marrow and peripheral blood stem cells were used as the grafts. 

Results
All five patients were successfully engrafted. Four patients survived, and one patient died. All exhibited complete response (CR) after HSCT. All of the patients who survived exhibited CR to HLH without severe regimen-related complications at a median of 29.5 months (range: 23–34 months) after HSCT. Four of the five patients had mixed donor chimerism. Three patients had 17% to 87% mixed donor chimerism but remained free of disease. Four patients received donor lymphocyte infusion (DLI), which improved the level of mixed donor chimerism. One patient experienced a decrease in donor chimerism to 1% and relapsed;Four patients developed acute graft-versus-host disease (GvHD) (grade I or II), and one patient developed grade IV GvHD. 

Conclusion
HID HSCT with RIC can be considered for treatment for patients with FHLH, but the conditions and DLI regimens need to be optimized for long-term use, and more prospective studies should be performed.

Session topic: 22. Stem cell transplantation - Clinical

Keyword(s): Haploidentical stem cell transplantation, Pediatric

By continuing to browse or by clicking “Accept Terms & all Cookies”, you agree to the storing of third-party cookies on your device to enhance your user experience and agree to the user terms and conditions of this learning management system (LMS).

Cookie Settings
Accept Terms & all Cookies