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Effect of different conditioning regimens on survival and engraftment for children with hemophagocytic lymphohistiocytosis undergoing allogeneic hematopoeitic stem cell transplantation: A single institution experience
Author(s) -
Ali Salah,
Wall Donna A.,
Ali Muhammad,
Chiang KuangYueh,
Naqvi Ahmed,
Weitzman Sheila,
Gassas Adam,
Gibson Paul,
Brager Rae,
Fernandez Conrad V.,
Crooks Bruce,
Schechter Tal,
Krueger Joerg
Publication year - 2020
Publication title -
pediatric blood and cancer
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.116
H-Index - 105
eISSN - 1545-5017
pISSN - 1545-5009
DOI - 10.1002/pbc.28477
Subject(s) - medicine , regimen , transplantation , hematopoietic stem cell transplantation , treosulfan , hemophagocytic lymphohistiocytosis , incidence (geometry) , cohort , busulfan , surgery , gastroenterology , disease , physics , optics
Background Hemophagocytic lymphohistiocytosis (HLH), a rare hyperinflammatory immuneregulatory disorder, is a challenge in hematopoietic stem cell transplantation (HSCT) because of the high rate of mixed chimerism, relapse, and graft failure (GF) unless intensive myeloablative regimens are used. However, historically conventional myeloablative regimens (conv MA) are associated with high toxicity and mortality. Procedure We retrospectively compared transplant outcomes between three preparative regimens of varying intensities: Conv MA (n = 15), reduced‐intensity conditioning (RIC, n = 12), and a treosulfan‐based reduced‐toxicity conditioning (RTC, n = 9). Results Patients in the RIC cohort had a higher incidence of mixed donor chimerism and five patients (42%) developed secondary GF ( P = .002) compared to the other two regimens. There was a higher incidence of veno‐occlusive disease and intensive care unit (ICU) admissions in the Conv MA cohort. With the RTC regimen, there was a similar 2‐year overall survival (89, 73, and 83%; P = .87), but improved compound EFS (lack of relapse, GF, second transplant or additional donor cell infusions, or death; 89, 73, and 42%, P = .041) in RTC, Conv MA, and RIC regimen, respectively. Conclusions The intensity of the preparative regimen has a significant impact on outcome of HSCT for HLH. The newly described treosulfan‐based RTC provides for a stable graft with a reasonable toxicity profile.