Hematopoietic Cell Transplantation and Novel Therapies in Hemophagocytic Lymphohistiocytosis

Chapter

Abstract

Hematopoietic cell transplantation (HCT) is currently the only curative therapy for primary HLH. While HCT carries substantial risks for patients with HLH, HCT is essential for preventing future episodes of fatal HLH in patients with genetic predisposition to HLH. Survival with HCT has increased significantly in recent years with the adoption of reduced intensity conditioning (RIC) regimens. However, RIC treatment protocols carry their own distinct issues including the frequent development of mixed chimerism. Patients with HLH are also challenging due to the potential for recurrence of HLH post HCT. Novel therapies, including interferon neutralization and genetic therapies, may facilitate initial and definitive therapy for future patients with HLH.

Keywords

Hematopoietic cell transplantation Reduced intensity conditioning Mixed chimerism Gene therapy 

Abbreviations

CNS

Central nervous system

GVHD

Graft-versus-host disease

HCT

Hematopoietic cell transplantation

HLH

Hemophagocytic lymphohistiocytosis

MAC

Myeloablative conditioning

RIC

Reduced intensity conditioning

UCB

Umbilical cord blood

VOD

Veno-occlusive disease

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Copyright information

© Springer International Publishing AG 2018

Authors and Affiliations

  1. 1.Clinical Research Division, Fred Hutchinson Cancer Research Center, Department of Pediatrics, University of WashingtonSeattleUSA
  2. 2.Divisions of Immunobiology, and Bone Marrow Transplantation and Immune Deficiency, Department of PediatricsCincinnati Children’s Hospital Medical Center, University of CincinnatiCincinnatiUSA

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