Reduced-intensity conditioning for hematopoietic cell transplant for HLH and primary immune deficiencies

Author:

Allen Carl E.12,Marsh Rebecca3,Dawson Peter4,Bollard Catherine M.5,Shenoy Shalini6,Roehrs Philip7,Hanna Rabi8,Burroughs Lauri9,Kean Leslie91011,Talano Julie-An12,Schultz Kirk R.13,Pai Sung-Yun14,Baker K. Scott1011,Andolina Jeffrey R.15,Stenger Elizabeth O.16,Connelly James17,Ramirez Alyssa4,Bryant Christopher4,Eapen Mary18,Pulsipher Michael A.19

Affiliation:

1. Texas Children’s Cancer Center, Texas Children’s Hospital, Houston, TX;

2. Division of Pediatric Hematology-Oncology, Department of Pediatrics, Baylor College of Medicine, Houston, TX;

3. Division of Bone Marrow Transplantation and Immune Deficiency, Cincinnati Children’s Hospital Medical Center, Cincinnati, OH;

4. The Emmes Corporation, Rockville, MD;

5. Center for Cancer and Immunology Research, Children’s National Health System and Department of Pediatrics, The George Washington University, Washington, DC;

6. Division of Pediatric Hematology-Oncology, Washington University School of Medicine, St. Louis, MO;

7. Levine Children’s Hospital, Carolinas HealthCare System, Charlotte, NC;

8. Department of Pediatric Hematology and Oncology and BMT, Cleveland Clinic, Cleveland, OH;

9. Ben Towne Center for Childhood Cancer Research, Seattle Children’s Research Institute, Seattle, WA;

10. Clinical Research Division, Fred Hutchinson Cancer Research Center, University of Washington School of Medicine, Seattle, WA;

11. Seattle Children’s Hospital, Seattle, WA;

12. Department of Pediatric Hematology and Oncology, Medical College of Wisconsin, Milwaukee, WI;

13. Division of Pediatric Hematology and Oncology, Department of Pediatrics, University of British Columbia, Vancouver, BC, Canada;

14. Division of Pediatric Hematology-Oncology, Boston Children’s Hospital and Department of Pediatric Oncology, Dana-Farber Cancer Institute, Boston, MA;

15. Division of Pediatric Hematology/Oncology, Department of Pediatrics, Golisano Children’s Hospital, University of Rochester Medical Center, Rochester, NY;

16. Aflac Center and Blood Disorders Center, Children’s Healthcare of Atlanta, Emory University, Atlanta, GA;

17. Division of Pediatric Hematology-Oncology, Department of Pediatrics, University of Michigan, Ann Arbor, MI;

18. Center for International Blood and Marrow Transplant Research, Department of Medicine, Medical College of Wisconsin, Milwaukee, WI; and

19. Division of Hematology, Oncology and Blood & Marrow Transplantation, Children’s Hospital Los Angeles, Los Angeles, CA

Abstract

Key PointsA prospective reduced-intensity HCT trial for HLH/primary immunodeficiency resulted in low early mortality and 1-year OS of 80%. Conditioning with fludarabine, melphalan, and alemtuzumab was associated with high rates of mixed chimerism and graft failure.

Publisher

American Society of Hematology

Subject

Cell Biology,Hematology,Immunology,Biochemistry

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