A single-center experience using alemtuzumab, fludarabine, melphalan, and thiotepa as conditioning for transplantation in pediatric patients with chronic granulomatous disease

Sima T. Bhatt, Ginny Schulz, Monica Hente, Ashley Slater, Lisa Murray, Shalini Shenoy, Jeffrey J. Bednarski

Research output: Contribution to journalArticlepeer-review

8 Scopus citations

Abstract

Chronic granulomatous disease (CGD) is an immune deficiency characterized by defective neutrophil function and increased risk of life-threatening infections. Allogeneic hematopoietic cell transplantation is curative for CGD, and conditioning regimen impacts transplant-related outcomes. We report a single-center prospective study (NCT01821781) of four patients with CGD transplanted using a reduced-intensity conditioning regimen (RIC) containing alemtuzumab, fludarabine, melphalan, and thiotepa. Patients had early immune reconstitution with low incidence of infections. Disease-free survival was 75% at a median of five years after transplant. This RIC regimen presents an alternative approach for transplant of patients with CGD who may not tolerate busulfan-based conditioning.

Original languageEnglish
Article numbere28030
JournalPediatric Blood and Cancer
Volume67
Issue number1
DOIs
StatePublished - Jan 1 2020

Keywords

  • chronic granulomatous disease
  • immune deficiencies
  • pediatric allogeneic transplant
  • reduced-intensity conditioning

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