Excellent survival after sibling or unrelated donor stem cell transplantation for chronic granulomatous disease.

Abstract

BACKGROUND:

Matched related donor (MRD) hematopoietic stem cell transplantation (HSCT) is a successful treatment for chronic granulomatous disease (CGD), but the safety and efficacy of HSCT from unrelated donors is less certain.

OBJECTIVE:

We evaluated the outcomes and overall survival in patients with CGD after HSCT.

METHODS:

We report the outcomes for 11 children undergoing HSCT from an MRD (n = 4) or an HLA-matched unrelated donor (MUD) (n = 7); 9 children were boys, and the median age was 3.8 years (range, 1-13 years). We treated both X-linked (n = 9) and autosomal recessive (n = 2) disease. Nine children had serious clinical infections before transplantation. The conditioning regimens contained busulfan, cyclophosphamide, cytarabine, or fludarabine according to the donor used. All patients received alemtuzumab (anti-CD52 antibody). Additional graft-versus-host disease (GvHD) prophylaxis included cyclosporine and methotrexate for MUD recipients and cyclosporine and prednisone for MRD recipients.

RESULTS:

Neutrophil recovery took a median of 16 days (range, 12-40 days) and 18 days (range, 13-24 days) for MRD and MUD recipients, respectively. Full donor neutrophil engraftment occurred in 9 patients, and 2 had stable mixed chimerism; all patients had sustained correction of neutrophil oxidative burst defect. Four patients had grade I skin acute GVHD responding to topical treatment. No patient had grade II to IV acute GvHD or chronic GvHD. All patients are alive between 1 and 8 years after HSCT.

CONCLUSION:

For CGD, equivalent outcomes can be obtained with MRD or MUD stem cells, and HSCT should be considered an early treatment option.

• Authors: Caridad A Martinez, Sweta Shah, William T Shearer, Howard M Rosenblatt, Mary E Paul, Javier Chinen, Kathryn S Leung, Alana Kennedy-Nasser, Malcolm K Brenner, Helen E Heslop, Hao Liu, Meng-Fen Wu, Imelda C Hanson, Robert A Krance
• Journal: The Journal of allergy and clinical immunology (J Allergy Clin Immunol) Vol. 129 Issue 1 Pg. 176-83 (Jan 2012) ISSN: 1097-6825 [Electronic] United States
• PMID: 22078471 (Publication Type: Journal Article, Research Support, N.I.H., Extramural)
• Copyright: Copyright © 2011 American Academy of Allergy, Asthma & Immunology. Published by Mosby, Inc. All rights reserved.

Topics

• Activities of Daily Living
• Adolescent
• Child
• Child, Preschool
• Educational Status
• Female
• Graft vs Host Disease (etiology, prevention & control)
• Granulomatous Disease, Chronic (immunology, mortality, therapy)
• Hematopoietic Stem Cell Transplantation (adverse effects, mortality)
• Humans
• Infant
• Infant, Newborn
• Male
• Quality of Life
• Sibling Relations
• Tissue Donors
• Transplantation, Homologous
• Treatment Outcome
• Unrelated Donors