Comparable outcomes of matched-related and alternative donor stem cell transplantation for pediatric severe aplastic anemia

Alana A Kennedy-Nasser; Kathryn S Leung; Anita Mahajan; Heidi L Weiss; James A Arce; Stephen Gottschalk; George Carrum; Shakila P Khan; Helen E Heslop; Malcolm K Brenner; Catherine M Bollard; Robert A Krance

doi:10.1016/j.bbmt.2006.07.011

Comparable outcomes of matched-related and alternative donor stem cell transplantation for pediatric severe aplastic anemia

Biol Blood Marrow Transplant. 2006 Dec;12(12):1277-84. doi: 10.1016/j.bbmt.2006.07.011.

Authors

Alana A Kennedy-Nasser¹, Kathryn S Leung, Anita Mahajan, Heidi L Weiss, James A Arce, Stephen Gottschalk, George Carrum, Shakila P Khan, Helen E Heslop, Malcolm K Brenner, Catherine M Bollard, Robert A Krance

Affiliation

¹ Center for Cell and Gene Therapy, Baylor College of Medicine, The Methodist Hospital and Texas Children's Hospital, Houston, Texas 77030, USA. aakenned@txccc.org

PMID: 17162209
DOI: 10.1016/j.bbmt.2006.07.011

Abstract

Matched sibling donor (MSD) bone marrow transplantation is the treatment of choice for pediatric patients with severe aplastic anemia (SAA); however, only about 33% of patients will have an HLA-identical sibling. Alternative donor (AD) transplants may be an option for these patients, but such therapies have been associated with greater incidence of graft failure and graft-versus-host disease (GVHD). We retrospectively analyzed 36 pediatric patients who received 38 bone marrow or peripheral blood stem cell transplants (15 MSD and 23 AD) for SAA at our institution from April 1997 to October 2005. Nineteen AD recipients received reduced intensity conditioning with cyclophosphamide, low-dose total body irradiation, and antithymocyte globulin (ATG) or Campath. The 4-year overall survival for MSD recipients was 93% versus 89% for AD recipients treated with reduced intensity conditioning regimens at a median follow-up of 52 months (range, 6-99 months). No patient receiving Campath, compared with 3 of 9 patients receiving ATG, developed extensive, chronic GVHD. We conclude that, for children with SAA, AD transplantation is as effective as MSD transplantation. Further, compared with ATG, preparatory regimens containing Campath may be associated with a lower incidence of extensive, chronic GHVD.

Publication types

Comparative Study
Evaluation Study

MeSH terms

Adolescent
Alemtuzumab
Anemia, Aplastic / surgery*
Antibodies, Monoclonal / therapeutic use
Antibodies, Monoclonal, Humanized
Antibodies, Neoplasm / therapeutic use
Antilymphocyte Serum
Bone Marrow Transplantation / immunology
Bone Marrow Transplantation / statistics & numerical data*
Child
Child, Preschool
Cyclosporine / therapeutic use
Female
Graft Survival
Graft vs Host Disease / epidemiology
Graft vs Host Disease / prevention & control
Histocompatibility
Humans
Immunosuppressive Agents / therapeutic use
Infant
Infections / epidemiology
Infections / etiology
Kaplan-Meier Estimate
Male
Methotrexate / therapeutic use
Peripheral Blood Stem Cell Transplantation / statistics & numerical data*
Postoperative Complications / epidemiology
Postoperative Complications / etiology
Retrospective Studies
Siblings
Survival Analysis
T-Lymphocyte Subsets
Tacrolimus / therapeutic use
Tissue Donors*
Transplantation Conditioning
Transplantation, Homologous / immunology
Transplantation, Homologous / statistics & numerical data*
Treatment Outcome
Whole-Body Irradiation

Substances

Antibodies, Monoclonal
Antibodies, Monoclonal, Humanized
Antibodies, Neoplasm
Antilymphocyte Serum
Immunosuppressive Agents
Alemtuzumab
Cyclosporine
Tacrolimus
Methotrexate