Engraftment and survival following hematopoietic stem cell transplantation for osteopetrosis using a reduced intensity conditioning regimen.

Full Abstract

Autosomal recessive osteopetrosis (OP) is a disease characterized by osteoclast dysfunction, leading to multisystem morbidity and death of most affected children. Hematopoietic stem cell transplantation (HSCT) is the treatment of choice for OP, but this patient population is particularly prone to post-transplant complications and death after myeloablative conditioning. To determine the potential of achieving improved overall outcomes in these patients by decreasing pre-transplant mortality, we investigated engraftment and survival following a reduced intensity regimen including busulfan, fludarabine and total lymphoid irradiation. We report outcomes in 11 patients. All six patients who received a bone marrow or peripheral stem cell graft engrafted with >75% donor chimerism. In contrast, all five recipients of unrelated cord blood as a stem cell source for a first graft failed to demonstrate donor hematopoietic chimerism. The day 100 and 6-month mortality was low at 9%. One year after HSCT, six of 11 patients (55%) were surviving. Our data suggest that this regimen results in low peri-transplant mortality without compromising engraftment when a marrow or peripheral stem cell graft is used. An umbilical cord blood graft, however, should be used with caution for patients with OP when this or a similar reduced intensity regimen is used.

Learn Faster Today Improve your study skills

Author information

Author/s: Tolar, J (J); Bonfim, C (C); Grewal, S (S); Orchard, P (P);

Affiliation: Division of Hematology, Oncology, Blood and Marrow Transplantation, Department of Pediatrics, University of Minnesota, Minneapolis, MN 55455, USA. tolar003(-atsign-)umn.edu

Journal and publication information

Publication Type: Clinical Trial; Journal Article; Research Support, Non-U.S. Gov't

Journal: Bone marrow transplantation (Bone Marrow Transplant), published in England. (Language: eng)

Reference: 2006-Dec; vol 38 (issue 12) : pp 783-7

Dates: Created 2006/11/29; Completed 2007/01/09;

PMID: 17086207, status: MEDLINE (last retrieval date: 12/26/2008)

Sourced from the National Library of Medicine. Abstract text and other information may be subject to copyright.

External Links for this article (including full text providers, if available):

Click Electronic Full-text Provider Links to see options for finding the electronic full text links to this article. Note there may be a subscription or fee required for access to the full text. See our FAQ for information on finding FREE full text articles.

This article may also be located in paper journal collections available in many libraries. Use the Journal and Publication Information above to find the full article.

MeSH headings (categories)

This article was linked to the MESH Headings shown below.

Child, Preschool
Female
Follow-Up Studies
Genetic Diseases, Inborn - mortality; therapy
Graft Survival
Graft vs Host Disease - etiology; mortality

Hematopoietic Stem Cell Transplantation - adverse effects
Humans
Infant
Male
Osteopetrosis - complications; mortality; therapy
Transplantation Conditioning - methods

These are the highest related articles currently in the database:

See 100+ related articles.