NEW YORK (Reuters Health) - Reduced intensity hematopoietic stem-cell transplantation (HSCT) is an effective therapy for many non-malignant diseases in children that produces less toxicity than conventional myeloablative HSCT, according to a report in The Lancet for July 10th.
Compared with myeloablative HSCT, “reduced intensity HSCT involves giving much lower doses of chemotherapy and radiotherapy--just enough to achieve a level of immunosuppression that allows donor cells to engraft,” lead author Dr. David A. Jacobsohn, from Children’s Memorial Hospital in Chicago, explained to Reuters Health. “The goal is to minimize short- and long-term toxicity.”
In the new study, 13 pediatric patients underwent reduced intensity HSCT at the authors’ institution between 2000 and 2004. The children had a variety of diseases including sickle cell disease, x-linked hyper-IGM, beta-thalassemia, Sandhoff disease, mucolipidosis, chronic granulomatous disease, Hurler’s syndrome, Omenn’s syndrome, x-linked lymphoproliferative disease, and severe combined immunodeficiency.
“As far as I’m aware, our study is the first to look at reduced intensity HSCT as a treatment for metabolic disorders, such as Hurler’s syndrome or mucolipidosis,” Dr. Jacobsohn said.
Two patients died before post-transplant day 100 of underlying disease and viral infection, respectively, the authors note. Full donor engraftment was achieved in 8 of 11 (72%) evaluable patients. Most patients with hemoglobinopathies rejected the graft. Platelet and neutrophil reconstitution occurred at a median of 13.5 and 18 days, respectively.
Short-term toxic effects were minimal and the median hospital stay was 7 days, the authors state. One of twelve patients (8%) developed acute graft-versus-host disease (GVHD).
Dr. Jacobsohn said that the engraftment rate achieved in the current study compares favorably with that of myeloablative HSCT and that the rate of GVHD was lower.
Because of poor engraftment, “reduced intensity HSCT is not indicated for hemoglobinopathies,” Dr. Jacobsohn said. “Sickle-cell disease and beta-thalassemia constitute a relatively large patient population, so finding a kinder, gentler regimen for such patients would be fantastic.”
Dr. Jacobsohn said his team is currently investigating the use of photophoresis to improve engraftment rates after HSCT and decrease the risk of GVHD.
Source: Lancet 2004;364:156-162. [ Google search on this article ]
MeSH Headings:Cell Transplantation: Hematopoietic Stem Cell TransplantationCopyright © 2002 Reuters Limited. All rights reserved. Republication or redistribution of Reuters content, including by framing or similar means, is expressly prohibited without the prior written consent of Reuters. Reuters shall not be liable for any errors or delays in the content, or for any actions taken in reliance thereon. Reuters and the Reuters sphere logo are registered trademarks and trademarks of the Reuters group of companies around the world.
