Doctors have been using hematopoietic stem cell (HSC) transplants to treat nonmalignant disorders in children for many years. This has included dozens of different blood disorders, immune disorders, and metabolic disorders.
Traditionally, the patient would receive a high dose chemotherapy treatment before receiving their stem cell transplant. This is performed to destroy any mutated cells and make room for the transplanted stem cells. Unfortunately, the chemotherapy treatment can have severe and sometimes life-threatening side effects.
To avoid these side effects, doctors began using reduced-intensity chemotherapy conditioning. While this approach decreased treatment-related morbidity and mortality, it would increase the risk of graft failure and infections in some patients.
A recent study, published in the journal Blood Advances, describes a new regime that combines reduced-intensity conditioning with umbilical cord blood stem cells. It has been shown to make stem cell transplants safer and more affective in children with genetic noncancerous disorders.
Senior author Dr. Paul Szabolcs said of the study: “I first encountered many of these diseases when I was at Duke University, and in 2008 I designed the predecessor of this trial, which already showed that engraftment [with a reduced-intensity conditioning regimen] was not inferior compared with myeloablative [conditioning].”
Dr. Szabolcs continued, “That was a big thing, because reduced-intensity conditioning, with its obvious benefits, has led to more graft failure. The predecessor trial had already had engraftment success, but the [OS] was not yet superior.”
The new treatment regimen tested by researchers involves several drugs including alemtuzumab, melphalan, hydroxyurea, fludarabine, and thiotepa. Once each patient had completed their drug treatments, they receive a single-unit umbilical cord blood graft.
The researchers have successfully tested the regimen on 44 children with a variety of conditions including Krabbe disease, sickle cell disease, thalassemia, and Hunter syndrome. All transplants obtained persistent engraftment, with extremely low transplant-related mortality. The researchers believe this type of universal stem cell treatment could be made available to the general public within a few years.
Source: Cord blood may be effective for treatment of 20 pediatric disorders