The CGD Cafe

[CGDAdmin]

One of the doctor's at NIH just sent me this. I also posted it to the front page.

Stem Cell Transplantation for Chronic Granulomatous Disease at the National Institutes of Health, U.S.A.

Stem cell transplantation is the only treatment available that can cure Chronic Granulomatous Disease (CGD). Stem cells are the seeds of all blood cells found in the bloodstream. Transplantation of stem cells from a normal donor allows for production of normal blood cells that can protect people with CGD from developing life-threatening infections. However, stem cell transplantation is a risky procedure. The National Institutes of Health (NIH) in Bethesda Maryland has a large group of doctors and scientists who are working hard to make stem cell transplantation safer. We have been studying stem cell transplantation for CGD since 1998. In a recently completed study, 13 CGD patients (eight children and five adults) were treated with stem cell transplantation. Of these thirteen patients, five children and two adults have been cured of CGD. Three children treated on the study rejected the stem cell transplant and retain the diagnosis of CGD. Three of the adult patients died from complications.

Each year, people with the most severe form of CGD face a 3-5% chance of dying. This means that the chance of dying before the age of 20 is about 50%. Investigators at the NIH have recently opened a new treatment protocol of stem cell transplantation for patients with CGD. The goal of this study is to show that our experimental approach to stem cell transplantation can reduce the long-term risk of dying and improve the quality of life for patients with CGD. The study focuses on treatment of patients under 18 years old with the most dangerous (X-linked or p22phox deficient) form of CGD. Children with suitably matched sibling donors capable of donating stem cells will be eligible for the stem cell transplant arm of the study. Children without a suitable donor will be eligible for the standard of care arm of the study.

Doctors at the NIH care for over 200 patients with CGD and are dedicated to providing state of the art treatment for such patients. The NIH will pay all travel, lodging and medical expenses associated with participation in this study. Special arrangements can be made for the parent or guardian of the patient as well.

If you or your child has CGD, we invite you to come NIH and learn more about our treatment protocols. To arrange for a visit, please contact Pat Woltz toll free at 1-888-659-0925 or 301-402-5964 (outside the United States) or by e-mail at pwoltz@niaid.nih.gov.

[KimMcFarland]

Hello,
My son is in the protocal..
Hes had the best care there(NIH). Currently he spent 3 weeks there at nih..He'll be going back in 3 for follow up. Its great to get a second opinion, not use to him being treated in advance for something so horrible as aspergillus. Imm use to him getting deathly ill before anyone notice, and seem to worry more than me and thats alot. I couldnt as for a better chance for advance treatment.