[CIS PIDD] [cis-pidd] BMT for CGD

[Cowan, Mort]

I agree with Joe.  Assuming he has low to no oxidase activity his prognosis is guarded and with an HLA matched sibling and the reduced toxicity regimens (even with high dose busulfan) available today, his chances of a good outcome are very good. We would definitely encourage transplant now before he gets something more serious.

Mort

Morton J. Cowan, M.D.
Professor of Pediatrics
Chief, Allergy, Immunology, and Blood and Marrow Transplant Division
UCSF Children's Hospital, Room M659
505 Parnassus Ave
San Francisco, CA 94143-1278

Phone: 415-476-2188
FAX: 415-502-4867

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From: Church, Joseph [mailto:JChurch at chla.usc.edu]
Sent: Wednesday, February 20, 2013 1:54 PM
To: CIS-PIDD
Subject: RE:[cis-pidd] BMT for CGD

Dr. Infante:

I believe that this patient's long term prognosis is poor.  I know there are exceptions, but particularly in this case (sibling donors) I would not bet on a good outcome without transplant.

I would suggest to the parents  that their son's "disease is medically manageable," but only to the point that he gets a major opportunistic infection  My current strategy is to refer every new CGD to our BMT group.  Those with donors will go to transplant.  Those without donors we manage as best we can.

Because these patients usually require full conditioning, the transplant does have significant risk, but I believe that this risk is far less than the liklihood of an early demise due to CGD complications.

Gene therapy trials are opening, but given his extraordinary luck in have two sibling donors I wouldn't wait.

If it were ME, I would opt for the transplant.

Joe Church
Children's Hospital Los Angeles

From: Infante, Anthony J [mailto:INFANTEA at uthscsa.edu]
Sent: Wednesday, February 20, 2013 1:29 PM
To: CIS-PIDD
Subject: [cis-pidd] BMT for CGD

I'm following a 17 year old young man with x-linked CGD, confirmed in infancy by both phenotypic NBT carrier analysis and NOX2 genetic mutation. He has been on prophylaxis with interferon-gamma, trimethoprim-sulfa and itraconazole for many years. His last major infection involved multiple abscesses of left axillary lymph nodes and skin from axilla to elbow  by Nocardia at the age of 5-6 years which took about a year to fully resolve despite sensitivity to ampicillin. Since that time he has had an occasional skin abscess. He has two HLA-matched siblings. His parents have consistently refused to seriously consider hematopoietic stem cell transplantation because of the peri-transplant risks and the opinion that his disease is medically manageable. On a recent routine clinic visit, the patient announced his intention to request HSCT upon achieving emancipation at age 18 because he is tired of taking medications. Your replies should address both the art and science of medicine!

Anthony J. Infante, MD, PhD
Professor, Department of Pediatrics
Associate Chairman for Research & Academic Affairs
Chief, Division of Immunology & Infectious Diseases
University of Texas Health Science Center at San Antonio
7703 Floyd Curl Drive MC7802
San Antonio, TX 78229-3900
infantea at uthscsa.edu<mailto:infantea at uthscsa.edu>
tel.210-567-0510
fax 210-567-6305
Admin. asst. Stella Wise 210-567-5250
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