[CIS PIDD] [cis-pidd] Treatment Options for Griscelli Syndrome Type ii

[CIS-PIDD]

I would expand the donor search. Both of his parents are unavailable. Does he have any other living blood relatives? There is a reasonable chance that aunts, uncles, cousins, even grandparents may be haploidentical. Would also look for cords. What is the child's ethnic background?
R


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Rob Sokolic, MD
Medical Officer
Office of Cellular, Tissue and Gene Therapies
Center for Biologics Evaluation and Research
Food and Drug Administration
White Oak Building 71, Room 5268
10903 New Hampshire Ave
Silver Spring, MD 20993-0002
Robert.Sokolic at fda.hhs.gov<mailto:Robert.Sokolic at fda.hhs.gov>
(240) 402-5564
FAX: (301) 595-1305

The above transmission is meant solely for the addressee. The information contained in this message may be of a private, medical, privileged or industrial nature, and may not be communicated beyond the initial recipient. If you are not the intended recipient of this message or the agent of such recipient, please destroy all physical copies of this message, delete all electronic copies and notify the sender of the error

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From: CIS-PIDD [mailto:cis-pidd at lists.clinimmsoc.org]
Sent: Friday, September 16, 2016 2:09 PM
To: CIS-PIDD
Subject: [cis-pidd] Treatment Options for Griscelli Syndrome Type ii

Colleagues:

I follow a 2yo boy with Griscelli Syndrome Type II with our Heme/Onc folks.  He has already had at least 2 major episodes of HLH, and although he survived he has reached his lifetime dose of etoposide.

Unfortunately, both of his parents have abandoned him and cannot be located.  A potential matched unrelated donor has not been identified after 1.5 years of searching.

Two questions:

*       Are there ANY other potential therapies for this boy's genetic disorder?

*       He is currently being limited to the home environment.  The Hematologist asked if there are any circumstances under which  the patient could attend pre-school?

Thank you all.

Joe Church
Children's Hospital Los Angeles

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