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

[CIS-PIDD]

Hi Joe,
Very sad story…as his neurological condition permits, I see now reason why this poor kid cannot/should not attend preschool. In terms of his curative approach, as long as you can use non-VP16-based accelerated phase treatments (eg IVIG, steroids, anti-cytokines, rituxan if EBV, etc) until finding a suitable donor he might have a chance (…and he might have not too many as none of our pts survived beyond a 3rd HLH/accelarated phase without a transplant).
Sergi

From: CIS-PIDD <cis-pidd at lists.clinimmsoc.org<mailto:cis-pidd at lists.clinimmsoc.org>>
Reply-To: CIS-PIDD <cis-pidd at lyris.dundee.net<mailto:cis-pidd at lyris.dundee.net>>
Date: Friday, September 16, 2016 at 2:08 PM
To: CIS-PIDD <cis-pidd at lyris.dundee.net<mailto:cis-pidd at lyris.dundee.net>>
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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