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

Sat Sep 17 04:28:07 EDT 2016

As stated before, he needs a transplant. If he doesn't have any relatives available, what is the best unrelated donor you have? If you have a 7 or 8/10 match you may try intensified post-grafting immunosuppression with cyclophosphamide.
Michael

PD Dr. med. Michael Albert
Attending physician / Head Pediatric SCT
Pediatrics / Pediatric Hematology/Oncology

Dr. von Hauner Children's Hospital
Klinikum der Universität München
Lindwurmstr. 4, 80337 München
Germany

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Von: CIS-PIDD [mailto:cis-pidd at lists.clinimmsoc.org]
Gesendet: Samstag, 17. September 2016 00:12
An: CIS-PIDD
Betreff: RE: [cis-pidd] Treatment Options for Griscelli Syndrome Type ii

I agree with colleagues that transplant is the only curative therapy in the long run for this patient. I realize the lack of MUD and haploidentical donors and agree with an expanded donor search. We have transplanted an infant with fulminant HLH who had features of Griscelli syndrome, using unrelated cord blood transplant. Patient is 4 years post-transplant and doing well.  Details can be accessed in the publication listed below. I am happy to discuss more details.

Congenital hemophagocytic lymphohistiocytosis presenting as thrombocytopenia in a newborn.
Hinson A, Owen W, Prose N, Parikh S, Thornburg C.
J Pediatr Hematol Oncol. 2015 May;37(4):300-3

Regards,

Suhag H. Parikh, M.D.
Associate Professor of Pediatrics
Duke University School of Medicine
Attending Physician
Pediatric Blood and Marrow Transplant Program
Duke University Medical Center
Box 3350, Durham, NC 27710
Tel: 919-668-1121
Fax: 919-668-1180

From: CIS-PIDD [mailto:cis-pidd at lists.clinimmsoc.org]
Sent: Friday, September 16, 2016 5:34 PM
To: CIS-PIDD
Subject: Re: [cis-pidd] Treatment Options for Griscelli Syndrome Type ii

I agree, the search needs to be expanded because transplant is what he needs. In the meantime, if he has another episode of HLH, he would be eligible for NI-0501, anti-interferon-gamma. He won't be eligible without active disease though, so you might want to establish contact with a center where the trial is open. I can email you off line with details.

Ashish

Ashish Kumar, MD, PhD
Associate Professor of Pediatrics
Cincinnati Children's Hospital Medical Center
Bone marrow transplantation and immune deficiency

On Sep 16, 2016, at 4:29 PM, CIS-PIDD <cis-pidd at lists.clinimmsoc.org<mailto:cis-pidd at lists.clinimmsoc.org>> wrote:
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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Food and Drug Administration
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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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