[CIS PIDD] [cis-pidd] STAT3 GOF: other treatments than HSCT?

[CIS-PIDD]

Hi Ele,
There have been some folks who have tried them anecdotally, Tocilizumab has been helpful in a number of cases (including the one in our blood paper) and there are yet others with different regimens tried -- hopefully those trying them could weigh in on that. We are in the midst of writing a protocol for Jak inhibitor treatment of stat3 and stat1 GOF here at the NIH, when it is live we will be sure to notify folks.
Josh

Joshua Milner, MD
Chief, Genetics and Pathogenesis of Allergy Section, LAD, NIAID
301-827-3662
Tinyurl.com/gpasmilner<http://tinyurl.com/gpasmilner>

On Feb 4, 2016, at 7:45 AM, CIS-PIDD <cis-pidd at lists.clinimmsoc.org<mailto:cis-pidd at lists.clinimmsoc.org>> wrote:

Dear all,

I have a patient affected by STAT3 GOF with multiple organ involvement (including liver and lung) and it will be quite risky to try HSCT. Is anyone aware of other form of treatment? JAK inhibitor trials?

Any suggestion will be well appreciated.

Thanks.

Ele

*******************************************************************
Eleonora Gambineri, MD
Researcher/Assistant Professor

Department of "NEUROFARBA": Section of Child's Health
University of Florence

Department of Haematology-Oncology: BMT Unit
Department of Fetal and Neonatal Medicine: Rare Diseases,
"Anna Meyer" Children's Hospital

Viale Gaetano Pieraccini,24
50139 FIRENZE
ITALY
Tel +39 055 5662405 (office)/055 5662606(BMT ward)
Fax +39 055 5662607
e-mail: eleonora.gambineri at unifi.it<mailto:eleonora.gambineri at unifi.it>; e.gambineri at meyer.it<mailto:e.gambineri at meyer.it>

********************************************************************


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