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

[CIS-PIDD]

Dear Ele,
I just tried to get a JAK inhibitor for another indication and got ruxolitinib permitted from social security (i.e. the regular pharmacy) off label. The company could not provide it because of legal constraints with the US-manufacturer. Tofacitinib and baricitinib are not at all licensed in EU and the companies could not help in this case either.
Best,
Markus

Univ.-Prof. Dr. Markus G. Seidel | P?d. H?m. Onk. | Med. Univ. Graz | Auenbruggerpl. 38, A-8036 Graz, Austria | Tel +4331638512621 | Fax +4331638513717 | Secr +4331638513485 | Bitte keine dringenden Patienteninformationen per Email | sent from my mobile phone | please excuse typos

Am 04.02.2016 um 13:45 schrieb CIS-PIDD <cis-pidd at lists.clinimmsoc.org<mailto:cis-pidd at lists.clinimmsoc.org>>:

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

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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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