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

Thu Feb 4 09:44:45 EST 2016

Just to reiterate what others have said, the patient we reported in our Blood paper on tocilizumab has now been on this medication for almost 3 years.  He also had autoimmune hepatitis (to the point that transplant was being considered), and his liver stabilized on tocilizumab to the point that all other immune suppressants were weaned off and he is maintained on tocilizumab alone.

Megan

Megan A. Cooper, MD, PhD
Assistant Professor, Department of Pediatrics
Division of Rheumatology
Washington University School of Medicine
Cooper_m at kids.wustl.edu
Lab website: http://research.peds.wustl.edu/Default.aspx?alias=research.peds.wustl.edu/Labs/Cooper_M
(lab office) 314-286-0262
(lab fax) 314-286-2895

-----Original Message-----
From: CIS-PIDD [mailto:cis-pidd at lists.clinimmsoc.org]
Sent: Thursday, February 04, 2016 8:27 AM
To: CIS-PIDD
Subject: Re: [cis-pidd] STAT3 GOF: other treatments than HSCT?

Thank you all for the great suggestions.
Joshua, please keep me updated on the trial.

Best wishes,
Ele
> On 04/feb/2016, at 14:30, CIS-PIDD <cis-pidd at lists.clinimmsoc.org> wrote:
>
> Dear Ele,
>
> We too have treated a patient with GOF-STAT3 with tocilizumab. She is 13yo with severe autoimmune hepatitis who prior to initiation was in fulminant liver failure. It has worked wonderfully in her, we are dosing every 4 weeks. Also in her case, serum IL-6 level was elevated at baseline, but this has not been the case in every patient treated with tocilizumab.
>
> We have also used rapamycin for treatment of lymphoproliferative disease in the mother of the patient with autoimmune hepatitis, not sure if lymphoproliferation is in your patient or not. It has worked well in decreasing her very significant lymphadenopathy and splenomegaly.
>
> Best of luck,
>
> Jen
>
> Jennifer Leiding, MD
> Assistant Professor
> Department of Pediatrics, Division of Allergy and Immunology
> University of South Florida All Children's Hospital St. Petersburg,
> Florida ________________________________________
> From: CIS-PIDD [cis-pidd at lists.clinimmsoc.org]
> Sent: Thursday, February 4, 2016 8:04 AM
> To: CIS-PIDD
> Subject: Re: [cis-pidd] STAT3 GOF: other treatments than HSCT?
>
> Dear Ele,
>
> I care for a young girl with STAT3 GOF with the same phenotype.  She required 24 hr BiPAP and her liver enzymes and GGT were quite elevated.  We decided to treat her with Tocilizumab, anti-IL-6, therapy starting in November.   She has had a good response for both liver and lung.  Her lungs responded quickly and she was able to come off Bipap during the day.  She now tolerates limited physical therapy with oxygen she is on Bipap during sleep.  After 3 doses, her liver enzymes and GGT have decreased by half.  Other colleagues have used it for liver disease and the effect has only been seen after the second dose.  Our patient's liver enzymes went up after the first dose, so we decreased the dose for the subsequent infusions.  She is tolerating it well.  We are compiling cases like this to write up the treatment experience, hopefully out this spring.  I am very happy to discuss this further.
>
> Best of Luck,
> Lisa
> Lisa Forbes, MD
> Assistant Professor, Department of Pediatrics Baylor College of
> Medicine Section of Immunology, Allergy, Rheumatology Texas Children's
> Hospital Texas Children's Hospital Center for Human Immunobiology,
> Medical Director
>
>
>
> 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: Thursday, February 4, 2016 at 6:45 AM
> To: CIS-PIDD
> <cis-pidd at lyris.dundee.net<mailto:cis-pidd at lyris.dundee.net>>
> Subject: [cis-pidd] STAT3 GOF: other treatments than HSCT?
>
> 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>
>
> ********************************************************************
>
>
> [cid:9760D3CB-437E-4668-9337-978E6C13F165 at station]
>
>
> ---
>
> You are currently subscribed to cis-pidd as: lisa.forbes at bcm.edu<mailto:lisa.forbes at bcm.edu>.
>
> To unsubscribe click here:
> http://cts.dundee.net/u?id=96396766.63dca3c8675cb6e4e57ac4d2f4bcc285&n
> =T&l=cis-pidd&o=3456160
>
> (It may be necessary to cut and paste the above URL if the line is
> broken)
>
> or send a blank email to
> leave-3456160-96396766.63dca3c8675cb6e4e57ac4d2f4bcc285 at lyris.dundee.n
> et<mailto:leave-3456160-96396766.63dca3c8675cb6e4e57ac4d2f4bcc285 at lyri
> s.dundee.net>
>
> ---
>
> You are currently subscribed to cis-pidd as: jleiding at health.usf.edu<mailto:jleiding at health.usf.edu>.
>
> To unsubscribe click here:
> http://cts.dundee.net/u?id=96396677.8032d543d21ea0e17bb1a686e8069e36&n
> =T&l=cis-pidd&o=3456205
>
> (It may be necessary to cut and paste the above URL if the line is
> broken)
>
> or send a blank email to
> leave-3456205-96396677.8032d543d21ea0e17bb1a686e8069e36 at lyris.dundee.n
> et<mailto:leave-3456205-96396677.8032d543d21ea0e17bb1a686e8069e36 at lyri
> s.dundee.net>
>
>
> ---
> You are currently subscribed to cis-pidd as: eleonora.gambineri at unifi.it.
> To unsubscribe click here:
> http://cts.dundee.net/u?id=96396511.a66ca48d1dc2d6c47043b86f63e2e761&n
> =T&l=cis-pidd&o=3456251 or send a blank email to
> leave-3456251-96396511.a66ca48d1dc2d6c47043b86f63e2e761 at lyris.dundee.n
> et<image001.jpg>

---
You are currently subscribed to cis-pidd as: cooper_m at kids.wustl.edu.
To unsubscribe click here: http://cts.dundee.net/u?id=96396471.9a016f51f09faba79ab9575dbdcfbf77&n=T&l=cis-pidd&o=3456364
or send a blank email to leave-3456364-96396471.9a016f51f09faba79ab9575dbdcfbf77 at lyris.dundee.net

The materials in this email are private and may contain Protected Health Information. If you are not the intended recipient, be advised that any unauthorized use, disclosure, copying, distribution or the taking of any action in reliance on the contents of this information is strictly prohibited. If you have received this email in error, please immediately notify the sender via telephone or return email.

---
You are currently subscribed to cis-pidd as: pagid at list.clinimmsoc.org.
To unsubscribe click here: http://cts.dundee.net/u?id=96396833.5a9591ccd1e327fe6bc4d1543298c482&n=T&l=cis-pidd&o=3456386
or send a blank email to leave-3456386-96396833.5a9591ccd1e327fe6bc4d1543298c482 at lyris.dundee.net