Difference between revisions of "Erdheim-Chester disease"
Jump to navigation
Jump to search
Warner-admin (talk | contribs) m (Text replacement - "<!-- <!--Is there a regimen missing from this list? Would you like to share a different dosage/schedule or an additional reference for a regimen? Have you noticed an error? Do you have an idea that will help the site grow to better meet your needs and the needs of many others? You are invited to contribute to the site-->. -->" to "<!--Is there a regimen missing from this list? Would you like to share a different dosage/schedule or an additional reference for a regimen? Have you noticed an error? Do you have an idea that will help the site grow to better meet your needs and the needs of many others? You are invited to contribute to the site.-->") |
Warner-admin (talk | contribs) m (Text replacement - "?Variant |limit=10000|format=sum}} variants" to "?Variant |limit=10000|format=sum}} variants") |
||
| Line 6: | Line 6: | ||
|- | |- | ||
|<div style="background-color: #fee0d1; border: 1px solid #808000; padding: 5px; {{border-radius|16px}}" align="right"><font size="4"><b>{{#ask: [[-Has subobject::{{FULLPAGENAME}}]] |?Regimen |limit=10000|format=sum}} regimens on this page</b></font></div> | |<div style="background-color: #fee0d1; border: 1px solid #808000; padding: 5px; {{border-radius|16px}}" align="right"><font size="4"><b>{{#ask: [[-Has subobject::{{FULLPAGENAME}}]] |?Regimen |limit=10000|format=sum}} regimens on this page</b></font></div> | ||
| − | <div style="background-color: #deebf6; border: 1px solid #808000; padding: 5px; {{border-radius|16px}}"><font size="4"><b>{{#ask: [[-Has subobject::{{FULLPAGENAME}}]] |?Variant |limit=10000|format=sum}} variants on this page</b></font></div> | + | <div style="background-color: #deebf6; border: 1px solid #808000; padding: 5px; {{border-radius|16px}}"><font size="4"><b>{{#ask: [[-Has subobject::{{FULLPAGENAME}}]] |?Variant |limit=10000|format=sum}} [[Tutorial#Variants|variants]] on this page</b></font></div> |
|} | |} | ||
{{TOC limit|limit=3}} | {{TOC limit|limit=3}} | ||
Revision as of 16:15, 22 March 2018
3 regimens on this page
4 variants on this page
|
Guidelines
- 2014: Consensus guidelines for the diagnosis and clinical management of Erdheim-Chester disease PubMed
All lines of therapy
Sirolimus & Prednisone
| back to top |
Regimen
| Study | Evidence |
|---|---|
| Gianfreda et al. 2015 | Pilot, <20 pts in this arm |
Immunosuppressive therapy
- Sirolimus (Rapamune) 2 mg PO once per day, titrated to achieve a blood level of 8 to 12 ng/mL
- Prednisone (Sterapred) as follows:
- Month 1: 0.75 mg/kg/day PO
- Month 2: 0.5 mg/k/day PO
- Months 3 & 4: 0.25 mg/kg/day PO
- Months 5 & 6: 0.125 mg/kg/day PO
- Month 7 onwards: tapered to a dose of 2.5 to 5 mg PO once per day
Given for up to 2 years for responders and stable disease, with optional extension beyond 2 years
References
- Gianfreda D, Nicastro M, Galetti M, Alberici F, Corradi D, Becchi G, Baldari G, De Filippo M, Ferretti S, Moroni G, Foti R, Di Gangi M, Jeannin G, Saffroy R, Emile JF, Buzio C, Vaglio A. Sirolimus plus prednisone for Erdheim-Chester disease: an open-label trial. Blood. 2015 Sep 3;126(10):1163-71. Epub 2015 Jun 3. link to original article contains verified protocol PubMed
Vemurafenib monotherapy
| back to top |
Regimen #1
| Study | Evidence |
|---|---|
| Hyman et al. 2015 | Phase II, <20 pts in this arm |
| Diamond et al. 2017 (VE-BASKET) | Phase II |
These were basket trials, and all patients had BRAF V600 mutations. This is the FDA-recommended dose.
Chemotherapy
- Vemurafenib (Zelboraf) 960 mg PO BID
Duration of treatment not specified
Regimen #2
| Study | Evidence |
|---|---|
| Haroche et al. 2014 | Pilot, <20 pts |
Patients enrolled all had BRAF V600E mutation. This is the dose after de-escalation after the first four patients had excess adverse events.
Chemotherapy
- Vemurafenib (Zelboraf) 480 mg PO BID
Given until progression of disease or unacceptable toxicity
References
- Haroche J, Cohen-Aubart F, Emile JF, Maksud P, Drier A, Tolédano D, Barete S, Charlotte F, Cluzel P, Donadieu J, Benameur N, Grenier PA, Besnard S, Ory JP, Lifermann F, Idbaih A, Granel B, Graffin B, Hervier B, Arnaud L, Amoura Z. Reproducible and sustained efficacy of targeted therapy with vemurafenib in patients with BRAFV600E-mutated Erdheim-Chester disease. J Clin Oncol. 2015 Feb 10;33(5):411-8. Epub 2014 Nov 24. link to original article contains verified protocol PubMed
- Hyman DM, Puzanov I, Subbiah V, Faris JE, Chau I, Blay JY, Wolf J, Raje NS, Diamond EL, Hollebecque A, Gervais R, Elez-Fernandez ME, Italiano A, Hofheinz RD, Hidalgo M, Chan E, Schuler M, Lasserre SF, Makrutzki M, Sirzen F, Veronese ML, Tabernero J, Baselga J. Vemurafenib in multiple nonmelanoma cancers with BRAF V600 mutations. N Engl J Med. 2015 Aug 20;373(8):726-736. link to original article contains protocol link to PMC article PubMed
- VE-BASKET: Diamond EL, Subbiah V, Lockhart AC, Blay JY, Puzanov I, Chau I, Raje NS, Wolf J, Erinjeri JP, Torrisi J, Lacouture M, Elez E, Martínez-Valle F, Durham B, Arcila ME, Ulaner G, Abdel-Wahab O, Pitcher B, Makrutzki M, Riehl T, Baselga J, Hyman DM. Vemurafenib for BRAF V600-mutant Erdheim-Chester disease and Langerhans cell histiocytosis: analysis of data from the histology-independent, phase 2, open-label VE-BASKET Study. JAMA Oncol. 2017 Nov 29.[Epub ahead of print] link to original article contains verified protocol PubMed