Difference between revisions of "Erdheim-Chester disease"
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# 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. [https://doi.org/10.1200/jco.2014.57.1950 link to original article] '''contains verified protocol''' [https://pubmed.ncbi.nlm.nih.gov/25422482 PubMed] | # 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. [https://doi.org/10.1200/jco.2014.57.1950 link to original article] '''contains verified protocol''' [https://pubmed.ncbi.nlm.nih.gov/25422482 PubMed] | ||
− | # '''VE-BASKET:''' 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. [https://www.nejm.org/doi/full/10.1056/NEJMoa1502309 link to original article] '''contains protocol''' [https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4971773/ link to PMC article] [https://pubmed.ncbi.nlm.nih.gov/26287849 PubMed] | + | # '''VE-BASKET:''' 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. [https://www.nejm.org/doi/full/10.1056/NEJMoa1502309 link to original article] '''contains protocol''' [https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4971773/ link to PMC article] [https://pubmed.ncbi.nlm.nih.gov/26287849 PubMed] NCT01524978 |
− | # ''' | + | ## '''Update:''' 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. 2018 Mar 1;4(3):384-388. Epub 2017 Nov 29. [https://jamanetwork.com/journals/jamaoncology/fullarticle/2664827 link to original article] '''contains verified protocol''' [http://www.ncbi.nlm.nih.gov/pmc/articles/pmc5844839/ link to PMC article] [https://pubmed.ncbi.nlm.nih.gov/29188284 PubMed] |
[[Category:Erdheim-Chester disease regimens]] | [[Category:Erdheim-Chester disease regimens]] | ||
[[Category:Disease-specific pages]] | [[Category:Disease-specific pages]] | ||
[[Category:Histiocytoses]] | [[Category:Histiocytoses]] |
Revision as of 12:42, 6 August 2022
Section editor | |
---|---|
Gaurav Goyal, MD UAB Birmingham, AL GauravGoyalMD |
3 regimens on this page
4 variants on this page
|
Guidelines
Consensus guidelines
- 2020: Goyal et al. Erdheim-Chester Disease: Consensus Recommendations for Evaluation, Diagnosis, and Treatment in the Molecular Era PubMed
Older
- 2014: Diamond et al. Consensus guidelines for the diagnosis and clinical management of Erdheim-Chester disease PubMed
NCCN
All lines of therapy
Cobimetinib monotherapy
Regimen
Study | Evidence |
---|---|
Diamond et al. 2019 (MSK 15-216) | Pilot, <20 pts in this arm |
Targeted therapy
- Cobimetinib (Cotellic) 60 mg PO once per day on days 1 to 21
28-day cycles
References
- MSK 15-216: Diamond EL, Durham BH, Ulaner GA, Drill E, Buthorn J, Ki M, Bitner L, Cho H, Young RJ, Francis JH, Rampal R, Lacouture M, Brody LA, Ozkaya N, Dogan A, Rosen N, Iasonos A, Abdel-Wahab O, Hyman DM. Efficacy of MEK inhibition in patients with histiocytic neoplasms. Nature. 2019 Mar;567(7749):521-524. Epub 2019 Mar 13. link to original article contains verified protocol link to PMC article PubMed
Sirolimus & Prednisone
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:
- Cycle 1: 0.75 mg/kg/day PO
- Cycle 2: 0.5 mg/k/day PO
- Cycles 3 & 4: 0.25 mg/kg/day PO
- Cycles 5 & 6: 0.125 mg/kg/day PO
- Cycle 7 onwards: tapered to a dose of 2.5 to 5 mg PO once per day
Monthly cycle for up to 24 cycles (2 years), 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
Regimen variant #1, 480 mg twice per day
Study | Evidence |
---|---|
Haroche et al. 2014 | Pilot, <20 pts |
Note: This is the dose after de-escalation after the first four patients had excess adverse events.
Biomarker eligibility crtieria
- BRAF p.V600E
Targeted therapy
- Vemurafenib (Zelboraf) 480 mg PO twice per day
Continued indefinitely
Regimen variant #2, 960 mg twice per day
FDA-recommended dose |
Study | Years of enrollment | Evidence |
---|---|---|
Hyman et al. 2015 (VE-BASKET) | 2012-2014 | Phase 2, <20 pts in this arm |
Diamond et al. 2017 (VE-BASKET ECD expansion) | 2012-2014 | Phase 2 (RT) |
Note: This was a basket study that was expanded in the ECD population, as described by Diamond et al. 2017.
Biomarker eligibility criteria
- BRAF p.V600 alterations
Targeted therapy
- Vemurafenib (Zelboraf) 960 mg PO twice per day
Duration of treatment not specified
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
- VE-BASKET: 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 NCT01524978
- Update: 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. 2018 Mar 1;4(3):384-388. Epub 2017 Nov 29. link to original article contains verified protocol link to PMC article PubMed