Difference between revisions of "Erdheim-Chester disease"
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{{TOC limit|limit=3}} | {{TOC limit|limit=3}} | ||
=Guidelines= | =Guidelines= | ||
− | '''Given the rapid change in evidence in many areas of hematology/oncology, readers are encouraged to consider any | + | '''Given the rapid change in evidence in many areas of hematology/oncology, readers are encouraged to consider any guideline published 5+ years ago to be for historical purposes, only.''' |
==Consensus guidelines== | ==Consensus guidelines== | ||
*'''2020:''' Goyal et al. [https://doi.org/10.1182/blood.2019003507 Erdheim-Chester Disease: Consensus Recommendations for Evaluation, Diagnosis, and Treatment in the Molecular Era] [https://pubmed.ncbi.nlm.nih.gov/32187362/ PubMed] | *'''2020:''' Goyal et al. [https://doi.org/10.1182/blood.2019003507 Erdheim-Chester Disease: Consensus Recommendations for Evaluation, Diagnosis, and Treatment in the Molecular Era] [https://pubmed.ncbi.nlm.nih.gov/32187362/ PubMed] | ||
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*'''2014:''' Diamond et al. [https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4110656/ Consensus guidelines for the diagnosis and clinical management of Erdheim-Chester disease] [https://pubmed.ncbi.nlm.nih.gov/24850756/ PubMed] | *'''2014:''' Diamond et al. [https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4110656/ Consensus guidelines for the diagnosis and clinical management of Erdheim-Chester disease] [https://pubmed.ncbi.nlm.nih.gov/24850756/ PubMed] | ||
==[https://www.nccn.org NCCN]== | ==[https://www.nccn.org NCCN]== | ||
− | *''NCCN does not have guidelines at this granular level; please see [https://www.nccn.org/ | + | *''NCCN does not currently have guidelines at this granular level; please see [https://www.nccn.org/guidelines/guidelines-detail?category=1&id=1502 NCCN Guidelines - Histiocytic Neoplasms].'' |
=All lines of therapy= | =All lines of therapy= | ||
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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 dosing details in manuscript''' [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 dosing details in manuscript''' [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://doi.org/10.1056/NEJMoa1502309 link to original article] '''contains dosing details in abstract''' [https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4971773/ link to PMC article] [https://pubmed.ncbi.nlm.nih.gov/26287849/ PubMed] [https://clinicaltrials.gov/study/NCT01524978 NCT01524978] | # '''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://doi.org/10.1056/NEJMoa1502309 link to original article] '''contains dosing details in abstract''' [https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4971773/ link to PMC article] [https://pubmed.ncbi.nlm.nih.gov/26287849/ PubMed] [https://clinicaltrials.gov/study/NCT01524978 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 dosing details in manuscript''' [ | + | ## '''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 dosing details in manuscript''' [https://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 12:10, 23 June 2024
Section editor | |
---|---|
Gaurav Goyal, MD UAB Birmingham, AL, USA |
3 regimens on this page
4 variants on this page
|
Guidelines
Given the rapid change in evidence in many areas of hematology/oncology, readers are encouraged to consider any guideline published 5+ years ago to be for historical purposes, only.
Consensus guidelines
- 2020: Goyal et al. Erdheim-Chester Disease: Consensus Recommendations for Evaluation, Diagnosis, and Treatment in the Molecular Era PubMed
- 2014: Diamond et al. Consensus guidelines for the diagnosis and clinical management of Erdheim-Chester disease PubMed
NCCN
- NCCN does not currently have guidelines at this granular level; please see NCCN Guidelines - Histiocytic Neoplasms.
All lines of therapy
Cobimetinib monotherapy
Regimen
FDA-recommended dose |
Study | Dates of enrollment | Evidence |
---|---|---|
Diamond et al. 2019 (MSK 15-216) | 2016-NR | Phase 2, fewer than 20 pts in this arm (RT) |
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 dosing details in manuscript link to PMC article PubMed NCT02649972
Sirolimus & Prednisone
Regimen
Study | Evidence |
---|---|
Gianfreda et al. 2015 | Pilot, fewer than 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/kg/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 dosing details in manuscript PubMed
Vemurafenib monotherapy
Regimen variant #1, 480 mg twice per day
Study | Evidence |
---|---|
Haroche et al. 2014 | Pilot, fewer than 20 pts |
Note: This is the dose after de-escalation after the first four patients had excess adverse events.
Biomarker eligibility criteria
- BRAF p.V600E
Regimen variant #2, 960 mg twice per day
FDA-recommended dose |
Study | Dates of enrollment | Evidence |
---|---|---|
Hyman et al. 2015 (VE-BASKET) | 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
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 dosing details in manuscript 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 dosing details in abstract 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 dosing details in manuscript link to PMC article PubMed