Erdheim-Chester disease

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2 regimens on this page
3 variants on this page


All lines of therapy

Sirolimus & Prednisone

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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


  1. 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

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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.


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.


Given until progression of disease or unacceptable toxicity


  1. 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
  2. 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
  3. 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