Sorafenib for Chester-Erdheim – pro
This is a disease in which targeted therpay is consdiered acceptable adn recommended. The 202 guideleins referenced below makes teh following recommendaitons:
For patients with BRAF-V600E ECD who have cardiac/neurologic disease or end-organ dysfunction, BRAF-inhibitor therapy with vemurafenib or dabrafenib should be implemented as first-line therapy A
For BRAF-V600E ECD without end-organ dysfunction, BRAF-inhibitors or conventional therapy may both be considered for first-line therapy based on toxicity profile and drug availability/experience of clinician A
For ECD patients without BRAF-V600E and cardiac/neurologic disease or end-organ dysfunction, empiric treatment with MEK-inhibitor should be strongly considered as first-line therapy A
Optimal duration and dosing of targeted therapies is not known, although relapse has been observed in the majority of cases following complete cessation of BRAF-inhibitors; maintenance treatment in the setting of metabolic remission with low-dose therapy as tolerated may be considered A
For patients without access to targeted therapies and high-burden disease, IFN-/PEG–IFN- or cladribine therapy may be considered ”
Although teh FLT-3 target is not lsited, the guidelines recommend targeted therapy and Smith et al justify the use of sorafenib using the same approach, especially sine it had been effective.
The use of sorafenib has been determined to
be not medically necessary because the use of sorafenib for Erdheim-Chester disease is
considered Experimental/Investigational.
Gaurav Goyal, Mark L. Heaney, Matthew Collin, Fleur Cohen-Aubart, Augusto Vaglio, Benjamin H. Durham, Oshrat Hershkovitz-Rokah, Michael Girschikofsky, Eric D. Jacobsen, Kazuhiro Toyama, Aaron M. Goodman, Paul Hendrie, Xin-xin Cao, Juvianee I. Estrada-Veras, Ofer Shpilberg, André Abdo, Mineo Kurokawa, Lorenzo Dagna, Kenneth L. McClain, Roei D. Mazor, Jennifer Picarsic, Filip Janku, Ronald S. Go, Julien Haroche, Eli L. Diamond, Erdheim-Chester disease: consensus recommendations for evaluation, diagnosis, and treatment in the molecular era. Blood (2020) 135 (22): 1929–1945.
hao AK, Meyer JA, Lee AG, Hecht A, Tarver T, Van Ziffle J, Koegel AK, Golden C, Braun BS, Sweet-Cordero EA, Smith CC, Dvorak CC, Loh ML, Stieglitz E. Fusion driven JMML: a novel CCDC88C-FLT3 fusion responsive to sorafenib identified by RNA sequencing. Leukemia. 2020 Feb;34(2):662-666.
Katherine Smith1, Patrick McGarrah1, Karen Rech2, Aishwarya Ravindran2, Jason Young3, Wasantha Ranatunga4, Julio Sartori-Valinotti5, Gaurav Goyal6, Ronald Go1, Jithma Abeykoon, PERSONALIZED MEDICINE IN ERDHEIM-CHESTER DISEASE WITHOUT MAPK PATHWAY ALTERATIONS, https://onlinelibrary.wiley.com/doi/10.1002/pbc.30097, Pediatric Blood & CancerVolume 70, Issue S1 e30097, 2022