Vemurafenib in the Treatment of Erdheim Chester Disease: A Systematic Review.

Authors :: Aziz SN
Proano L
Cruz C
Tenemaza MG
Monteros G
Hassen G
Baskar A
Argudo JM
Duenas JB
Fabara SP
Source :: Cureus [Cureus] 2022 Jun 14; Vol. 14 (6), pp. e25935. Date of Electronic Publication: 2022 Jun 14 (Print Publication: 2022).
Publication Year :: 2022
Abstract: Erdheim Chester disease (ECD) is a type of histiocytosis characterized by a variable clinical presentation. The treatment of ECD is complex and mainly unknown. We aim to conduct a literature review of the treatment of ECD and consolidate the knowledge about the most recent and updated treatment for ECD. To conduct the systematic review, we used the preferred reporting items for systematic reviews and meta-analysis (PRISMA) protocol.  To analyze the bias, we used the Cochrane collaboration risk-of-bias tool to assess the bias. We included observational studies and clinical trials on humans, which were written in English. Papers not fulfilling the objective of our study were excluded. Overall, the drug showed efficacy in the clinical trials, showing prolonged improvement and high rates of response rate. Overall, the drug was not well tolerated, and patients had a long list of side effects. Nevertheless, the drug seems to be a good option for second-line treatment for patients with ECD and BRAFV600 mutation.<br />Competing Interests: The authors have declared that no competing interests exist.<br /> (Copyright © 2022, Aziz et al.)