Hairy Cell Leukemia
Associated Genetic Biomarkers
NCI Definition: A neoplasm of small B-lymphocytes with "hairy" projections in bone marrow, spleen, and peripheral blood. Most patients present with splenomegaly and pancytopenia. (WHO, 2001) 
Hairy cell leukemias most frequently harbor alterations in BRAF, WT1, WRN, U2AF1, and TRAF3 .
BRAF V600E, BRAF Mutation, BRAF Codon 600 Missense, WT1 P136S, and WT1 Mutation are the most common alterations in hairy cell leukemia .
BRAF is the most frequent predictive biomarker for hairy cell leukemia therapies.
Of the biomarker-directed therapies for hairy cell leukemia, 0 are FDA-approved in at least one setting and 1 has NCCN guidelines in at least one setting .
Disease is predicted to be sensitive: -
Sample must match all of the following:
|Clinical Setting(s): Refractory (NCCN), Relapse (NCCN)|
|Note: Hairy Cell Leukemia, a subtype of Mature B-Cell Lymphoma/Leukemia, harboring BRAF V600E mutation responded to vemurafenib in two patients who had refractory disease or were in relapse after conventional therapies.|
There are 8 clinical trials for hairy cell leukemia, of which 7 are open and 1 is completed or closed. Of the trials that contain hairy cell leukemia as an inclusion criterion, 2 are phase 1/phase 2 (1 open) and 6 are phase 2 (6 open).
BRAF and MYC are the most frequent gene inclusion criteria for hairy cell leukemia clinical trials .
Rituximab, lmb-2 immunotoxin, and autologous hematopoietic stem cell transplatation are the most common interventions in hairy cell leukemia clinical trials.
Significant Genes in Hairy Cell Leukemia
BRAF is mutated in 58.33% of hairy cell leukemia patients .
BRAF is an inclusion eligibility criterion in 3 clinical trials for hairy cell leukemia, of which 3 are open and 0 are closed. Of the trials that contain BRAF status and hairy cell leukemia as inclusion criteria, 3 are phase 2 (3 open) .
Vemurafenib is currently approved for use in patients with BRAF mutation in hairy cell leukemia .
Dabrafenib, trametinib, and vemurafenib are the most frequent therapies in hairy cell leukemia trials with BRAF mutations as inclusion criteria .
MYC is an inclusion eligibility criterion in 1 clinical trial for hairy cell leukemia, of which 1 is open and 0 are closed. Of the trial that contains MYC status and hairy cell leukemia as inclusion criteria, 1 is phase 1/phase 2 (1 open) .
Autologous hematopoietic stem cell transplatation, carmustine, and cyclophosphamide are the most frequent therapies in hairy cell leukemia trials with MYC mutations as inclusion criteria .
2. The AACR Project GENIE Consortium. AACR Project GENIE: powering precision medicine through an international consortium. Cancer Discovery. 2017;7(8):818-831. Dataset Version 4. This dataset does not represent the totality of the genetic landscape; see paper for more information.