Improving treatment for hairy cell leukemia (Vemurafenib)

Medical Xpress / Northwestern University / Blood ^ | Jan. 13, 2023 | Melissa Rohman / Shivani Handa et al

[ConservativeMind]

Patients with relapsed or refractory hairy cell leukemia who were treated with vemurafenib experienced excellent response and relapse-free survival, according to a trial.

"This finding is important because it represents a targeted and relatively nontoxic and nonchemotherapeutic treatment," said Martin S. Tallman, MD,

Hairy cell leukemia (HCL), a rare type of leukemia, manifests when bone marrow produces too many lymphocytes, a type of white blood cell. The disease can be treated with the chemotherapy drug Cladribine either with or without the anti-CD20 monoclonal antibody Rituximab. Unfortunately, 30 to 40 percent of patients will still relapse.

In the case of relapsed or refractory HCL, patients are prescribed vemurafenib, a small molecular inhibitor drug.

In the current clinical trial, investigators measured patient outcomes of 36 individuals with relapsed or refractory HCL who were treated with vemurafenib at study sites located across the U.S.

Of these patients, the investigators found that 33 percent had a complete response to the drug and 53 percent of patients had a partial response.

A 40-month follow-up evaluation revealed that 68 percent of patients experienced relapse, with an average relapse-free survival rate of 19 months. Of the 21 patients who relapsed, 14 were re-treated with vemurafenib and of these patients, 86 percent had their white blood cell levels return to normal.

Overall, patient survival was 82 percent at four years. Additionally, increasing drug dosage or extending treatment duration did not improve the overall treatment response.

The investigators noted that the duration of remission was shorter with each subsequent relapse and suggest that combining vemurafenib with monoclonal antibodies may shorten treatment duration and prolong remission.

The findings suggest vemurafenib monotherapy can achieve high response rates in patients with relapsed or refractory HCL with acceptable side effects, according to Tallman.

[ConservativeMind]

Overall survival for those using it was 82% at the four year mark.

[ConservativeMind]

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