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HEALTH PROFESSIONAL · SOURCE READING

BRAF inhibitors (vemurafenib or dabrafenib) with or without rituximab or trametinib

Source: Hairy Cell Leukemia Treatment (PDQ®)–Health Professional Version, National Cancer Institute.

Source updated: February 21, 2025 · Captured 2026-09-09.

Selected source text with whitespace normalised. This Triangle page is not an NCI PDQ summary.

Context: Treatment of Hairy Cell Leukemia / Treatment Options for Hairy Cell Leukemia

BRAF V600E pathogenic variants are found in almost 100% of patients with classic-form hairy cell leukemia and almost never found in patients with other B-cell lymphomas and leukemias, including HCL-v.[20][Level of evidence C3] Vemurafenib or other BRAF inhibitors such as dabrafenib can be given with rituximab or obinutuzumab.[10,21] The FDA has not approved BRAF inhibitors for hairy cell leukemia, but they can be used off-label in clinical practice.[22]

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Evidence (vemurafenib with or without rituximab):

Several multicenter studies evaluated vemurafenib, given orally alone for 4 months or orally for 2 months with rituximab infused in eight doses over 18 weeks, in patients with relapsed or refractory hairy cell leukemia.[7,8,10,23][Level of evidence C3]

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Several multicenter studies evaluated vemurafenib, given orally alone for 4 months or orally for 2 months with rituximab infused in eight doses over 18 weeks, in patients with relapsed or refractory hairy cell leukemia.[7,8,10,23][Level of evidence C3]

After a median follow-up of 23 to 40 months, for the 86 patients treated with vemurafenib alone, the following was reported in two studies:[7,8,23][Level of evidence C3]

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Several multicenter studies evaluated vemurafenib, given orally alone for 4 months or orally for 2 months with rituximab infused in eight doses over 18 weeks, in patients with relapsed or refractory hairy cell leukemia.[7,8,10,23][Level of evidence C3] › After a median follow-up of 23 to 40 months, for the 86 patients treated with vemurafenib alone, the following was reported in two studies:[7,8,23][Level of evidence C3]

The overall response rate was 86% to 98%.

Several multicenter studies evaluated vemurafenib, given orally alone for 4 months or orally for 2 months with rituximab infused in eight doses over 18 weeks, in patients with relapsed or refractory hairy cell leukemia.[7,8,10,23][Level of evidence C3] › After a median follow-up of 23 to 40 months, for the 86 patients treated with vemurafenib alone, the following was reported in two studies:[7,8,23][Level of evidence C3]

The complete response rate was 33% to 38%.

Several multicenter studies evaluated vemurafenib, given orally alone for 4 months or orally for 2 months with rituximab infused in eight doses over 18 weeks, in patients with relapsed or refractory hairy cell leukemia.[7,8,10,23][Level of evidence C3] › After a median follow-up of 23 to 40 months, for the 86 patients treated with vemurafenib alone, the following was reported in two studies:[7,8,23][Level of evidence C3]

The median treatment-free survival was 18 to 25 months.

Several multicenter studies evaluated vemurafenib, given orally alone for 4 months or orally for 2 months with rituximab infused in eight doses over 18 weeks, in patients with relapsed or refractory hairy cell leukemia.[7,8,10,23][Level of evidence C3] › After a median follow-up of 23 to 40 months, for the 86 patients treated with vemurafenib alone, the following was reported in two studies:[7,8,23][Level of evidence C3]

Retreatment at relapse resulted in an 86% response rate, and the median relapse-free survival was 12.7 months in one of the trials, with a 40-month median follow-up.[23][Level of evidence C2]

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Several multicenter studies evaluated vemurafenib, given orally alone for 4 months or orally for 2 months with rituximab infused in eight doses over 18 weeks, in patients with relapsed or refractory hairy cell leukemia.[7,8,10,23][Level of evidence C3]

After a median follow-up of 37 months, for the 30 patients treated with vemurafenib plus rituximab, the following was reported:[10][Level of evidence C3]

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Several multicenter studies evaluated vemurafenib, given orally alone for 4 months or orally for 2 months with rituximab infused in eight doses over 18 weeks, in patients with relapsed or refractory hairy cell leukemia.[7,8,10,23][Level of evidence C3] › After a median follow-up of 37 months, for the 30 patients treated with vemurafenib plus rituximab, the following was reported:[10][Level of evidence C3]

The complete response rate was 87%.

Several multicenter studies evaluated vemurafenib, given orally alone for 4 months or orally for 2 months with rituximab infused in eight doses over 18 weeks, in patients with relapsed or refractory hairy cell leukemia.[7,8,10,23][Level of evidence C3] › After a median follow-up of 37 months, for the 30 patients treated with vemurafenib plus rituximab, the following was reported:[10][Level of evidence C3]

The PFS rate was 78% at 37 months.

Several multicenter studies evaluated vemurafenib, given orally alone for 4 months or orally for 2 months with rituximab infused in eight doses over 18 weeks, in patients with relapsed or refractory hairy cell leukemia.[7,8,10,23][Level of evidence C3] › After a median follow-up of 37 months, for the 30 patients treated with vemurafenib plus rituximab, the following was reported:[10][Level of evidence C3]

In patients who had a complete response, 65% had no MRD.

Evidence (dabrafenib plus trametinib):

In a phase II trial of patients with relapsed or refractory disease, 55 patients received dabrafenib and trametinib orally until their disease progressed, they experienced unacceptable toxicity, or death occurred.[24]

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In a phase II trial of patients with relapsed or refractory disease, 55 patients received dabrafenib and trametinib orally until their disease progressed, they experienced unacceptable toxicity, or death occurred.[24]

With a median follow-up of 43.2 months, the overall response rate was 89.0% (95% CI, 77.8%–95.9%), the complete response rate was 65.5%, the 2-year PFS rate was 94.5%, and the 2-year OS rate was 95.5%.[24][Level of evidence C1]

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Preserved source evidence · Not medical advice