Low‐dose vemurafenib in hairy cell leukemia patients with active infection

Bohn, Jan‐Paul; Pircher, Andreas; Wanner, David; Vill, David; Foeger, Bernhard; Wolf, Dominik; Steurer, Michael

doi:10.1002/ajh.25474

Cited by 33 publications

(28 citation statements)

References 5 publications

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“…More recently, vemurafenib, dabrafenib, trametinib and ibrutinib are currently under investigation in patients unsuitable for PNAs [13,[24][25][26][27][28][29]. These agents have been proposed as relatively safe drugs, even in pretreated patients, with severe neutropenia and (in the case of BRAF inhibitors) infections.…”

Section: Discussionmentioning

confidence: 99%

Vemurafenib and Rituximab in Patients with Hairy Cell Leukemia Previously Treated with Moxetumomab Pasudotox

Robak

Janus

Jamroziak

et al. 2021

JCM

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The purine nucleoside analogues cladribine and pentostatin are highly-active first-line therapeutic treatments for hairy cell leukemia (HCL), resulting in complete response rates of 80% to 90%. However, HCL patients continue to relapse, and sooner or later, most require subsequent lines of treatment. This report presents the cases of four relapsed patients with classic HCL who were treated with vemurafenib (mostly at the low dose of 240 mg twice daily for 16 weeks) combined with rituximab after the failure of several lines of therapy including cladribine with or without rituximab and moxetumomab pasudotox. Two patients achieved minimal residual disease negative complete response after combined treatment with vemurafenib and rituximab, with a hematologic response ongoing after 38 months from the end of treatment in one patient and a relapse of cytopenias occurring after 13 months in the other patient. A third patient normalized her blood counts and this hematologic response, which was not evaluated in the bone marrow at the end of treatment, was lost after 18 months. The last patient died due to infection and multi-organ failure, too early to verify response to vemurafenib. Two patients who had relapsed after vemurafenib and rituximab derived meaningful clinical benefit from retreatment with the same agents, but eventually relapsed again and started indefinite therapy with dabrafenib and trametinib leading to normalization of the blood counts (despite heavy bone marrow infiltration in the only patient so far evaluable in that regard). The outcomes of these cases indicate that novel targeted agents and, in particular, vemurafenib, combined with rituximab, improve the prognosis of HCL patients, even those heavily pretreated with PNAs and moxetumomab pasudotox.

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Section: Discussionmentioning

confidence: 99%

Vemurafenib and Rituximab in Patients with Hairy Cell Leukemia Previously Treated with Moxetumomab Pasudotox

Robak

Janus

Jamroziak

et al. 2021

JCM

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“…The fourth patient, who had received 7 lines of therapy over 15 years, was not evaluable as he died too early (3 weeks after starting vemurafenib plus rituximab) of a severe infection caused by a pre-existing deep neutropenia, which even such a high effective treatment was not expected to necessarily resolve earlier (indeed, the median time to resolution of neutropenia after vemurafenib plus rituximab is 4 weeks [17]). Nevertheless, previous reports based on small numbers of cases suggested that vemurafenib is a safe and effective drug even in patients with HCL presenting with severe neutropenia, fever and infection [26,27,30].…”

Section: Discussionmentioning

confidence: 99%

Vemurafenib and Rituximab in Patients with Hairy Cell Leukemia Previously Treated with Moxetumomab Pasudotox

Robak

Janus

Jamroziak

et al. 2021

Preprint

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The purine nucleoside analogues cladribine and pentostatin are highly-active first-line therapeutic treatments for hairy cell leukemia (HCL), resulting in complete response rates of 80 to 90%. However, HCL patients continue to relapse, and sooner or later, most require subsequent lines of treatment. This report presents the relapsed patients with classic HCL who were treated with vemurafenib (mostly at the low dose of 240 mg twice daily for 16 weeks) combined with rituximab after the failure of several lines of therapy including cladribine with or without rituximab and moxetumomab pasudotox. Two patients achieved CR MRD (-) after combined treatment with vemurafenib and rituximab, with a hematologic response ongoing after 38 months from the end of treatment in one patient and a relapse of cytopenias occurring after 13 months in the other patient. A third patient normalized her blood counts and this hematologic response, which was not evaluated in the bone marrow at the end of treatment, was lost after 18 months. The last patient died due to infection and multi-organ failure, too early to verify response to vemurafenib. Two patients who had relapsed after vemurafenib and rituximab derived meaningful clinical benefit from retreatment with the same agents, but eventually relapsed again and started indefinite therapy with dabrafenib and trametinib with normalization of the blood counts despite heavy bone marrow infiltration in the only patient so far evaluable. The outcomes of these cases indicate that novel targeted agents, and in particular vemurafenib combined with rituximab, improve the prognosis of HCL patients, even those heavily pretreated with PNAs and moxetumomab pasudotox

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“…Recently, anecdotal reports of inhibitors of BRAF V600E (e.g. vemurafenib) have been utilised as a bridge to improve blood counts and control infection in patients with HCLc 17‐19 . Vemurafenib is approved by the FDA in the United States for the treatment of patients with malignant melanoma, but is ‘off‐label’ for use in HCL.…”

Section: A North American Viewpoint (Mike Grever)mentioning

confidence: 99%

The revised guidelines for the diagnosis and management of hairy cell leukaemia and the hairy cell leukaemia variant

Troussard

Grever

2020

Br J Haematol

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Low‐dose vemurafenib in hairy cell leukemia patients with active infection

Cited by 33 publications

References 5 publications

Vemurafenib and Rituximab in Patients with Hairy Cell Leukemia Previously Treated with Moxetumomab Pasudotox

Vemurafenib and Rituximab in Patients with Hairy Cell Leukemia Previously Treated with Moxetumomab Pasudotox

Vemurafenib and Rituximab in Patients with Hairy Cell Leukemia Previously Treated with Moxetumomab Pasudotox

The revised guidelines for the diagnosis and management of hairy cell leukaemia and the hairy cell leukaemia variant

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