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Article

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

1
Department of Hematology, Medical University of Lodz, 93-510 Lodz, Poland
2
Copernicus Memorial Hospital, 93-510 Lodz, Poland
3
Department of Hematology, Transplantation and Internal Medicine, Medical University of Warsaw, 02-097 Warsaw, Poland
4
Institute of Hematology, University and Hospital of Perugia, 06132 Perugia, Italy
5
Laboratory of Molecular Biology, Clinical Immunotherapy Section, National Cancer Institute, National Institutes of Health, 9000 Rockville Pike, Bethesda, MD 20892, USA
*
Author to whom correspondence should be addressed.
These authors contributed equally.
Academic Editor: Francesco Albano
J. Clin. Med. 2021, 10(13), 2800; https://doi.org/10.3390/jcm10132800
Received: 29 May 2021 / Revised: 18 June 2021 / Accepted: 22 June 2021 / Published: 25 June 2021
(This article belongs to the Section Hematology)
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. View Full-Text
Keywords: BRAF; cladribine; dabrafenib; hairy cell leukemia; moxetumomab pasudotox; rituximab; vemurafenib BRAF; cladribine; dabrafenib; hairy cell leukemia; moxetumomab pasudotox; rituximab; vemurafenib
MDPI and ACS Style

Robak, T.; Janus, A.; Jamroziak, K.; Tiacci, E.; Kreitman, R.J. Vemurafenib and Rituximab in Patients with Hairy Cell Leukemia Previously Treated with Moxetumomab Pasudotox. J. Clin. Med. 2021, 10, 2800. https://doi.org/10.3390/jcm10132800

AMA Style

Robak T, Janus A, Jamroziak K, Tiacci E, Kreitman RJ. Vemurafenib and Rituximab in Patients with Hairy Cell Leukemia Previously Treated with Moxetumomab Pasudotox. Journal of Clinical Medicine. 2021; 10(13):2800. https://doi.org/10.3390/jcm10132800

Chicago/Turabian Style

Robak, Tadeusz, Agnieszka Janus, Krzysztof Jamroziak, Enrico Tiacci, and Robert J. Kreitman 2021. "Vemurafenib and Rituximab in Patients with Hairy Cell Leukemia Previously Treated with Moxetumomab Pasudotox" Journal of Clinical Medicine 10, no. 13: 2800. https://doi.org/10.3390/jcm10132800

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