Vol 53, No 1 (2022)
Review article
Published online: 2021-10-05
New therapeutic options for hairy cell leukemia
1, 1, 12
Acta Haematol Pol 2022;53(1):39-47.
Abstract
Hairy cell leukemia (HCL) is a rare B-cell lymphoproliferative disorder characterized by pancytopenia, splenomegaly and increased susceptibility to infections. In 2011, BRAF gene mutation was identified in almost all the patients with the classical type of HCL. The purine analogs cladribine and pentostatin induce long-term remission in the majority of patients, and they remain the standard treatment for this type of leukemia.
However, more than half of patients in complete response relapse over the long term, with a quarter of them relapsing within the first five years.
Recently, new drugs have been developed and have demonstrated efficacy in refractory or relapsed HCL. The immunotoxin Moxetumomab pasudotox was registered for HCL in 2018. The BRAF kinase inhibitors vemurafenib and dabrafenib, as well as the Bruton kinase inhibitor ibrutinib, are also proven highly effective in clinical trials.
Keywords: BRAFdabrafenibhairy cell leukemiaibrutinibmoxetumomab pasudotoxpurine analogsrituximabvemurafenib
References
- Grever MR, Abdel-Wahab O, Andritsos LA, et al. Consensus guidelines for the diagnosis and management of patients with classic hairy cell leukemia. Blood. 2017; 129(5): 553–560.
- Robak T, Matutes E, Catovsky D, et al. ESMO Guidelines Committee. Hairy cell leukaemia: ESMO Clinical Practice Guidelines for diagnosis, treatment and follow-up. Ann Oncol. 2015; 26 Suppl 5: v100–v107.
- Robak P, Jesionek-Kupnicka D, Kupnicki P, et al. Bone lesions in hairy cell leukemia: diagnosis and treatment. Eur J Haematol. 2020; 105(6): 682–691.
- Tadmor T, Polliack A. Hairy cell leukemia: uncommon clinical features, unusual sites of involvement and some rare associations. Best Pract Res Clin Haematol. 2015; 28(4): 193–199.
- Robak E, Jesionek-Kupnicka D, Robak T. Skin changes in hairy cell leukemia. Ann Hematol. 2021; 100(3): 615–625.
- Tiacci E, Trifonov V, Schiavoni G, et al. BRAF mutations in hairy-cell leukemia. N Engl J Med. 2011; 364(24): 2305–2315.
- Teras LR, DeSantis CE, Cerhan JR, et al. 2016 US lymphoid malignancy statistics by World Health Organization subtypes. CA Cancer J Clin. 2016; 66(6): 443–459.
- Morton LM, Wang SS, Devesa SS, et al. Lymphoma incidence patterns by WHO subtype in the United States, 1992-2001. Blood. 2006; 107(1): 265–276.
- Matutes E. Diagnostic and therapeutic challenges in hairy cell leukemia-variant: where are we in 2021? Expert Rev Hematol. 2021; 14(4): 355–363.
- Swerdlow SH, Campo E, Pileri SA, et al. The 2016 revision of the World Health Organization classification of lymphoid neoplasms. Blood. 2016; 127(20): 2375–2390.
- Robak T. Hairy-cell leukemia variant: recent view on diagnosis, biology and treatment. Cancer Treat Rev. 2011; 37(1): 3–10.
- Paillassa J, Cornet E, Noel S, et al. Analysis of a cohort of 279 patients with hairy-cell leukemia (HCL): 10 years of follow-up. Blood Cancer J. 2020; 10(5): 62.
- Saven A, Burian C, Koziol JA, et al. Long-term follow-up of patients with hairy cell leukemia after cladribine treatment. Blood. 1998; 92(6): 1918–1926.
- Puła A, Robak T. Hairy cell leukemia: a brief update on current knowledge and treatment prospects. Curr Opin Oncol. 2021; 33(5): 412–419.
- Falini B, Tiacci E. New treatment options in hairy cell leukemia with focus on BRAF inhibitors. Hematol Oncol. 2019; 37 Suppl 1: 30–37.
- Cassileth PA, Cheuvart B, Spiers AS, et al. Pentostatin induces durable remissions in hairy cell leukemia. J Clin Oncol. 1991; 9(2): 243–246.
- Else M, Dearden CE, Catovsky D. Long-term follow-up after purine analogue therapy in hairy cell leukaemia. Best Pract Res Clin Haematol. 2015; 28(4): 217–229.
- Ramos Perez J, Ravandi-Kashani F. The pharmacological management of hairy cell leukemia. Expert Opin Pharmacother. 2020; 21(11): 1337–1344.
- Flinn IW, Kopecky KJ, Foucar MK, et al. Long-term follow-up of remission duration, mortality, and second malignancies in hairy cell leukemia patients treated with pentostatin. Blood. 2000; 96(9): 2981–2986.
- Goodman GR, Burian C, Koziol JA, et al. Extended follow-up of patients with hairy cell leukemia after treatment with cladribine. J Clin Oncol. 2003; 21(5): 891–896.
- Zinzani PL, Pellegrini C, Stefoni V, et al. Hairy cell leukemia: evaluation of the long-term outcome in 121 patients. Cancer. 2010; 116(20): 4788–4792.
- Else M, Dearden CE, Matutes E, et al. Long-term follow-up of 233 patients with hairy cell leukaemia, treated initially with pentostatin or cladribine, at a median of 16 years from diagnosis. Br J Haematol. 2009; 145(6): 733–740.
- Robak T, Lech-Maranda E, Korycka A, et al. Purine nucleoside analogs as immunosuppressive and antineoplastic agents: mechanism of action and clinical activity. Curr Med Chem. 2006; 13(26): 3165–3189.
- Nieva J, Bethel K, Saven A. Phase 2 study of rituximab in the treatment of cladribine-failed patients with hairy cell leukemia. Blood. 2003; 102(3): 810–813.
- Chihara D, Kantarjian H, O'Brien S, et al. Long-term durable remission by cladribine followed by rituximab in patients with hairy cell leukaemia: update of a phase II trial. Br J Haematol. 2016; 174(5): 760–766.
- Chihara D, Arons E, Stetler-Stevenson M, et al. Randomized phase II study of first-line cladribine with concurrent or delayed rituximab in patients with hairy cell leukemia. J Clin Oncol. 2020; 38(14): 1527–1538.
- Gerrie AS, Zypchen LN, Connors JM. Fludarabine and rituximab for relapsed or refractory hairy cell leukemia. Blood. 2012; 119(9): 1988–1991.
- Burotto M, Stetler-Stevenson M, Arons E, et al. Bendamustine and rituximab in relapsed and refractory hairy cell leukemia. Clin Cancer Res. 2013; 19(22): 6313–6321.
- Parry-Jones N, Joshi A, Forconi F, et al. BSH guidelines committee. Guideline for diagnosis and management of hairy cell leukaemia (HCL) and hairy cell variant (HCL-V). Br J Haematol. 2020; 191(5): 730–737.
- Kreitman RJ, Tallman MS, Robak T, et al. Phase I trial of anti-CD22 recombinant immunotoxin moxetumomab pasudotox (CAT-8015 or HA22) in patients with hairy cell leukemia. J Clin Oncol. 2012; 30(15): 1822–1828.
- Kreitman RJ, Tallman MS, Robak T, et al. Minimal residual hairy cell leukemia eradication with moxetumomab pasudotox: phase 1 results and long-term follow-up. Blood. 2018; 131(21): 2331–2334.
- Kreitman RJ, Dearden C, Zinzani PL, et al. Study 1053 investigators. Moxetumomab pasudotox in relapsed/refractory hairy cell leukemia. Leukemia. 2018; 32(8): 1768–1777.
- Kreitman RJ, Dearden C, Zinzani PL, et al. Study 1053 investigators. Moxetumomab pasudotox in heavily pre-treated patients with relapsed/refractory hairy cell leukemia (HCL): long-term follow-up from the pivotal trial. J Hematol Oncol. 2021; 14(1): 35.
- Tiacci E, Park JH, De Carolis L, et al. Targeting mutant BRAF in relapsed or refractory hairy-cell leukemia. N Engl J Med. 2015; 373(18): 1733–1747.
- Tiacci E, De Carolis L, Simonetti E, et al. Safety and efficacy of the BRAF inhibitor dabrafenib in relapsed or refractory hairy cell leukemia: a pilot phase-2 clinical trial. Leukemia. 2021 [Epub ahead of print].
- Rogers KA, Andritsos LA, Wei L, et al. Phase 2 study of ibrutinib in classic and variant hairy cell leukemia. Blood. 2021; 137(25): 3473–3483.
- Janus A, Robak T. Moxetumomab pasudotox for the treatment of hairy cell leukemia. Expert Opin Biol Ther. 2019; 19(6): 501–508.
- Yurkiewicz IR, Coutre S, Ghesquieres H, et al. Moxetumomab pasudotox as re-treatment for heavily-pretreated relapsed hairy cell leukemia. Leuk Lymphoma. 2021 [Epub ahead of print]; 62(11): 2812–2814.
- Siddiqui R, Sardar M, Shahzad M, et al. Management of relapsed hairy cell leukemia: a systematic review of novel agents and targeted therapies. Clin Lymphoma Myeloma Leuk. 2021 [Epub ahead of print]; 21(10): 659–666.
- Troussard X, Cornet E. Hairy cell leukemia 2018: update on diagnosis, risk-stratification, and treatment. Am J Hematol. 2017; 92(12): 1382–1390.
- Andritsos LA, Grever MR. Historical overview of hairy cell leukemia. Best Pract Res Clin Haematol. 2015; 28(4): 166–174.
- Liebers N, Roider T, Bohn JP, et al. BRAF inhibitor treatment in classic hairy cell leukemia: a long-term follow-up study of patients treated outside clinical trials. Leukemia. 2020; 34(5): 1454–1457.
- Moore JE, Delibert K, Baran AM, et al. Targeted therapy for treatment of patients with classical hairy cell leukemia. Leuk Res. 2021; 102: 106522.
- Dietrich S, Pircher A, Endris V, et al. BRAF inhibition in hairy cell leukemia with low-dose vemurafenib. Blood. 2016; 127(23): 2847–2855.
- Tiacci E, De Carolis L, Simonetti E, et al. Vemurafenib plus rituximab in refractory or relapsed hairy-cell leukemia. N Engl J Med. 2021; 384(19): 1810–1823.
- Robak T, Janus A, Jamroziak K, et al. Vemurafenib and rituximab in patients with hairy cell leukemia previously treated with moxetumomab pasudotox. J Clin Med. 2021; 10(13): 280.
- Robak E, Jesionek-Kupnicka D, Iskierka-Jazdzewska E, et al. Cutaneous leukocytoclastic vasculitis at diagnosis of hairy cell leukemia successfully treated with vemurafenib and rituximab. Leuk Res. 2021; 104: 106571.
- Caeser R, Collord G, Yao WQ, et al. Targeting MEK in vemurafenib-resistant hairy cell leukemia. Leukemia. 2019; 33(2): 541–545.
- Kreitman RJ, Moreau P, Hutchings M, et al. Treatment with combination of dabrafenib and trametinib in patients with recurrent/refractory BRAF V600E-mutated hairy cell leukemia (HCL). Blood. 2018; 132(Suppl 1): 391–391.
- Moore DC, Thompson D. A review of the Bruton tyrosine kinase inhibitors in B-cell malignancies. J Adv Pract Oncol. 2021; 12(4): 439–447.
- Liu Q, Harris N, Epperla N, et al. Current and emerging therapeutic options for hairy cell leukemia variant. Onco Targets Ther. 2021; 14: 1797–1805.
- Visentin A, Imbergamo S, Trimarco V, et al. Ibrutinib in relapsed hairy cell leukemia variant: a case report and review of the literature. Hematol Oncol. 2020; 38(5): 823–826.
- Visentin A, Imbergamo S, Frezzato F, et al. Bendamustine plus rituximab is an effective first-line treatment in hairy cell leukemia variant: a report of three cases. Oncotarget. 2017; 8(66): 110727–110731.
- Imoto N, Koyama D, Sugiura I, et al. Long-term follow-up after rituximab plus bendamustine in a patient with relapsed or refractory hairy cell leukemia variant: A case report. Medicine (Baltimore). 2021; 100(5): e24457.
- Matutes E, Wotherspoon A, Brito-Babapulle V, et al. The natural history and clinico-pathological features of the variant form of hairy cell leukemia. Leukemia. 2001; 15(1): 184–186.
- Yoshida T, Mihara K, Sugihara S, et al. Splenectomy followed by administration of rituximab is useful to treat a patient with hairy cell leukemia-variant. Ann Hematol. 2013; 92(5): 711–713.
- Kreitman RJ, Dearden C, Zinzani PL, et al. Study 1053 investigators. Responses in refractory hairy cell leukemia to a recombinant immunotoxin. Blood. 1999; 94(10): 3340–3348.