BRAF in the cross-hairs

References

• Bouroncle BA, Wiseman BK, Doan CA. Leukemic reticuloendotheliosis. Blood. 1958;13(7):609–630.
   PubMed Web of Science ®Google Scholar
• Catovsky D. Hairy-cell leukaemia and prolymphocytic leukaemia. Clin Haematol. 1977;6(1):245–268.
   PubMedGoogle Scholar
• Golomb HM, Catovsky D, Golde DW. Hairy cell leukemia: a clinical review based on 71 cases. Ann Intern Med. 1978;89(5 Pt 1):677–683.
   PubMed Web of Science ®Google Scholar
• 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.
   PubMed Web of Science ®Google Scholar
• 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.
   PubMed Web of Science ®Google Scholar
• Hakimian D, Tallman MS, Hogan DK, et al. Prospective evaluation of internal adenopathy in a cohort of 43 patients with hairy cell leukemia. J Clin Oncol. 1994;12(2):268–272.
   PubMed Web of Science ®Google Scholar
• 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.
   PubMed Web of Science ®Google Scholar
• 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.
   PubMed Web of Science ®Google Scholar
• Robak T, Blasinska-Morawiec M, Krykowski E, et al. 2-chlorodeoxyadenosine (2-CdA) in 2-hour versus 24-hour intravenous infusion in the treatment of patients with hairy cell leukemia. Leuk Lymphoma. 1996;22(1–2):107–111.
   PubMed Web of Science ®Google Scholar
• von Rohr A, Schmitz SF, Tichelli A, et al. Treatment of hairy cell leukemia with cladribine (2-chlorodeoxyadenosine) by subcutaneous bolus injection: a phase II study. Ann Oncol. 2002;13(10):1641–1649.
   PubMed Web of Science ®Google Scholar
• 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.
   PubMed Web of Science ®Google Scholar
• Maloisel F, Benboubker L, Gardembas M, et al. Long-term outcome with pentostatin treatment in hairy cell leukemia patients. A French retrospective study of 238 patients. Leukemia. 2003;17(1):45–51.
   PubMed Web of Science ®Google Scholar
• Lotfi K, Juliusson G, Albertioni F. Pharmacological basis for cladribine resistance. Leuk Lymphoma. 2003;44(10):1705–1712.
   PubMed Web of Science ®Google Scholar
• Forconi F, Cencini E, Sozzi E, et al. Insight into the behavior of hairy cell leukemia by immunogenetic analysis. Leuk Lymphoma. 2011;52(Suppl 2):103–107.
   PubMedGoogle Scholar
• Forconi F, Sozzi E, Cencini E, et al. Hairy cell leukemias with unmutated IGHV genes define the minor subset refractory to single-agent cladribine and with more aggressive behavior. Blood. 2009;114(21):4696–4702.
   PubMed Web of Science ®Google Scholar
• Keshet Y, Seger R. The MAP kinase signaling cascades: a system of hundreds of components regulates a diverse array of physiological functions. Methods Mol Biol. 2010;661:3–38.
   PubMedGoogle Scholar
• Davies H, Bignell GR, Cox C, et al. Mutations of the BRAF gene in human cancer. Nature. 2002;417(6892):949–954.
   PubMed Web of Science ®Google Scholar
• Dong J, Phelps RG, Qiao R, et al. BRAF oncogenic mutations correlate with progression rather than initiation of human melanoma. Cancer Res. 2003;63(14):3883–3885.
   PubMed Web of Science ®Google Scholar
• Flaherty KT, Puzanov I, Kim KB, et al. Inhibition of mutated, activated BRAF in metastatic melanoma. N Engl J Med. 2010;363(9):809–819.
   PubMed Web of Science ®Google Scholar
• Chapman PB, Hauschild A, Robert C, et al. Improved survival with vemurafenib in melanoma with BRAF V600E mutation. N Engl J Med. 2011;364(26):2507–2516.
   PubMed Web of Science ®Google Scholar
• Nazarian R, Shi H, Wang Q, et al. Melanomas acquire resistance to B-RAF(V600E) inhibition by RTK or N-RAS upregulation. Nature. 2010;468(7326):973–977.
   PubMed Web of Science ®Google Scholar
• Poulikakos PI, Persaud Y, Janakiraman M, et al. RAF inhibitor resistance is mediated by dimerization of aberrantly spliced BRAF(V600E). Nature. 2011;480(7377):387–390.
   PubMed Web of Science ®Google Scholar
• Poulikakos PI, Zhang C, Bollag G, et al. RAF inhibitors transactivate RAF dimers and ERK signalling in cells with wild-type BRAF. Nature. 2010;464(7287):427–430.
   PubMed Web of Science ®Google Scholar
• Hatzivassiliou G, Song K, Yen I, et al. RAF inhibitors prime wild-type RAF to activate the MAPK pathway and enhance growth. Nature. 2010;464(7287):431–435.
   PubMed Web of Science ®Google Scholar
• Flaherty KT, Infante JR, Daud A, et al. Combined BRAF and MEK inhibition in melanoma with BRAF V600 mutations. N Engl J Med. 2012;367(18):1694–1703.
   PubMed Web of Science ®Google Scholar
• Long GV, Stroyakovskiy D, Gogas H, et al. Combined BRAF and MEK inhibition versus BRAF inhibition alone in melanoma. N Engl J Med. 2014;371(20):1877–1888.
   PubMed Web of Science ®Google Scholar
• Larkin J, Ascierto PA, Dreno B, et al. Combined vemurafenib and cobimetinib in BRAF-mutated melanoma. N Engl J Med. 2014;371(20):1867–1876.
   PubMed Web of Science ®Google Scholar
• Tiacci E, Trifonov V, Schiavoni G, et al. BRAF mutations in hairy-cell leukemia. N Engl J Med. 2011;364(24):2305–2315.
   PubMed Web of Science ®Google Scholar
• Boyd EM, Bench AJ, van ‘T Veer MB, et al. High resolution melting analysis for detection of BRAF exon 15 mutations in hairy cell leukaemia and other lymphoid malignancies. Br J Haematol. 2011;155(5):609–612.
   PubMed Web of Science ®Google Scholar
• Tiacci E, Schiavoni G, Forconi F, et al. Simple genetic diagnosis of hairy cell leukemia by sensitive detection of the BRAF-V600E mutation. Blood. 2012;119(1):192–195.
   PubMed Web of Science ®Google Scholar
• Schnittger S, Bacher U, Haferlach T, et al. Development and validation of a real-time quantification assay to detect and monitor BRAFV600E mutations in hairy cell leukemia. Blood. 2012;119(13):3151–3154.
   PubMed Web of Science ®Google Scholar
• Tschernitz S, Flossbach L, Bonengel M, et al. Alternative BRAF mutations in BRAF V600E-negative hairy cell leukaemias. Br J Haematol. 2014;165(4):529–533.
   PubMed Web of Science ®Google Scholar
• Bielski CM, Donoghue MTA, Gadiya M, et al. Widespread selection for oncogenic mutant allele imbalance in cancer. Cancer Cell. 2018;34(5):852–862 e854.
   PubMed Web of Science ®Google Scholar
• Durham BH, Getta B, Dietrich S, et al. Genomic analysis of hairy cell leukemia identifies novel recurrent genetic alterations. Blood. 2017;130(14):1644–1648.
   PubMed Web of Science ®Google Scholar
• Xi L, Arons E, Navarro W, et al. Both variant and IGHV4-34-expressing hairy cell leukemia lack the BRAF V600E mutation. Blood. 2012;119(14):3330–3332.
   PubMed Web of Science ®Google Scholar
• Waterfall JJ, Arons E, Walker RL, et al. High prevalence of MAP2K1 mutations in variant and IGHV4-34-expressing hairy-cell leukemias. Nat Genet. 2014;46(1):8–10.
   PubMed Web of Science ®Google Scholar
• Angelova EA, Medeiros LJ, Wang W, et al. Clinicopathologic and molecular features in hairy cell leukemia-variant: single institutional experience. Mod Pathol. 2018;31(11):1717–1732.
   PubMed Web of Science ®Google Scholar
• Mason EF, Brown RD, Szeto DP, et al. Detection of activating MAP2K1 mutations in atypical hairy cell leukemia and hairy cell leukemia variant. Leuk Lymphoma. 2017;58(1):233–236.
   PubMed Web of Science ®Google Scholar
• Chung SS, Kim E, Park JH, et al. Hematopoietic stem cell origin of BRAFV600E mutations in hairy cell leukemia. Sci Transl Med. 2014;6(238):238ra271.
   Web of Science ®Google Scholar
• Richards JM, Mick R, Latta JM, et al. Serum soluble interleukin-2 receptor is associated with clinical and pathologic disease status in hairy cell leukemia. Blood. 1990;76(10):1941–1945.
   PubMed Web of Science ®Google Scholar
• Mercer K, Giblett S, Green S, et al. Expression of endogenous oncogenic V600EB-raf induces proliferation and developmental defects in mice and transformation of primary fibroblasts. Cancer Res. 2005;65(24):11493–11500.
   PubMed Web of Science ®Google Scholar
• Pettirossi V, Santi A, Imperi E, et al. BRAF inhibitors reverse the unique molecular signature and phenotype of hairy cell leukemia and exert potent antileukemic activity. Blood. 2015;125(8):1207–1216.
   PubMed Web of Science ®Google Scholar
• Dietrich S, Glimm H, Andrulis M, et al. BRAF inhibition in refractory hairy-cell leukemia. N Engl J Med. 2012;366(21):2038–2040.
   PubMed Web of Science ®Google Scholar
• Dietrich S, Hullein J, Hundemer M, et al. Continued response off treatment after BRAF inhibition in refractory hairy cell leukemia. J Clin Oncol. 2013;31(19):e300–e303.
   PubMed Web of Science ®Google Scholar
• Follows GA, Sims H, Bloxham DM, et al. Rapid response of biallelic BRAF V600E mutated hairy cell leukaemia to low dose vemurafenib. Br J Haematol. 2013;161(1):150–153.
   PubMed Web of Science ®Google Scholar
• Peyrade F, Re D, Ginet C, et al. Low-dose vemurafenib induces complete remission in a case of hairy-cell leukemia with a V600E mutation. Haematologica. 2013;98(2):e20–e22.
   PubMed Web of Science ®Google Scholar
• Munoz J, Schlette E, Kurzrock R. Rapid response to vemurafenib in a heavily pretreated patient with hairy cell leukemia and a BRAF mutation. J Clin Oncol. 2013;31(20):e351–e352.
   PubMed Web of Science ®Google Scholar
• Bailleux C, Robert G, Ginet C, et al. Successful re-treatment of a relapsed V600E mutated HCL patient with low-dose vemurafenib. Oncoscience. 2015;2(1):44–49.
   PubMedGoogle Scholar
• Maurer H, Haas P, Wengenmayer T, et al. Successful vemurafenib salvage treatment in a patient with primary refractory hairy cell leukemia and pulmonary aspergillosis. Ann Hematol. 2014;93(8):1439–1440.
   PubMed Web of Science ®Google Scholar
• McDowell MM, Zhu X, Agarwal N, et al. Response of relapsed central nervous system hairy cell leukemia to vemurafenib. Leuk Lymphoma. 2016;57(12):2952–2954.
   PubMed Web of Science ®Google Scholar
• McArthur GA, Maio M, Arance A, et al. Vemurafenib in metastatic melanoma patients with brain metastases: an open-label, single-arm, phase 2, multicentre study. Ann Oncol. 2017;28(3):634–641.
   PubMed Web of Science ®Google Scholar
• Blachly JS, Lozanski G, Lucas DM, et al. Cotreatment of hairy cell leukemia and melanoma with the BRAF inhibitor dabrafenib. J Natl Compr Canc Netw. 2015;13(1):9–13. quiz 13.
   PubMed Web of Science ®Google Scholar
• Dietrich S, Pircher A, Endris V, et al. BRAF inhibition in hairy cell leukemia with low-dose vemurafenib. Blood. 2016;127(23):2847–2855.
   PubMed Web of Science ®Google Scholar
• 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.
   PubMed Web of Science ®Google Scholar
• Ambrosetti A, Nadali G, Vinante F, et al. Soluble interleukin-2 receptor in hairy-cell leukemia: a reliable marker of disease. Int J Clin Lab Res. 1993;23(1):34–37.
   PubMedGoogle Scholar
• Lacouture ME, Duvic M, Hauschild A, et al. Analysis of dermatologic events in vemurafenib-treated patients with melanoma. Oncologist. 2013;18(3):314–322.
   PubMed Web of Science ®Google Scholar
• Su F, Viros A, Milagre C, et al. RAS mutations in cutaneous squamous-cell carcinomas in patients treated with BRAF inhibitors. N Engl J Med. 2012;366(3):207–215.
   PubMed Web of Science ®Google Scholar
• Callahan MK, Rampal R, Harding JJ, et al. Progression of RAS-mutant leukemia during RAF inhibitor treatment. N Engl J Med. 2012;367(24):2316–2321.
   PubMed Web of Science ®Google Scholar
• Dietrich S, Hullein J, Lee SC, et al. Recurrent CDKN1B (p27) mutations in hairy cell leukemia. Blood. 2015;126(8):1005–1008.
   PubMed Web of Science ®Google Scholar
• Caeser R, Collord G, Yao WQ, et al. Targeting MEK in vemurafenib-resistant hairy cell leukemia. Leukemia. 2019;33(2):541–545.
   PubMed Web of Science ®Google Scholar
• Morris EJ, Jha S, Restaino CR, et al. Discovery of a novel ERK inhibitor with activity in models of acquired resistance to BRAF and MEK inhibitors. Cancer Discov. 2013;3(7):742–750.
   PubMed Web of Science ®Google Scholar
• Carlino MS, Todd JR, Gowrishankar K, et al. Differential activity of MEK and ERK inhibitors in BRAF inhibitor resistant melanoma. Mol Oncol. 2014;8(3):544–554.
   PubMed Web of Science ®Google Scholar
• Germann U, Furey B, Roix J, et al. Abstract 4693: the selective ERK inhibitor BVD-523 is active in models of MAPK pathway-dependent cancers, including those with intrinsic and acquired drug resistance. Cancer Res. 2015;75(15 Supplement):4693.
   Google Scholar
• Sullivan RJ, Infante JR, Janku F, et al. First-in-class erk1/2 inhibitor ulixertinib (BVD-523) in patients with mapk mutant advanced solid tumors: results of a phase i dose-escalation and expansion study. Cancer Discov. 2018;8(2):184–195.
   PubMed Web of Science ®Google Scholar
• Tallman MS, Hakimian D, Variakojis D, et al. A single cycle of 2-chlorodeoxyadenosine results in complete remission in the majority of patients with hairy cell leukemia. Blood. 1992;80(9):2203–2209.
   PubMed Web of Science ®Google Scholar
• Piro LD, Carrera CJ, Carson DA, et al. Lasting remissions in hairy-cell leukemia induced by a single infusion of 2-chlorodeoxyadenosine. N Engl J Med. 1990;322(16):1117–1121.
   PubMed Web of Science ®Google Scholar
• Tallman MS, Hakimian D, Kopecky KJ, et al. Minimal residual disease in patients with hairy cell leukemia in complete remission treated with 2-chlorodeoxyadenosine or 2-deoxycoformycin and prediction of early relapse. Clin Cancer Res. 1999;5(7):1665–1670.
   PubMed Web of Science ®Google Scholar
• Sanmamed MF, Fernandez-Landazuri S, Rodriguez C, et al. Quantitative cell-free circulating BRAFV600E mutation analysis by use of droplet digital PCR in the follow-up of patients with melanoma being treated with BRAF inhibitors. Clin Chem. 2015;61(1):297–304.
   PubMed Web of Science ®Google Scholar
• Tiacci E, De Carolis L, Zaja F, et al. The chemotherapy-free combination of vemurafenib and rituximab produces deep and durable responses in relapsed or refractory Hairy Cell Leukemia (HCL) patients. Blood. 2017;130(Suppl 1):409.
   Google Scholar
• Goede V, Fischer K, Busch R, et al. Obinutuzumab plus chlorambucil in patients with CLL and coexisting conditions. N Engl J Med. 2014;370(12):1101–1110.
   PubMed Web of Science ®Google Scholar
• Kreitman RJ, Dearden C, Zinzani PL, et al. Moxetumomab pasudotox in relapsed/refractory hairy cell leukemia. Leukemia. 2018;32(8):1768–1777.
   PubMed Web of Science ®Google Scholar
• Jones J, Andritsos L, Kreitman RJ, et al. Efficacy and safety of the bruton tyrosine kinase inhibitor ibrutinib in patients with hairy cell leukemia: stage 1 results of a phase 2 study. Blood. 2016;128(22):1215.
   Google Scholar