Title |
Busulfan in patients with polycythemia vera or essential thrombocythemia refractory or intolerant to hydroxyurea
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Published in |
Annals of Hematology, July 2014
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DOI | 10.1007/s00277-014-2152-7 |
Pubmed ID | |
Authors |
Alberto Alvarez-Larrán, Luz Martínez-Avilés, Juan Carlos Hernández-Boluda, Francisca Ferrer-Marín, María Luisa Antelo, Carmen Burgaleta, M. Isabel Mata, Blanca Xicoy, Alejandra Martínez-Trillos, M. Teresa Gómez-Casares, M. Antonia Durán, Bárbara Marcote, Agueda Ancochea, Alicia Senín, Anna Angona, Montse Gómez, Vicente Vicente, Francisco Cervantes, Beatriz Bellosillo, Carles Besses |
Abstract |
Therapeutic options for patients with polycythemia vera (PV) and essential thrombocythemia (ET) resistant or intolerant to hydroxyurea are limited. Busulfan is effective as first-line therapy, but there is scarce information on this drug as second-line treatment. The efficacy of busulfan in patients with advanced PV or ET refractory or intolerant to hydroxyurea was assessed in 36 patients (PV n = 15, ET n = 21) treated for a median of 256 days. Complete hematological response (CHR) was achieved in 83 % of patients, after a median time of 203 days (range 92-313). The probability of sustained CHR at 1 and 2 years was 87 and 62 %, respectively. Time to CHR was shorter in patients treated with ≥14 mg of busulfan per week than with lower doses (141 versus 336 days, p = 0.01). Partial molecular response was achieved in three out of nine (33 %) patients. Busulfan was stopped in 27 patients (75 %) due to CHR achievement in 18 cases (67 %), hematological toxicity in 8 cases (30 %), and disease transformation in 1 case. With a median follow-up of 721 days, six patients have died, with the probability of survival at 2 years being 85 %. The probability of thrombosis at 2 years was 11 %. Transformation into acute leukemia or myelodysplastic syndrome was observed in three cases, all of them in a JAK2V617F-negative clone carrying additional mutations. Busulfan, at a dose of 2 mg/day, is an effective option for elderly patients with PV or ET who fail to hydroxyurea, but a significant rate of transformation was observed. |
X Demographics
Geographical breakdown
Country | Count | As % |
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Spain | 1 | 100% |
Demographic breakdown
Type | Count | As % |
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Members of the public | 1 | 100% |
Mendeley readers
Geographical breakdown
Country | Count | As % |
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United States | 2 | 3% |
Unknown | 59 | 97% |
Demographic breakdown
Readers by professional status | Count | As % |
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Researcher | 7 | 11% |
Student > Master | 7 | 11% |
Student > Doctoral Student | 5 | 8% |
Student > Bachelor | 5 | 8% |
Student > Ph. D. Student | 5 | 8% |
Other | 13 | 21% |
Unknown | 19 | 31% |
Readers by discipline | Count | As % |
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Medicine and Dentistry | 23 | 38% |
Biochemistry, Genetics and Molecular Biology | 4 | 7% |
Nursing and Health Professions | 3 | 5% |
Environmental Science | 2 | 3% |
Pharmacology, Toxicology and Pharmaceutical Science | 2 | 3% |
Other | 5 | 8% |
Unknown | 22 | 36% |