Assuntos
Anemia Mielopática , Medula Óssea , Neoplasias da Mama , Leucemia Mieloide Aguda , Anemia Mielopática/genética , Anemia Mielopática/metabolismo , Anemia Mielopática/patologia , Medula Óssea/metabolismo , Medula Óssea/patologia , Neoplasias da Mama/genética , Neoplasias da Mama/metabolismo , Neoplasias da Mama/patologia , Feminino , Humanos , Leucemia Mieloide Aguda/genética , Leucemia Mieloide Aguda/metabolismo , Pessoa de Meia-IdadeRESUMO
We present results of 2 similarly designed but separate phase 2 studies involving single-agent lenalidomide (CC-5013, Revlimid) in a total of 68 patients with symptomatic myelofibrosis with myeloid metaplasia (MMM). Protocol treatment consisted of oral lenalidomide at 10 mg/d (5 mg/d if baseline platelet count < 100 x 10(9)/L) for 3 to 4 months with a plan to continue treatment for either 3 or 24 additional months, in case of response. Overall response rates were 22% for anemia, 33% for splenomegaly, and 50% for thrombocytopenia. Response in anemia was deemed impressive in 8 patients whose hemoglobin level normalized from a baseline of either transfusion dependency or hemoglobin level lower than 100 g/L. Additional treatment effects in these patients included resolution of leukoerythroblastosis (4 patients), a decrease in medullary fibrosis and angiogenesis (2 patients), and del(5)(q13q33) cytogenetic remission accompanied by a reduction in JAK2(V617F) mutation burden (1 patient). Grade 3 or 4 adverse events included neutropenia (31%) and thrombocytopenia (19%). We conclude that lenalidomide engenders an intriguing treatment activity in a subset of patients with MMM that includes an unprecedented effect on peripheral blood and bone marrow abnormalities.
Assuntos
Mielofibrose Primária/tratamento farmacológico , Talidomida/análogos & derivados , Administração Oral , Adulto , Idoso , Anemia/sangue , Anemia/complicações , Anemia/tratamento farmacológico , Anemia/genética , Anemia/patologia , Anemia Mielopática/sangue , Anemia Mielopática/complicações , Anemia Mielopática/tratamento farmacológico , Anemia Mielopática/genética , Anemia Mielopática/patologia , Feminino , Hemoglobinas/análise , Humanos , Janus Quinase 2 , Lenalidomida , Masculino , Neovascularização Patológica/sangue , Neovascularização Patológica/complicações , Neovascularização Patológica/tratamento farmacológico , Neovascularização Patológica/genética , Neovascularização Patológica/patologia , Neutropenia/sangue , Neutropenia/induzido quimicamente , Neutropenia/genética , Neutropenia/patologia , Contagem de Plaquetas , Mutação Puntual , Mielofibrose Primária/sangue , Mielofibrose Primária/complicações , Mielofibrose Primária/genética , Mielofibrose Primária/patologia , Proteínas Tirosina Quinases/genética , Proteínas Proto-Oncogênicas/genética , Indução de Remissão , Deleção de Sequência , Esplenomegalia/sangue , Esplenomegalia/complicações , Esplenomegalia/tratamento farmacológico , Esplenomegalia/genética , Esplenomegalia/patologia , Talidomida/administração & dosagem , Talidomida/efeitos adversos , Trombocitopenia/sangue , Trombocitopenia/induzido quimicamente , Trombocitopenia/tratamento farmacológico , Trombocitopenia/genética , Trombocitopenia/patologiaRESUMO
The case is presented of a 64-year-old man who has had recurrent psychiatric symptoms over several years, and now has minor evidence of a myeloproliferative disorder. He had a buccal carcinoma successfully treated 33 years previously, thus, the possibility of bone marrow infiltration has been excluded. An acquired translocation that was found in his bone marrow cells has not been previously reported in association with any neoplasm. The possible significance of the translocation to this patient is discussed.
