RESUMO
Operative Rigid Bronchoscopy treatment.
Assuntos
Plasmocitoma , Broncoscopia , Tosse/etiologia , Humanos , Plasmocitoma/complicações , Plasmocitoma/diagnóstico por imagemRESUMO
Ruxolitinib (RUX) is extensively used in myelofibrosis (MF). Despite its early efficacy, most patients lose response over time and, after discontinuation, have a worse overall survival (OS). Currently, response criteria able to predict OS in RUX-treated patients are lacking, leading to uncertainty regarding the switch to second-line treatments. In this study, we investigated predictors of survival collected after 6 months of RUX in 209 MF patients participating in the real-world ambispective observational RUXOREL-MF study (NCT03959371). Multivariable analysis identified the following risk factors: (1) RUX dose <20 mg twice daily at baseline, months 3 and 6 (hazard ratio [HR], 1.79; 95% confidence interval [CI], 1.07-3.00; P = .03), (2) palpable spleen length reduction from baseline ≤30% at months 3 and 6 (HR, 2.26; 95% CI, 1.40-3.65; P = .0009), (3) red blood cell (RBC) transfusion need at months 3 and/or 6 (HR, 1.66; 95% CI, 0.95-2.88; P = .07), and (4) RBC transfusion need at all time points (ie, baseline and months 3 and 6; HR, 2.32; 95% CI, 1.19-4.54; P = .02). Hence, we developed a prognostic model, named Response to Ruxolitinib After 6 Months (RR6), dissecting 3 risk categories: low (median OS, not reached), intermediate (median OS, 61 months; 95% CI, 43-80), and high (median OS, 33 months; 95% CI, 21-50). The RR6 model was validated and confirmed in an external cohort comprised of 40 MF patients. In conclusion, the RR6 prognostic model allows for the early identification of RUX-treated MF patients with impaired survival who might benefit from a prompt treatment shift.
Assuntos
Mielofibrose Primária , Humanos , Nitrilas , Mielofibrose Primária/induzido quimicamente , Mielofibrose Primária/diagnóstico , Mielofibrose Primária/tratamento farmacológico , Prognóstico , Pirazóis/efeitos adversos , Pirimidinas , Estudos RetrospectivosRESUMO
BACKGROUND AND OBJECTIVES: Standard treatment for patients with multiple myeloma is debulking chemotherapy with non-alkylating agents followed by a regimen to mobilize peripheral blood stem cells (PBSC) and the transplantation of the mobilized, autologous PBSC. The aim of this study was to evaluate the efficacy of a new regimen and compare it with that of a previous regimen. DESIGN AND METHODS: In a large cohort of 106 patients (group I) we administered a new pre-transplant program which includes 2 courses of pulsed-VAD (vincristine, adriamycin, dexamethasone) followed by 2 courses of DCEP (dexamethasone, cyclophosphamide, etoposide and cis-platinum). We compared the efficacy of this new VAD-DCEP sequence, in terms of mobilizing capacity, toxicity and anti-myeloma activity in comparison with that of the previous VAD-high-dose cyclophosphamide program (group II, 40 patients). RESULTS: In group I 81/106 (76.4%) patients yielded >or= 4x10(6)/kg CD34+ cells, as did 30/40 (75%) in group II but with a significantly higher toxicity in this latter group. In detail, 9 patients in group I (8.5%) had WHO grade III neutropenia versus 35 in group II (87.5%), 5 patients of group I (4.7%) had grade III thrombocytopenia versus 12 patients in group II (30%), and 8 patients in group I (7.5%) experienced an infections fever versus 9 patients in group II (22.5%). Therefore, nearly all patients in group II had to be admitted to hospital (39/40, 97.5%). There was a higher percentage of responses (CR+VGPR+PR) in group I than in group II: 73% versus 50% (p=0.02). INTERPRETATION AND CONCLUSIONS: the VAD-DCEP sequence has an adequate mobilizing capacity, without significant toxicity, and a good anti-myeloma activity, and therefore represents a safe and effective therapeutic approach for multiple myeloma patients at the onset of their disease.
Assuntos
Protocolos de Quimioterapia Combinada Antineoplásica/uso terapêutico , Mieloma Múltiplo/tratamento farmacológico , Adulto , Idoso , Protocolos de Quimioterapia Combinada Antineoplásica/administração & dosagem , Protocolos de Quimioterapia Combinada Antineoplásica/efeitos adversos , Cisplatino/administração & dosagem , Cisplatino/efeitos adversos , Estudos de Coortes , Terapia Combinada , Ciclofosfamida/administração & dosagem , Ciclofosfamida/efeitos adversos , Dexametasona/administração & dosagem , Dexametasona/efeitos adversos , Doxorrubicina/administração & dosagem , Doxorrubicina/efeitos adversos , Esquema de Medicação , Etoposídeo/administração & dosagem , Etoposídeo/efeitos adversos , Feminino , Mobilização de Células-Tronco Hematopoéticas , Humanos , Masculino , Melfalan/uso terapêutico , Pessoa de Meia-Idade , Mieloma Múltiplo/cirurgia , Terapia Neoadjuvante/efeitos adversos , Neutropenia/induzido quimicamente , Trombocitopenia/induzido quimicamente , Condicionamento Pré-Transplante/efeitos adversos , Transplante Autólogo , Resultado do Tratamento , Vincristina/administração & dosagem , Vincristina/efeitos adversosRESUMO
Pure red cell aplasia (PRCA) is a rare syndrome characterized by a normochromic normocytic anemia and the absence of mature erythroid precursors in an otherwise normocellular bone marrow. Acquired PRCA has been associated with autoimmune, viral or neoplastic conditions. We report the case of a patient with B-cell chronic lymphocytic leukemia (B-CLL) and PRCA. Conventional antileukemic treatment had no effect on the PRCA, while the B-CLL showed partial remission according to the International Workshop on Chronic Lymphocytic Leukemia response criteria. The patient was treated with cyclosporin A that resulted in complete response of the PRCA lasting 12 months. PRCA relapse, suggested by a hemoglobin decrease and by bone marrow biopsy, did not respond to prednisone and cyclophosphamide treatment. Fludarabine treatment was started in July 1999; the B-CLL remained stable according to the International Workshop on Chronic Lymphocytic Leukemia response criteria, the PRCA did not improve. In October 1999, because of intercurrent pneumonia, the patient was treated with intravenous immunoglobulin (IVIG) concentrate. This treatment resulted in total resolution of the pneumonia and a complete response of the PRCA. Therefore the patient remained on monthly IVIG. In view of the known efficacy of IVIG in the treatment of PRCA induced by parvovirus B19 and since serial polymerase chain reaction determinations of parvovirus B19 were repeatedly negative in our subject, we hypothesize that the IVIG per se may have a therapeutic effect on PRCA. The present case suggests that IVIG may be of benefit for PRCA patients even if the disease is unrelated to parvovirus B19 infection.