A subgroup analysis of JUMP, a phase IIIb, expanded-access study evaluating the safety and efficacy of ruxolitinib in patients with myelofibrosis in a Brazilian cohort

Abstract Introduction Ruxolitinib has been approved for the treatment of myelofibrosis (MF). In this study, we present safety and efficacy findings from an analysis of 104 patients with intermediate- and high-risk MF in a Brazilian cohort of the JUMP study who received treatment with ruxolitinib. Methods JUMP is a single-arm, open-label, phase IIIb, expanded-access study. The primary endpoint was to evaluate the safety and tolerability (frequency, duration, and severity of adverse events [AEs]) of ruxolitinib. Results All of the 104 patients received the treatment. Median duration of exposure was 35.8 months. The most common hematologic AEs were anemia (57.7), thrombocytopenia (38.5%), neutropenia (11.5%), and leukopenia (9.6%). Second malignancies (all grades) occurred in 19.2% of patients (n = 20). Serious AEs were reported in 62.5% of patients (n = 65). The proportions of patients with ≥50% reduction from baseline in palpable spleen length at weeks 24 and 48 were 62.7% and 69.2%, respectively. The mean change from the baseline in the Functional Assessment of Cancer Therapy (FACT)-Lymphoma total score was 10.8 [15.6%] at week 4, 12.6 [14.1%] at week 24, and 12.2 [14.3%] at week 48. The mean change from the baseline for the Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue scale was 3.9 [42.8%] at week 4, 4.9 [29.9%] at week 24, and 4.7 [28%] at week 48. At week 48, the estimated progression-free survival, leukemia-free survival, and overall survival probabilities were 91%, 91% and 93%, respectively Overall, 21 deaths were observed in the present study. Conclusion Findings from this study suggest that ruxolitinib could be evaluated as a standard-of-care treatment for the MF population in need of a viable treatment option. NCT01493414

Primary myelofibrosis: risk stratification by IPSS identifies patients with poor clinical outcome

OBJECTIVES: To evaluate whether risk scores used to classify patients with primary myelofibrosis and JAK-2 V617F mutation status can predict clinical outcome. METHODS: A review of clinical and laboratory data from 74 patients with primary myelofibrosis diagnosed between 1992 and 2011. The IPSS and Lille scores were calculated for risk stratification and correlated with overall survival. RESULTS: A V617F JAK2 mutation was detected in 32 cases (47%), with no significant correlation with overall survival. The patients were classified according to the scores: Lille - low, 53 (73.%); intermediate, 13 (18%); and high, 5 (7%); and IPSS- low, 15 (26%); intermediate-1, 23 (32%); intermediate-2, 19 (26%); and high, 15 (31%). Those patients presenting a higher risk according to the IPSS (high and intermediate-2) had a significantly shorter overall survival relative to the low risk groups (intermediate-1 and low) (p = 0.02). CONCLUSIONS: These results emphasize the importance of the IPSS prognostic score for risk assessment in predicting the clinical outcome of primary myelofibrosis patients.

Metaplasia mieloide agnogénica: reporte de un caso atípico / Agnogenic myeloid metaplasia: case of report atipic

Se presenta un paciente varón de 19 años de edad, procedente de Papayacu, departamento de Amazonas, con metaplasia mieloide agnogénica (MMA), cuya sintomatología y evolución-con excepción de la fiebre y la esplenomegalia-fueron inusuales, lo cual obscureció el diagnóstico que sólo pudo evidenciarse post-mortem. Paralelamente era portador de una severa desnutrición calórico-proteica y de parasitismo intestinal múltiple, lo que contribuyó a modificar el curso clínico del proceso. Creemos por ello que este caso de metaplasia mieloide agnogénica de presentación muy singular debe ser reportado en la literatura nacional.

Mielofibrose idiopática / Idiopathic myoelofibrosis

Os autores fazem uma revisäo bibliográfica recente e comparam os dados obtidos aos de prontuários médicos do Serviço de Hematologia do Hospital Brigadeiro - Säo Paulo, no período de 1983 a 1988. Foram analisados oito casos de mielofibrose idiopática (MFI), confirmados através de biópsia óssea de crista ilíaca posterior com agulha de Jamshidi. A faixa etária preodminante foi de 51 a 60 anos, sendo cinco pacientes do sexo masculino e três do sexo feminino. Os sintomas mais referidos foram adinamia, fraqueza e sangramento (seis-oito casos), emagrecimento e dores ósseas (quatro-oito casos). Os sinais encontrados com maior freqüência foram explenomegalia e anemia (sete-oito casos), hepatomegalia (cinco-oito casos) e icterícia (dois-oito casos). O hematoma näo apresentou padräo definido. A maior parte dos casos foram diagnosticados em estádios avançados da doença. O tratamento isolado ou combinado com bussulfan, prednisona, oximetalona e radioterapia esplênica mostrou-se útil para alívio de sintomas abdominais compressivos. A análise retrospectiva dos pacientes con MFI demonstra que essa patologia deve ser obrigatoriamente incluída no diagnóstico diferencial de hepatoesplenomegalia associada a anemia