Humans ; Primary Myelofibrosis ; therapy

Myelofibrosis, a clonal stem-cell disorder which is difficult to cure, The treatment for most of patients is conservative treatment, but the treatment effect is not ideal. Lenalidomide as a novel immunomodulator in the treatment of blood diseases showed good results in recent years, Its studies have shown that lenalidomide in myelofibrosis also showed a certain effect. As companed with single drug lenalidomide, the thalidomide has a higher response rate, clinical symptoms improved significantly. Ruxolitinib, JAK2 inhibitor, combined with lenalidomide not only can improve the quality of life of patients, but also extend the survival of patients. In addition, lenalidomide combined with prednisone for the treatment of bone marrow fibrosis is more effective and more safe, lenalidomide can significantly improve the clinical symptoms of patients, especially anemia, and prednisone can reduce the hematologic toxicity of lenalidomids. The purpose of this review is to evaluate the efficacy and safety of lenalidomide in the treatment of myelofibrosis, and focuses on the newest clinical research and application progress of lenalidomide for myelofibrosis.
Humans ; Lenalidomide ; therapeutic use ; Prednisone ; Primary Myelofibrosis ; drug therapy ; Quality of Life ; Thalidomide

Objective: To analyze the influencing factors of iron metabolism assessment in patients with myelodysplastic syndrome. Methods: MRI and/or DECT were used to detect liver and cardiac iron content in 181 patients with MDS, among whom, 41 received regular iron chelation therapy during two examinations. The adjusted ferritin (ASF) , erythropoietin (EPO) , cardiac function, liver transaminase, hepatitis antibody, and peripheral blood T cell polarization were detected and the results of myelofibrosis, splenomegaly, and cyclosporine were collected and comparative analyzed in patients. Results: We observed a positive correlation between liver iron concentration and ASF both in the MRI group and DECT groups (r=0.512 and 0.606, respectively, P<0.001) , only a weak correlation between the heart iron concentration and ASF in the MRI group (r=0.303, P<0.001) , and no significant correlation between cardiac iron concentration and ASF in the DECT group (r=0.231, P=0.053) . Moreover, transfusion dependence in liver and cardiac [MRI group was significantly associated with the concentration of iron in: LIC: (28.370±10.706) mg/g vs (7.593±3.508) mg/g, t=24.30, P<0.001; MIC: 1.81 vs 0.95, z=2.625, P<0.05; DECT group: liver VIC: (4.269±1.258) g/L vs (1.078±0.383) g/L, t=23.14, P<0.001: cardiac VIC: 1.69 vs 0.68, z=3.142, P<0.05]. The concentration of EPO in the severe iron overload group was significantly higher than that in the mild to moderate iron overload group and normal group (P<0.001) . Compared to the low-risk MDS group, the liver iron concentration in patients with MDS with cyclic sideroblasts (MDS-RS) was significantly elevated [DECT group: 3.80 (1.97, 5.51) g/L vs 1.66 (0.67, 2.94) g/L, P=0.004; MRI group: 13.7 (8.1,29.1) mg/g vs 11.6 (7.1,21.1) mg/g, P=0.032]. Factors including age, bone marrow fibrosis, splenomegaly, T cell polarization, use of cyclosporine A, liver aminotransferase, and hepatitis antibody positive had no obvious effect on iron metabolism. Conclusion: There was a positive correlation between liver iron concentration and ASF in patients with MDS, whereas there was no significant correlation between cardiac iron concentration and ASF. Iron metabolism was affected by transfusion dependence, EPO concentration, and RS.
Ferritins ; Humans ; Iron ; Iron Overload ; Liver/metabolism* ; Myelodysplastic Syndromes/therapy* ; Primary Myelofibrosis ; Retrospective Studies ; Splenomegaly

Primary myelofibrosis (PMF) is easily confused with cirrhosis, due to its main clinical manifestations of splenomegaly and the blood cytopenia. This review focuses on clinical studies to identify primary myelofibrosis and cirrhosis related portal hypertension, to analyze the differences between the two diseases, in order to distinguish PMF and cirrhosis from the pathogenesis, clinical manifestations, laboratory examinations and treatment principles, and simultaneously improve clinicians' understanding of PMF, which is a reference for exploring the early screening or diagnostic indicators of PMF, also provides a clinical basis for the application of new targeted drugs such as ruxolitinib.
Humans ; Primary Myelofibrosis/drug therapy* ; Hypertension, Portal/complications* ; Liver Cirrhosis/pathology* ; Splenomegaly/pathology* ; Anemia

Child ; Cord Blood Stem Cell Transplantation ; Hematopoietic Stem Cell Transplantation ; Humans ; Primary Myelofibrosis/therapy* ; Thiotepa ; Transplantation Conditioning

We report a case of bilateral peripheral retinal neovascularization and chronic idiopathic myelofibrosis in a 69-year-old man. Ophthalmic examination revealed peripheral retinal nonperfusion with retinal neovascularization in both eyes and vitreous hemorrhage in the right eye. Fluorescein angiography of both eyes showed a marked midperipheral and peripheral avascular retina temporally with arteriovenous anastomosis and sea-fan neovascularizations. Blood tests showed pancytopenia and teardrop-shaped red blood cells, and bone marrow examination showed hypocellular marrow with severe fibrosis. The neovascularization was regressed following pars plana vitrectomy in the right eye and scatter laser photocoagulation in the left. The results suggest that peripheral retinal vessel occlusion and neovascularization may be associated with idiopathic myelofibrosis.
Aged ; Chronic Disease ; Fluorescein Angiography ; Humans ; Light Coagulation ; Male ; Primary Myelofibrosis/*complications ; Retinal Neovascularization/*complications/therapy ; Visual Acuity ; Vitrectomy ; Vitreous Hemorrhage/*complications/therapy

We experienced a case of acute panmyelosis with myelofibrosis, one of the acute myeloblastic leukemia (AML) subtypes according to WHO classification. A 44 year old man presented with a laborious dyspnea, dizziness, and generalized weakness. On admission, the peripheral blood revealed pancytopenia (hemoglobin 4.0 g/dL, WBC 2, 300/microliter, platelet 92, 000/microliter) with leukoerythroblastosis. The bone marrow (BM) aspirate yielded inadequate material but histological sections showed hyper-cellularity, proliferation of trilineage cell lines (panmyelosis) with extensive myelofibrosis, and clusters of immature cells at paratrabecular area. On ultrasonographic examination, no evidence of hepato-splenomegaly or any other abnormalities were noted. The follow-up BM study after the chemotherapy with Ara C for 7 days and idarubicin for 3 days showed no significant changes in pancytopenia and myelofibrosis but revealed a significant decrease in BM cellularity and blasts. We review and report the literature on acute panmyelosis with myelofibrosis.
Blood Platelets ; Bone Marrow ; Cell Line ; Classification ; Dizziness ; Drug Therapy ; Dyspnea ; Follow-Up Studies ; Idarubicin ; Leukemia, Myeloid, Acute ; Pancytopenia ; Primary Myelofibrosis*

Idiopathic myelofibrosis a Philadelphia-negative chronic myeloproliferative disorder. Potentially curative therapies, such as stem-cell transplantation, are reserved only for a minority of patients. Currently palliative therapies such as androgen and hydroxycarbamide are commonly used but with poor results. Thalidomide has anti-angiogenic effect and also can inhibit cytokines, and therefore plays a certain role in the treatment of a subset of idiopathic myelofibrosis.
Angiogenesis Inhibitors ; adverse effects ; therapeutic use ; Humans ; Immunosuppressive Agents ; adverse effects ; therapeutic use ; Primary Myelofibrosis ; drug therapy ; Thalidomide ; adverse effects ; therapeutic use ; Treatment Outcome

Objective: To evaluate the efficacy and tolerability of ruxolitinib combined with prednisone, thalidomide and danazol for treatment of in myelofibrosis (MF). Methods: Patients of MF according to the WHO 2016 criteria, received ruxolitinib (RUX) combined with prednisone, thalidomide and danazol (PTD). The response, changes of blood counts and adverse events were evaluated. Results: Six PMF and one post-ET MF patients were enrolled. Four patients presented JAK2V617F mutation, one CALR mutation, one MPL mutation, one triple-negative. Responses per IWG-MRT criteria were clinical improvement in 5 patients, stable disease in 2 ones, spleen response in 6 ones. All of 7 patients were symptomatic responses, four patients achieved at least 50% improvement from baseline on MPN-SAF TSS. Three patients initially treated with RUX alone, all of 3 patients experienced treatment-associated anemia and thrombocytopenia. Then these 3 patients received RUX combined with PTD, both hemoglobin and platelet increased significantly. Four patients initially treated with RUX combined with PTD. Increased levels of hemoglobin and platelet were seen in all of 7 patients received RUX combined with PTD with maximum increased hemoglobin of 30(18-54) g/L and maximum increased platelets of 116(13-369)×10(9)/L, respectively from baseline. The treatment dose of RUX increased due to improved platelet count in 3 patients. The frequent non-hematologic adverse events grade 1-2 were constipation, abdominal distension, crura edema and increased ALT. Conclusions: RUX combined with PTD for treatment of MF may modulate initial hematologic toxicity observed when RUX alone, and may increase response due to improved levels of hemoglobin or platelet.
Danazol ; Drug Combinations ; Humans ; Nitriles ; Pilot Projects ; Prednisone ; Primary Myelofibrosis/drug therapy* ; Pyrazoles/therapeutic use* ; Pyrimidines ; Thalidomide/therapeutic use* ; Treatment Outcome

To evaluate the efficacy and safety of interferon-alpha-2b (IFN-α-2b) in polycythemia vera patients(PV patient) with or without post-polycythemic myelofibrosis (post-PV MF), 30 patients with mutated JAK2V617F were enrolled in this study, from which 29 patients were evaluable. The percentage of mutated JAK2V617F allele (V617F%) was evaluated by real-time polymerase chain reaction (RT-PCR) before and after treatment with IFN-α-2b. The correlation of V617F allele burden with the major clinical outcomes was studied. Adverse effects appeared in patients was observed. The results showed that the median follow-up was 24 (12 - 42) months for 29 evaluable patients. Complete hematologic response was achieved in 10%, 48%, 72% and 78% of patients after treatment for 6, 12, 24 and 36 months respectively. The detection of V617F allele burden revealed that the molecular remission of patients (V617F%) was achieved in 41%, 76%, 89% and 89% after treatment for 6, 12, 24 and 36 months respectively. Molecular complete remission (JAK2V617F undetectable) was achieved in 4 patients, lasted from 6 to 12 months after IFN-α-2b discontinuation. The decrease of V617F% in patients with post-PV MF was significantly higher than that in patients without post-PV MF (53 ± 18% vs 32 ± 22%, respectively; p = 0.031) after treatment for 12 months. PV patients had a good tolerance to IFN-α-2b. It is concluded that IFN-α-2b can decrease the mutated V617F allele burden. Patients with PV, especially with post-PV MF, can achieve molecular remission after treatment with IFN-α-2b.
Adult ; Alleles ; Female ; Humans ; Interferon-alpha ; therapeutic use ; Janus Kinase 2 ; genetics ; Male ; Middle Aged ; Mutation ; Polycythemia Vera ; drug therapy ; genetics ; pathology ; Primary Myelofibrosis ; drug therapy ; genetics ; pathology ; Recombinant Proteins ; therapeutic use