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1.
Nursing ; 50(8): 22-30, 2020 Aug.
Artigo em Inglês | MEDLINE | ID: mdl-32618765

RESUMO

A group of rare hematologic cancers, myeloproliferative neoplasms (MPNs) evolve when bone marrow dysfunction causes overproduction of one or more blood cell types. This article explores the diagnosis, treatment, and nursing care of patients diagnosed with one of three classic MPNs: essential thrombocythemia, polycythemia vera, and primary myelofibrosis.


Assuntos
Policitemia Vera/enfermagem , Mielofibrose Primária/enfermagem , Trombocitemia Essencial/enfermagem , Humanos , Diagnóstico de Enfermagem
2.
Ann Hematol ; 99(7): 1441-1451, 2020 Jul.
Artigo em Inglês | MEDLINE | ID: mdl-32417942

RESUMO

Myelofibrosis (MF) is a chronic myeloproliferative neoplasm which can lead to massive splenomegaly secondary to extramedullary hematopoiesis. Patients frequently exhibit debilitating symptoms including pain and early satiety, in addition to cellular sequestration causing severe cytopenias. JAK 1/2 inhibitors, such as ruxolitinib and fedratinib, are the mainstay of therapy and produce significant and durable reductions in spleen volume. However, many patients are not eligible for JAK 2 inhibitor therapy or become refractory to treatment over time. Novel therapies are in development that can reduce the degree of splenomegaly for some of these patients. However, splenectomy, splenic irradiation, and partial splenic artery embolization remain valuable therapeutic options in select patients. In this review, we will discuss currently available pharmacologic therapies and describe promising drugs currently in development. We will also delve into the efficacy and safety concerns of splenectomy, splenic irradiation, and partial splenic artery embolization. Finally, we will propose a treatment algorithm to help guide clinicians in the management of symptomatic splenomegaly in patients with MF.


Assuntos
Mielofibrose Primária/complicações , Mielofibrose Primária/terapia , Esplenomegalia/etiologia , Esplenomegalia/terapia , Embolização Terapêutica/métodos , Humanos , Inibidores de Proteínas Quinases/uso terapêutico , Baço/irrigação sanguínea , Baço/patologia , Baço/cirurgia , Esplenectomia/métodos , Artéria Esplênica/patologia , Artéria Esplênica/cirurgia
3.
Gan To Kagaku Ryoho ; 47(2): 279-285, 2020 Feb.
Artigo em Inglês | MEDLINE | ID: mdl-32381964

RESUMO

A 63-year-old woman was referred to our department in 2015 because of anemia and thrombocytosis. MPL W515/K was positive, JAK-2V617F and CALR exon 9 were negative. Bone marrow(BM)biopsy led to a diagnosis of primary myelofibrosis (PMF)in the prefibrotic/early stage(Grade 1). BMbiopsy performed in 2016 showed overt fibrotic stage(Grade 2). She was classified according to the Dynamic International Prognostic Scoring System(DIPSS)as intermediate(Int)-Ⅱrisk. Ruxolitinib 10 mg daily was initiated. Ruxolitinib was suspended for hepatic dysfunction after the dose was increased to 15 mg. Subsequently, ruxolitinib was resumed at 10 mg. BM biopsy performed in 2017 showed progression of myelofibrosis(MF)to Grade 3. BM biopsy performed in 2018 showed improved to Grade 0-1, however, BM was fatty. Currently in 2019, she continues to be on ruxolitinib. Results of immunohistochemical staining of BM biopsy specimens for cytokines and CD34 suggested the role of cytokines in the pathogenesis of the PMF. It was speculated that ruxolitinib blocked the production of cytokines to ameliorate the MF and restore the hematopoietic function of the BM. Although the pathogenesis of the fatty marrow remained unclear, the possibility of involvement of ruxolitinib cannot be denied.


Assuntos
Mielofibrose Primária , Medula Óssea , Feminino , Fibrose , Humanos , Pessoa de Meia-Idade , Mielofibrose Primária/tratamento farmacológico , Pirazóis
6.
Ann Hematol ; 99(6): 1161-1176, 2020 Jun.
Artigo em Inglês | MEDLINE | ID: mdl-32333155

RESUMO

Ruxolitinib is a targeted drug to treat myelofibrosis (MF). Ruxolitinib has significant advantages in spleen reduction and increasing 5-year overall survival (OS), and ruxolitinib-based combinations might provide more benefits than ruxolitinib monotherapy. In this review, we focus on the data of ruxolitinib-based combinations therapies and treatment-related adverse events (AEs) and safety. We analyzed and summarized the data of ruxolitinib-based combinations. Ruxolitinib combined with prednisone + thalidomide + danazol (TPD), panobinostat, pracinostat, azacytidine, or hydroxyurea has well reduced spleen. Ruxolitinib combined with danazol or TPD had well therapies in improvement of hemoglobin (Hgb) and platelets (PLT). Most ruxolitinib-based combinations therapies showed a superior benefit on reduced treatment-related AEs than ruxolitinib monotherapy. Treatment-related AEs and dose modification affect the safety and tolerability of ruxolitinib-based combinations. Genetic testing before treatment is recommended. To provide better clinical guidance, comparisons of these randomized controlled trials with the trials of ruxolitinib alone are necessary. This review suggests that the clinical application of ruxolitinib-based combinations is worth waiting for.


Assuntos
Fatores Imunológicos/administração & dosagem , Mielofibrose Primária/tratamento farmacológico , Pirazóis/administração & dosagem , Ensaios Clínicos como Assunto/métodos , Quimioterapia Combinada , Humanos , Mielofibrose Primária/diagnóstico , Resultado do Tratamento
7.
Ann Hematol ; 99(5): 983-989, 2020 May.
Artigo em Inglês | MEDLINE | ID: mdl-32277273

RESUMO

Molecular detection of JAK2 mutation (V617F or exon 12) is included as a major diagnostic criterion for polycythemia vera (PV) by the WHO 2016 guidelines. JAK2 exon 12 mutations are seen in about 2-5% of JAK2V617F-negative cases of PV. Mutations in JAK2 cause constitutive activation of JAK-STAT pathway which results in variable phenotypes. PV patients with exon 12 mutations in JAK2 present characteristically with erythrocytosis. There are limited reports describing the spectrum of JAK2 exon12 mutations in myeloproliferative neoplasms (MPNs). Here, we describe the characteristics of a series of MPN patients with mutations in exon 12 of JAK2 of which two were novel variants associated with polycythemia. Interestingly, we noted two patients presenting as myelofibrosis having JAK2 exon 12 mutations.


Assuntos
Éxons , Janus Quinase 2/genética , Mutação de Sentido Incorreto , Policitemia Vera/genética , Mielofibrose Primária/genética , Adulto , Substituição de Aminoácidos , Feminino , Humanos , Masculino , Pessoa de Meia-Idade
8.
Medicine (Baltimore) ; 99(13): e19587, 2020 Mar.
Artigo em Inglês | MEDLINE | ID: mdl-32221077

RESUMO

We herein report a 76-year-old Japanese man with myelofibrosis who developed cryptococcal meningitis. After treatment for 5 months with ruxolitinib, the patient presented with fever and disturbance of consciousness. Marked nuchal stiffness was noted. The magnetic resonance imaging results of the brain were normal. Lumbar puncture showed an opening cerebrospinal fluid (CSF) pressure of 110 mm H2O, pleocytosis (85 mononuclear cells and 222 polymorphonuclear cells/µL), decreased CSF/serum glucose ratio (43%), and elevated protein (194 mg/dL). Blood and CSF cultures grew no bacteria or fungi. However, cryptococcal antigen was detected in the blood and CSF samples. We discontinued ruxolitinib and started administration of amphotericin B. His condition improved gradually 1 week after initiation of treatment. There have been only a few reports on cryptococcal meningitis associated with ruxolitinib. Physicians should consider the possibility of cryptococcal meningitis in patients receiving ruxolitinib.


Assuntos
Meningite Criptocócica/etiologia , Mielofibrose Primária/tratamento farmacológico , Pirazóis/efeitos adversos , Idoso , Anfotericina B/uso terapêutico , Humanos , Masculino , Pirazóis/uso terapêutico
9.
Am J Hematol ; 95(6): 594-603, 2020 06.
Artigo em Inglês | MEDLINE | ID: mdl-32129512

RESUMO

Fedratinib is an oral, selective Janus kinase 2 (JAK2) inhibitor. The phase II JAKARTA2 study assessed fedratinib in patients with intermediate- or high-risk myelofibrosis (MF) who were resistant or intolerant to prior ruxolitinib per investigator assessment. Patients received fedratinib 400 mg/day in 28-day cycles. The JAKARTA2 outcomes were initially reported using a last-observation-carried forward (LOCF) analysis in a "Per Protocol" population. This updated analysis of JAKARTA2 employs intention-to-treat analysis principles without LOCF for all treated patients (ITT Population; N = 97), and for a patient subgroup who met more stringent definitions of prior ruxolitinib failure (Stringent Criteria Cohort; n = 79). Median duration of prior ruxolitinib exposure was 10.7 months. The primary endpoint was spleen volume response rate (SVRR; ≥35% spleen volume decrease from baseline to end of cycle 6 [EOC6]). The SVRR was 31% in the ITT Population and 30% in the Stringent Criteria Cohort. Median duration of spleen volume response was not reached. Symptom response rate (≥50% reduction from baseline to EOC6 in total symptom score [TSS] on the modified Myelofibrosis Symptom Assessment Form [MFSAF]) was 27%. Grade 3-4 anemia and thrombocytopenia rates were 38% and 22%, respectively. Patients with advanced MF substantially pretreated with ruxolitinib attained robust spleen responses and reduced symptom burden with fedratinib.


Assuntos
Mielofibrose Primária/tratamento farmacológico , Mielofibrose Primária/patologia , Pirrolidinas/administração & dosagem , Baço/patologia , Sulfonamidas/administração & dosagem , Adulto , Idoso , Idoso de 80 Anos ou mais , Feminino , Humanos , Masculino , Pessoa de Meia-Idade , Tamanho do Órgão/efeitos dos fármacos , Pirazóis/administração & dosagem
10.
Ann Hematol ; 99(5): 1007-1016, 2020 May.
Artigo em Inglês | MEDLINE | ID: mdl-32157418

RESUMO

Controversy regarding the risk of non-hematologic malignancies in myelofibrosis patients still exists. We aimed to examine the association between myelofibrosis and non-hematologic malignancies. A cohort of 1,469,790 adults without a diagnosis of myelofibrosis was identified on 1 January 2007, from the electronic medical records of the largest healthcare provider in Israel. Participants were followed up until 31 December 2015, for the occurrence of myelofibrosis. All cases of myelofibrosis were adjudicated by reviewing patients' electronic medical files. Using risk set sampling, four randomly selected controls (without myelofibrosis) were matched to each case of myelofibrosis on age, sex, ethnicity, and index date. Patients with and without myelofibrosis were followed from the index date until 31 December 2016 for the occurrence of non-hematologic malignancies based on the data from the Israel National Cancer Registry. The study included 550 patients with myelofibrosis and 2200 matched controls. Non-hematologic cancers occurred in 35 patients with myelofibrosis and 138 patients without myelofibrosis, reflecting a crude incidence rate of 27.9 and 15.3 per 1000 person-years, respectively. Myelofibrosis was independently associated with increased risk of non-hematologic malignancies with propensity score adjusted HR of 1.85 (95% CI, 1.09-3.15). No significant association was detected between myelofibrosis and the specific sites of non-hematologic malignancies. Treatment with ruxolitinib was not significantly associated with non-hematologic malignancies HR 1.36 (0.60-3.11). In conclusion, myelofibrosis appears to be associated with increased risk of non-hematologic malignancies. However, this study raises concerns about surveillance bias, suggesting that the association might be attributed to earlier detection rather than real increased risk.


Assuntos
Registros Eletrônicos de Saúde , Neoplasias/epidemiologia , Mielofibrose Primária/epidemiologia , Sistema de Registros , Idoso , Feminino , Humanos , Incidência , Masculino , Pessoa de Meia-Idade , Estudos Retrospectivos , Fatores de Risco
11.
Pediatr Blood Cancer ; 67(5): e28232, 2020 05.
Artigo em Inglês | MEDLINE | ID: mdl-32134181

RESUMO

OBJECTIVES: Pediatric myelofibrosis is a rare entity with the largest reported series of 19 cases. We describe here the clinicopathological spectrum and outcomes of 15 cases of pediatric myelofibrosis. METHODS: Case files of myelofibrosis of patients less than 18 years were retrieved from January 2016 to January 2019, and patients with idiopathic myelofibrosis after exhaustive work-up were studied. Their clinicopathological profiles were studied and then followed up for resolution and malignant transformation. RESULTS: Of the 15 cases of idiopathic myelofibrosis, transfusion-dependent anemia (14/15) was most common presentation. Only one patient showed leukoerythroblastosis with dacryocytes. Myeloid hyperplasia was seen in 13 of 15 patients and megakaryocytic hyperplasia in 10 patients. Dysmegakaryopoiesis was seen in 8 of 15 patients, and only three had small loose megakaryocytic clustering. None showed hyperchromatic megakaryocytes, intrasinusoidal hematopoiesis, or osteosclerosis. One patient with trisomy 8 tested positive for JAK2V617F. Bone marrow biopsy was hypercellular in 13, and 8 had world health organization (WHO) MF-3 fibrosis. None of the patients developed malignancy, one had spontaneous resolution, and one patient required allogenic stem cell transplant. CONCLUSIONS: Pediatric myelofibrosis is a distinct entity from primary myelofibrosis in adults and merits mention in the WHO manual as a distinct entity.


Assuntos
Transformação Celular Neoplásica , Janus Quinase 2 , Mutação de Sentido Incorreto , Proteínas de Neoplasias , Trombopoese , Adolescente , Adulto , Substituição de Aminoácidos , Transformação Celular Neoplásica/genética , Transformação Celular Neoplásica/metabolismo , Transformação Celular Neoplásica/patologia , Criança , Pré-Escolar , Feminino , Humanos , Lactente , Janus Quinase 2/genética , Janus Quinase 2/metabolismo , Masculino , Megacariócitos/metabolismo , Megacariócitos/patologia , Proteínas de Neoplasias/genética , Proteínas de Neoplasias/metabolismo , Osteosclerose/genética , Osteosclerose/metabolismo , Osteosclerose/patologia , Mielofibrose Primária/genética , Mielofibrose Primária/metabolismo , Mielofibrose Primária/patologia , Estudos Retrospectivos
12.
Rinsho Ketsueki ; 61(2): 110-115, 2020.
Artigo em Japonês | MEDLINE | ID: mdl-32147609

RESUMO

Angioimmunoblastic T-cell lymphoma (AITL) is frequently associated with immunological abnormalities, such as hypergammaglobulinemia, autoimmune cytopenia, and the presence of various autoantibodies. Few reports on AITL have also described the development of myelofibrosis resulting from the invasion of lymphoma cells that produced various cytokines, including TGF-ß. Interestingly, recent studies demonstrated that autoimmunity can directly cause autoimmune myelofibrosis (AIMF). Usually, bone marrow fibrosis associated with AIMF is rapidly improved by treatment. Here, we describe our experience with a case of AITL complicated with the presence of numerous autoimmune abnormalities, including positive Coombs, anti-nuclear antibody, anti-ds-DNA antibody, anti-phospholipid antibody, and cold agglutinin tests. The patient presented with severe bone marrow fibrosis (MF-3) at the initial diagnosis. After two courses of the CHASE therapy, myelofibrosis rapidly disappeared, and the autoimmune abnormalities were ameliorated. These findings suggest that the bone marrow fibrosis observed in this case was partly attributable to an AIMF-like mechanism.


Assuntos
Linfadenopatia Imunoblástica , Linfoma de Células T , Mielofibrose Primária , Autoanticorpos , Fibrose , Humanos
13.
Ann Hematol ; 99(6): 1177-1191, 2020 Jun.
Artigo em Inglês | MEDLINE | ID: mdl-32198525

RESUMO

Myelofibrosis is a BCR-ABL1-negative myeloproliferative neoplasm characterized by anemia, progressive splenomegaly, extramedullary hematopoiesis, bone marrow fibrosis, constitutional symptoms, leukemic progression, and shortened survival. Constitutive activation of the Janus kinase/signal transducers and activators of transcription (JAK-STAT) pathway, and other cellular pathways downstream, leads to myeloproliferation, proinflammatory cytokine expression, and bone marrow remodeling. Transplant is the only curative option for myelofibrosis, but high rates of morbidity and mortality limit eligibility. Several prognostic models have been developed to facilitate treatment decisions. Until the recent approval of fedratinib, a JAK2 inhibitor, ruxolitinib was the only available JAK inhibitor for treatment of intermediate- or high-risk myelofibrosis. Ruxolitinib reduces splenomegaly to some degree in almost all treated patients; however, many patients cannot tolerate ruxolitinib due to dose-dependent drug-related cytopenias, and even patients with a good initial response often develop resistance to ruxolitinib after 2-3 years of therapy. Currently, there is no consensus definition of ruxolitinib failure. Until fedratinib approval, strategies to overcome ruxolitinib resistance or intolerance were mainly different approaches to continued ruxolitinib therapy, including dosing modifications and ruxolitinib rechallenge. Fedratinib and two other JAK2 inhibitors in later stages of clinical development, pacritinib and momelotinib, have been shown to induce clinical responses and improve symptoms in patients previously treated with ruxolitinib. Fedratinib induces robust spleen responses, and pacritinib and momelotinib may have preferential activity in patients with severe cytopenias. Reviewed here are strategies to ameliorate ruxolitinib resistance or intolerance, and outcomes of clinical trials in patients with myelofibrosis receiving second-line JAK inhibitors after ruxolitinib treatment.


Assuntos
Gerenciamento Clínico , Janus Quinases/antagonistas & inibidores , Mielofibrose Primária/tratamento farmacológico , Pirazóis/uso terapêutico , Pirrolidinas/uso terapêutico , Sulfonamidas/uso terapêutico , Ensaios Clínicos como Assunto/métodos , Humanos , Mielofibrose Primária/diagnóstico , Falha de Tratamento
14.
Rinsho Ketsueki ; 61(1): 3-10, 2020.
Artigo em Japonês | MEDLINE | ID: mdl-32023599

RESUMO

Recently, monocyte-derived fibroblast-like cells, called fibrocytes, garnered attention as involved in the novel pathogenesis of various fibrotic diseases. They also play a role in the induction of myelofibrosis (MF). Neoplastic fibrocytes are overrepresented in the bone marrow of patients with primary MF, and the suppression of fibrocyte differentiation by serum amyloid P has been shown to remarkably improve MF. Further, thrombopoietin (TPO) or a TPO receptor agonist directly induces fibrocyte differentiation, and fibrocyte elimination reversed the MF phenotype in a murine model. Human fibrocytes highly express signaling lymphocytic activation molecule-F7 (SLAMF7) compared with macrophages. Myeloproliferative neoplasm (MPN) patients harboring JAK2V617F with MF had a significantly elevated SLAMF7high monocyte percentage, which correlated positively with the JAK2V617F allele burden. Furthermore, the JAK2V617F allele burden and the tendency to differentiate into fibrocytes of SLAMF7high monocytes was significantly higher than that of JAK2V617Flow monocytes and could be a potential target of elotuzumab (Elo), an anti-SLAMF7 antibody used to treat multiple myeloma. Elo independently inhibited the differentiation of fibrocytes derived not only from healthy controls but also from MF patients in vitro. Elo also ameliorated MF and splenomegaly induced by romiplostim administration in humanized NOG mice. Thus, Elo could be a therapeutic agent for MPN patients harboring JAK2V617F with MF.


Assuntos
Transtornos Mieloproliferativos , Mielofibrose Primária , Animais , Medula Óssea , Humanos , Macrófagos , Camundongos , Monócitos
15.
Ann Hematol ; 99(4): 781-789, 2020 Apr.
Artigo em Inglês | MEDLINE | ID: mdl-32076825

RESUMO

Pulmonary hypertension (PH) has been described in myelofibrosis (MF), but it is rare and typically found in advanced disease. Although the etiology of PH in MF is unclear, early predictors may be detected by echocardiogram. The goals of our study were to evaluate the prevalence of PH as determined by echocardiography in a cohort of MF patients and to identify clinical risk factors for PH. We performed a retrospective review of MF patients from October 2015 to May 2017 at MD Anderson Cancer Center in the ambulatory clinic, and those with echocardiogram were included. Clinical, echocardiographic, and laboratory data were reviewed. Patients with and without PH were compared using a chi-square or Fisher's exact test, and logistic regression was performed with an outcome variable of PH. There were 143 patients with MF who underwent echocardiogram, and 20 (14%) had echocardiographic findings consistent with PH. Older age, male gender, hypertension, hyperlipidemia, coronary artery disease, dyspnea, hematocrit, brain natriuretic peptide (BNP), and N-terminal prohormone BNP (NT-proBNP) were significantly different between those without PH and those with PH (p < 0.05). Female gender was protective (OR 0.21, 95% CI 0.049-0.90, p = 0.035), and NT-proBNP was a significant clinical predictor of PH (OR 1.07, CI 1.02 = 1.12, p = 0.006). PH in MF is lower than previously reported in our MF cohort, but many patients had cardiac comorbidities. PH due to left-sided heart disease may be underestimated in MF. Evaluation of respiratory symptoms and elevated NT-proBNP should prompt a baseline echocardiogram. Early detection of PH with a multidisciplinary approach may allow treatment of reversible etiologies.


Assuntos
Hipertensão Pulmonar/etiologia , Mielofibrose Primária/complicações , Adolescente , Adulto , Idoso , Idoso de 80 Anos ou mais , Comorbidade , Doença das Coronárias/epidemiologia , Dispneia/epidemiologia , Ecocardiografia , Feminino , Humanos , Hiperlipidemias/epidemiologia , Hipertensão/epidemiologia , Hipertensão Pulmonar/sangue , Hipertensão Pulmonar/diagnóstico por imagem , Hipertensão Pulmonar/epidemiologia , Masculino , Pessoa de Meia-Idade , Peptídeo Natriurético Encefálico/sangue , Fragmentos de Peptídeos/sangue , Prevalência , Estudos Retrospectivos , Adulto Jovem
16.
Ann Hematol ; 99(4): 791-798, 2020 Apr.
Artigo em Inglês | MEDLINE | ID: mdl-32086587

RESUMO

Patients with polycythemia vera (PV) or essential thrombocythemia (ET) presenting with splanchnic vein thrombosis (SVT) might have a specific clinico-biological profile. To investigate this hypothesis, 3705 PV/ET patients from three national registers, 118 of them presenting with SVT, were reviewed. After correction for age and sex, PV/ET patients with SVT showed an increased risk of death (HR 2.47, 95% CI 1.5-4.01, p < 0.001), venous thrombosis (IRR 3.4, 95%CI 2.1-5.5, p < 0.001), major bleeding (IRR 3.6, 95%CI 2.3-5.5, p < 0.001), and second cancer (IRR 2.37, 95%CI 1.4-4.1, p = 0.002). No case of acute leukemia was documented among patients with PV/ET presenting with SVT and seven of them (6%) progressed to myelofibrosis. SVT was not associated with lower risk of MF after correction by age and sex. Patients with SVT more frequently died from complications related to hepatic disease, major bleeding, or second cancer, resulting in a 5-year reduction of age- and sex-adjusted median survival. In conclusion, PV and ET patients presenting with SVT have shorter survival than patients with PV and ET of the same age and sex. This excess mortality is related to liver disease, major bleeding, and second cancer rather than to the natural evolution of the MPN.


Assuntos
Policitemia Vera/complicações , Circulação Esplâncnica , Trombocitemia Essencial/complicações , Trombose Venosa/etiologia , Adulto , Progressão da Doença , Feminino , Seguimentos , Hemorragia/epidemiologia , Humanos , Estimativa de Kaplan-Meier , Hepatopatias/epidemiologia , Masculino , Veias Mesentéricas , Pessoa de Meia-Idade , Segunda Neoplasia Primária/epidemiologia , Veia Porta , Mielofibrose Primária/etiologia , Modelos de Riscos Proporcionais , Sistema de Registros , Risco , Espanha/epidemiologia , Veia Esplênica
18.
Cytogenet Genome Res ; 160(1): 18-21, 2020.
Artigo em Inglês | MEDLINE | ID: mdl-32008001

RESUMO

Multiple isodicentric Y chromosomes [idic(Y)] is a rare cytogenetic abnormality, most exclusively described in constitutional karyotypes. Only recently has this entity been reported in hematologic neoplasms such as myeloid disorders, albeit these cases remain very scarce. The possible involvement of increasing copies of potential proto-oncogenes located on the multiple idic(Y) led to consider one of them, CRLF2, as a target for kinase inhibitors. We report here, to our knowledge, the first case of multiple idic(Y) in a patient with myelofibrosis secondary to essential thrombocythemia. The patient received ruxolitinib therapy with initial good clinical response.


Assuntos
Cromossomos Humanos Y/genética , Mielofibrose Primária/complicações , Aberrações dos Cromossomos Sexuais , Trombocitemia Essencial/complicações , Idoso , Alelos , Humanos , Hibridização in Situ Fluorescente , Cariótipo , Cariotipagem , Masculino , Mosaicismo , Contagem de Plaquetas , Mielofibrose Primária/genética , Prognóstico , Pirazóis/uso terapêutico , Receptores de Citocinas/genética , Trombocitemia Essencial/genética
19.
J Bone Miner Metab ; 38(2): 145-150, 2020 Mar.
Artigo em Inglês | MEDLINE | ID: mdl-31897749

RESUMO

The bone marrow (BM) is located inside the bone. Now, it appears that bone tissue functionally communicates with the BM hematopoietic system. Osteoblast lineage cells serve as a part of the microenvironment for immature hematopoietic (stem/progenitor) cells. In contrast, mature hematopoietic cells such as neutrophils and macrophages play a critical role to regulate osteoblast activity. A progressive distortion of this precise inter-organ communication between hematopoietic and skeletal systems may lead to hematologic disorders. Recent studies have revealed that vitamin D receptor is a pivotal bridging molecule for this network and for the pathogenesis of myelofibrosis.


Assuntos
Osso e Ossos/fisiologia , Hematologia , Animais , Osso e Ossos/metabolismo , Hematopoese , Células-Tronco Hematopoéticas , Humanos , Mielofibrose Primária/metabolismo , Receptores de Calcitriol/metabolismo
20.
Drugs ; 80(1): 85-90, 2020 Jan.
Artigo em Inglês | MEDLINE | ID: mdl-31939073

RESUMO

Luspatercept (REBLOZYL®) is an erythroid maturation agent developed by Acceleron Pharma and Celgene Corporation for the treatment of anaemia associated with myelodysplastic syndromes, myelofibrosis and beta-thalassaemia. Based primarily on the results of the phase III BELIEVE trial, subcutaneous luspatercept was recently approved in the USA for the treatment of anaemia associated with beta-thalassaemia. This article summarizes the milestones in the development of luspatercept leading to this first approval.


Assuntos
Receptores de Activinas Tipo II/uso terapêutico , Anemia/tratamento farmacológico , Hematínicos/uso terapêutico , Fragmentos Fc das Imunoglobulinas/uso terapêutico , Proteínas Recombinantes de Fusão/uso terapêutico , Anemia/etiologia , Animais , Aprovação de Drogas , Humanos , Síndromes Mielodisplásicas/complicações , Síndromes Mielodisplásicas/tratamento farmacológico , Mielofibrose Primária/complicações , Mielofibrose Primária/tratamento farmacológico , Talassemia beta/complicações , Talassemia beta/tratamento farmacológico
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