{"title":"使用临床、突变和移植特异性资料选择原发性骨髓纤维化患者进行干细胞移植","authors":"Nico Gagelmann, Nicolaus Kröger","doi":"10.1002/acg2.80","DOIUrl":null,"url":null,"abstract":"<p>As with other hematologic malignancies, the management of patients with MF has very much entered the molecular era. The prognostic impact of several driver and nondriver mutations is now well-established; this has obvious relevance to both patient selection for allogeneic hematopoietic cell transplantation and posttransplant outcomes. For transplant, present <i>JAK2</i> or triple-negative driver mutation genotype together with present <i>ASXL1</i> mutation may provide best utility for counseling patients with respect to posttransplant outcome, together with clinical (performance status, age, and leukocyte and platelet counts) and transplant-related factors (donor relation). Different conditioning intensities do not seem to play a role with regards to outcome posttransplant but still need to be compared in the molecular era. While ruxolitinib provides clinical benefits for patients before transplant, more studies on the finetuning and integration of ruxolitinib into the transplant algorithm are needed. In conclusion, as clinically important risk stratification has been achieved throughout the disease course and new targeted agents become increasingly available, a collaborative and integrative approach is needed to translate new biological insights to personalized/tailored and timed therapy for patients with myelofibrosis.</p>","PeriodicalId":72084,"journal":{"name":"Advances in cell and gene therapy","volume":"3 2","pages":""},"PeriodicalIF":0.0000,"publicationDate":"2020-01-29","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://sci-hub-pdf.com/10.1002/acg2.80","citationCount":"0","resultStr":"{\"title\":\"Selecting patients with primary myelofibrosis for stem cell transplant using clinical, mutational, and transplant-specific profiles\",\"authors\":\"Nico Gagelmann, Nicolaus Kröger\",\"doi\":\"10.1002/acg2.80\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"<p>As with other hematologic malignancies, the management of patients with MF has very much entered the molecular era. The prognostic impact of several driver and nondriver mutations is now well-established; this has obvious relevance to both patient selection for allogeneic hematopoietic cell transplantation and posttransplant outcomes. For transplant, present <i>JAK2</i> or triple-negative driver mutation genotype together with present <i>ASXL1</i> mutation may provide best utility for counseling patients with respect to posttransplant outcome, together with clinical (performance status, age, and leukocyte and platelet counts) and transplant-related factors (donor relation). Different conditioning intensities do not seem to play a role with regards to outcome posttransplant but still need to be compared in the molecular era. While ruxolitinib provides clinical benefits for patients before transplant, more studies on the finetuning and integration of ruxolitinib into the transplant algorithm are needed. In conclusion, as clinically important risk stratification has been achieved throughout the disease course and new targeted agents become increasingly available, a collaborative and integrative approach is needed to translate new biological insights to personalized/tailored and timed therapy for patients with myelofibrosis.</p>\",\"PeriodicalId\":72084,\"journal\":{\"name\":\"Advances in cell and gene therapy\",\"volume\":\"3 2\",\"pages\":\"\"},\"PeriodicalIF\":0.0000,\"publicationDate\":\"2020-01-29\",\"publicationTypes\":\"Journal Article\",\"fieldsOfStudy\":null,\"isOpenAccess\":false,\"openAccessPdf\":\"https://sci-hub-pdf.com/10.1002/acg2.80\",\"citationCount\":\"0\",\"resultStr\":null,\"platform\":\"Semanticscholar\",\"paperid\":null,\"PeriodicalName\":\"Advances in cell and gene therapy\",\"FirstCategoryId\":\"1085\",\"ListUrlMain\":\"https://onlinelibrary.wiley.com/doi/10.1002/acg2.80\",\"RegionNum\":0,\"RegionCategory\":null,\"ArticlePicture\":[],\"TitleCN\":null,\"AbstractTextCN\":null,\"PMCID\":null,\"EPubDate\":\"\",\"PubModel\":\"\",\"JCR\":\"\",\"JCRName\":\"\",\"Score\":null,\"Total\":0}","platform":"Semanticscholar","paperid":null,"PeriodicalName":"Advances in cell and gene therapy","FirstCategoryId":"1085","ListUrlMain":"https://onlinelibrary.wiley.com/doi/10.1002/acg2.80","RegionNum":0,"RegionCategory":null,"ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"","JCRName":"","Score":null,"Total":0}
Selecting patients with primary myelofibrosis for stem cell transplant using clinical, mutational, and transplant-specific profiles
As with other hematologic malignancies, the management of patients with MF has very much entered the molecular era. The prognostic impact of several driver and nondriver mutations is now well-established; this has obvious relevance to both patient selection for allogeneic hematopoietic cell transplantation and posttransplant outcomes. For transplant, present JAK2 or triple-negative driver mutation genotype together with present ASXL1 mutation may provide best utility for counseling patients with respect to posttransplant outcome, together with clinical (performance status, age, and leukocyte and platelet counts) and transplant-related factors (donor relation). Different conditioning intensities do not seem to play a role with regards to outcome posttransplant but still need to be compared in the molecular era. While ruxolitinib provides clinical benefits for patients before transplant, more studies on the finetuning and integration of ruxolitinib into the transplant algorithm are needed. In conclusion, as clinically important risk stratification has been achieved throughout the disease course and new targeted agents become increasingly available, a collaborative and integrative approach is needed to translate new biological insights to personalized/tailored and timed therapy for patients with myelofibrosis.