Savona Michael R, Malcovati Luca, Komrokji Rami, Tiu Ramon V, Mughal Tariq I, Orazi Attilio, Kiladjian Jean-Jacques, Padron Eric, Solary Eric, Tibes Raoul, Itzykson Raphael, Cazzola Mario, Mesa Ruben, Maciejewski Jaroslaw, Fenaux Pierre, Garcia-Manero Guillermo, Gerds Aaron, Sanz Guillermo, Niemeyer Charlotte M, Cervantes Francisco, Germing Ulrich, Cross Nicholas C P, List Alan F
Vanderbilt-Ingram Cancer Center/Vanderbilt University Medical Center, TN;
University of Pavia and Fondazione IRCCS Policlinico San Matteo, Pavia, Italy;
Blood. 2015 Mar 19;125(12):1857-65. doi: 10.1182/blood-2014-10-607341. Epub 2015 Jan 26.
Myelodysplastic syndromes (MDS) and myeloproliferative neoplasms (MPN) are hematologically diverse stem cell malignancies sharing phenotypic features of both myelodysplastic syndromes and myeloproliferative neoplasms. There are currently no standard treatment recommendations for most adult patients with MDS/MPN. To optimize efforts to improve the management and disease outcomes, it is essential to identify meaningful clinical and biologic end points and standardized response criteria for clinical trials. The dual dysplastic and proliferative features in these stem cell malignancies define their uniqueness and challenges. We propose response assessment guidelines to harmonize future clinical trials with the principal objective of establishing suitable treatment algorithms. An international panel comprising laboratory and clinical experts in MDS/MPN was established involving 3 independent academic MDS/MPN workshops (March 2013, December 2013, and June 2014). These recommendations are the result of this collaborative project sponsored by the MDS Foundation.
骨髓增生异常综合征(MDS)和骨髓增殖性肿瘤(MPN)是血液学上不同的干细胞恶性肿瘤,兼具骨髓增生异常综合征和骨髓增殖性肿瘤的表型特征。目前,大多数成年MDS/MPN患者尚无标准治疗推荐。为优化改善管理和疾病预后的工作,确定有意义的临床和生物学终点以及临床试验的标准化反应标准至关重要。这些干细胞恶性肿瘤中的双重发育异常和增殖特征决定了它们的独特性和挑战性。我们提出反应评估指南,以使未来的临床试验协调一致,主要目标是建立合适的治疗算法。成立了一个由MDS/MPN实验室和临床专家组成的国际小组,该小组参与了3次独立的学术MDS/MPN研讨会(2013年3月、2013年12月和2014年6月)。这些建议是由MDS基金会赞助的这个合作项目的成果。