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骨髓增生异常综合征和骨髓增生异常综合征/骨髓增殖性肿瘤重叠综合征患者报告的结局:与时俱进登上舞台。

Patient-Reported Outcomes in Myelodysplastic Syndromes and MDS/MPN Overlap Syndromes: Stepping Onto the Stage with Changing Times.

作者信息

Patel Sagar S, Gerds Aaron T

机构信息

Leukemia and Myeloid Disorders Program, Department of Hematology and Medical Oncology, Taussig Cancer Institute, Cleveland Clinic, 9500 Euclid Ave, CA-60, Cleveland, OH, 44120, USA.

出版信息

Curr Hematol Malig Rep. 2017 Oct;12(5):455-460. doi: 10.1007/s11899-017-0406-x.

Abstract

Quality of life (QOL) and symptom burden are important measures captured by patient-reported outcomes (PROs). Myelodysplastic and myelodysplastic/myeloproliferative (MDS/MPN) neoplasm overlap syndromes are notable for significant morbidity and mortality, including a wide spectrum of physical and psychosocial effects. Thus, the development and application of PROs can provide meaningful information to facilitate communication and assist in follow up care. Disease-specific measures can more accurately reflect the full breadth of functional restrictions and symptoms. While traditional endpoints include remission, relapse, and survival rates, adoption of PROs in myeloid neoplasm clinical trials can facilitate drug approval. Integration of PROs in myeloid neoplasms is an important measure to capture QOL and symptoms, which can improve disease management.

摘要

生活质量(QOL)和症状负担是患者报告结局(PROs)所涵盖的重要指标。骨髓增生异常综合征和骨髓增生异常/骨髓增殖性(MDS/MPN)肿瘤重叠综合征以显著的发病率和死亡率为特征,包括广泛的身体和心理社会影响。因此,PROs的开发和应用可以提供有意义的信息,以促进沟通并协助后续护理。疾病特异性指标可以更准确地反映功能限制和症状的全貌。虽然传统的终点包括缓解率、复发率和生存率,但在髓系肿瘤临床试验中采用PROs可以促进药物获批。将PROs纳入髓系肿瘤是一项捕捉QOL和症状的重要措施,可改善疾病管理。

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