一项中国对骨髓增殖性肿瘤的影响和治疗目标认识的Landmark调查结果分析

Analysis of the MPN Landmark survey for impact and perceptions of treatment goals among Chinese physicians and myeloproliferative neoplasm patients

目的:调查我国医师和患者对Ph阴性骨髓增殖性肿瘤(MPN)症状负荷、治疗目标和疾病管理的理解,探寻医师和患者对疾病治疗沟通的差异,观察MPN疾病负荷对患者生活的影响。方法:采用国际MPN Landmark调查问卷,于2018年9月至11月进行现场调查。结果:共100名医师和298例患者[原发性血小板增多症(ET)122例,真性红细胞增多症(PV)116例,骨髓纤维化(MF)60例]参与问卷调查。90%的医师认为他们会根据预后风险评估对患者进行危度分组,然而只有67%的患者知道自己的预后分组。MF患者最常见的症状是疲劳/乏力(63%)、活动力不佳(48%)和腹部不适(47%);PV患者最常见的症状是头晕/眩晕(63%)、面部潮红(54%)和活动力不佳(48%);ET患者最常见的症状是头晕/眩晕(58%)、活动力不佳(49%)和疲劳/乏力(40%)。与PV(46%)和ET(52%)患者相比,MF患者情绪负担大的比例最高(62%)。分别有19%和30%的患者认为MPN严重影响了工作或日常活动的能力。47%的MPN患者有时或经常需要被看护,最近7 d看护人员的平均帮助时间为14.4 h。25%的患者认为疾病没有导致经济困难,66%的患者认为在不同程度上因病致贫,9%的患者认为疾病明显致贫。在诊断时,医师对19%的MF、23%的PV和26%的ET患者建议观察,而不推荐药物治疗。MF的治疗目标医师和患者基本一致,认为MF的主要治疗目标除了治愈以外,为改善症状、提高生活质量和延缓疾病进展。PV和ET患者认为他们的主要治疗目标除了治愈以外为症状改善(分别为82%和80%),这与医师的首要目标为预防血管/血栓事件(分别为53%和70%)是不一致的。从医师的角度看,对医患沟通的满意度为88%;从患者的角度看,对医患沟通的满意度为89%。18%的患者经常认为自己的状况比医师意识到的要差得多。结论:高症状负荷明显影响MPN患者的生活质量,导致沉重的情绪负担、降低工作效率、导致经济困难,给MPN患者的治疗尚需提供额外的支持来应对MPN带来的情绪影响。医师和患者在治疗目标上存在不一致性,应采用更积极开放的沟通以使患者和医师能够在治疗目标和疾病管理上达成一致。

Objective To assess patient and physician understanding of symptom burden,treatment goal,disease management,and perceptions as well as to identify potential disparities pertaining to the understanding of patients and physicians about them to enable patient-physician alignment on optimal disease management plans that best address patient needs.Methods The MPN landmark survey was conducted in China and was a cross-sectional survey of patients diagnosed with Ph-negative MPN and physicians treating patients with Ph-negative MPN from August 2018 to November 2018.Results Total 100 physicians and 298 patients(ET 122,PV 116,and MF 60)participated in the survey;90%of the physicians classified their patients as per a prognostic risk score;however,only 67%patients knew their score.The most common symptoms reported by the MF patients to the physicians as per both,frequency and severity were fatigue/tiredness(63%),inactivity(48%),and abdominal discomfort(47%).The most common symptoms of PV patients were dizziness/vertigo/light-headedness(63%),facial flushing(54%),and inactivity(48%).The most common symptoms of ET patients were dizziness/vertigo/light-headedness(58%),inactivity(49%),and fatigue/tiredness(40%).Patients with MF(62%)showed larger emotional burden then those with PV(46%)and ET(52%).Further,19%and 30%patients reported that MPN has severely affected their productivity while working and performing daily activities,respectively.There was a need for caregivers,sometimes or often,among 47%of patients in China.Total 25%patients considered that the condition has not caused any financial hardship,66%patients considered that the condition had caused some financial hardship,9%patients considered that the condition had caused great financial hardship.At diagnosis,approximately 19%MF,23%PV,and 26%ET patients were observed by the physicians instead of being prescribed a drug treatment.MF patients were in alignment with physicians on major treatment goals other than cure,including better QOL,symptom reduction,and slow/delayed disease progression.However,PV(82%)and ET(80%)patients had a different interaction with physicians;they considered first treatment goals other than cure was symptom improvement.Satisfaction with patient communication was reported by 88%of the physicians;satisfaction with physician communication was reported by 89%of the patients.Further,18%patients admitted to feeling a lot worse than their physician was aware of,indicating the need for better patient-physician communication.Conclusions MPN patients with high symptom burden have a low QOL and large emotional burden that severely affects productivity while working and causes financial hardship.This study highlighted the need for provided patients with additional support for managing emotional health.More open communication would enable patient-physician alignment on the treatment goals and optimal disease management plans that best address patient needs.