615例Ph染色体／BCR—ABL融合基因阴性骨髓增殖性肿瘤患者的症状负荷评估

The assessment of symptomatic burden among Ph/BCR-ABL negative myeloproliferative neoplasm patients

目的评估Ph染色体和BCR—ABL阴性骨髓增殖性肿瘤（MPN）患者体质性症状负荷。方法用骨髓增殖性肿瘤总症状评估量表（MPN—SAF．TSS）对628例MPN患者进行现场调查。结果疲劳在真性红细胞增多症（PV）、原发性血小板增多症（ET）和原发性骨髓纤维化（PMF）患者中的症状负荷和发生率均最高[分别为（3．46±2．97）、（3．47±2．99）、（4．74±3．04）分和76．O％、76．2％、89．9％]。患者的总症状负荷由高到低依次为PMF、PV、ET[分别为（28．9±19．1）、（19．2±16．8）、（17．1±15．3）分]，PMF与Pv、ET比较差异均有统计学意义（x2=6．371，P=-0．021；x2=14．020，P〈0．001），PV与ET之间差异无统计学意义（x2=2．281，P=0．191）。结论MPN-SAF-TSS评估量表可有效评估MPN患者体质性症状，PV、ET和PMF患者中PMF的总症状负荷最高，用MPN—SAF—TSS评估量表进行症状评估应列为MPN治疗研究的评价指标。

Objective To investigate the value of myeloproliferative neoplasms Symptom Assessment Form total symptom score （MPN-SAF-TSS） in assessing constitutional symptoms among Ph/ BCR-ABL negative myeloproliferative neoplasm （MPN） patients. Methods A cohort of 628 MPN patients were evaluated by MPN-SAF-TSS. Results Fatigue was the most common symptom （76.0%, 76.2% vs 89.9%） and the highest average severity of all the symptoms （3.46±2.97, 3.47±2.99 vs 4.74±3.04 scores） among polycythemia vera （PV）, essential thrombocythemia （ET） and primary myelofibrosis （PMF） patients. Using the MPN-SAF-TSS analysis, PMF patients showed highest burden of symptoms （28.9 ± 19.1）, followed by PV patients （19.2 ± 16.8）, and finally ET patients （17.1 ± 15.3）. Instinct differences were observed between PMF and PV patients （x2=6.371,p=0.021 ）, PMF and ET patients （Z2= 14.020, P〈0.001 ）. No significant difference was found between PV and ET patients （Z2=2.281, P=-0.191 ）. Conclusion MPN-SAF-TSS was effective in evaluating the symptomatic burden among Ph/BCRABL negative MPN patients and could be used for serial assessment in this clinical setting.