Laurenti Luca, Gaidano Gianluca, Mauro Francesca Romana, Molica Stefano, Pasqualetti Patrizio, Scarfò Lydia, Ghia Paolo
Fondazione Policlinico Universitario A. Gemelli IRCCS, Roma, Italy.
Division of Hematology, Department of Translational Medicine, University of Eastern Piedmont, Novara, Italy.
Hemasphere. 2022 Aug 31;6(9):e771. doi: 10.1097/HS9.0000000000000771. eCollection 2022 Sep.
Several treatment options are available for chronic lymphocytic leukemia (CLL) and, for this reason, treatment choice can result challenging after introducing oral targeted agents. This study aims at comparing patients' and hematologists' preferences for attributes of CLL treatments. An online cross-sectional survey has been delivered to clinicians and patients affected by CLL in Italy. A discrete choice experiment has been conducted so to estimate each attribute's relative importance (RI) and assess the preference weight for each level of each attribute. An expert panel agreed on investigating the following attributes: progression-free survival (PFS) and measurable residual disease, route of administration/therapy duration and follow-up frequency, incidence of diarrhea (episodes/day), serious infections (grade 3 or 4), and atrial fibrillation. Overall, 746 patients and 109 clinicians accessed the survey, and 215 and 69, respectively, filled it in. The most important attributes were PFS (RI 30%) for hematologists and the risk of severe infections (RI 24%) for patients. Clinicians rated preference for maximum efficacy and lowest risk of severe infection very high (30%). Both patients and clinicians preferred oral administration while considering duration of therapy less relevant. The frequency of hospital appointments was negligible for patients, while clinicians preferred a quarterly frequency. Considering all attributes, diarrhea was weighted more by clinicians than by patients. Atrial fibrillation was not relevant for clinicians, while it was not negligible for patients. In conclusion, clinicians and patients favor an oral therapy, including continuous treatment, if associated with prolonged PFS, albeit with particular attention to the risk of serious infections.
慢性淋巴细胞白血病(CLL)有多种治疗选择,因此,在引入口服靶向药物后,治疗选择可能具有挑战性。本研究旨在比较患者和血液科医生对CLL治疗属性的偏好。已在意大利对受CLL影响的临床医生和患者进行了一项在线横断面调查。进行了一项离散选择实验,以估计每个属性的相对重要性(RI),并评估每个属性每个水平的偏好权重。一个专家小组商定调查以下属性:无进展生存期(PFS)和可测量的残留疾病、给药途径/治疗持续时间和随访频率、腹泻发生率(发作次数/天)、严重感染(3级或4级)和心房颤动。总体而言,746名患者和109名临床医生访问了该调查,分别有215名患者和69名临床医生填写了问卷。最重要的属性对血液科医生来说是PFS(RI 30%),对患者来说是严重感染风险(RI 24%)。临床医生对最大疗效和最低严重感染风险的偏好评分非常高(30%)。患者和临床医生都更喜欢口服给药,同时认为治疗持续时间不太重要。患者对医院预约频率的关注度可忽略不计,而临床医生更喜欢每季度一次的频率。考虑所有属性,临床医生对腹泻的权重高于患者。心房颤动对临床医生无关紧要,而对患者来说并非可忽略不计。总之,临床医生和患者都倾向于口服治疗,包括持续治疗,如果与延长的PFS相关,尽管要特别注意严重感染的风险。