UCSF Benioff Children's Hospital, San Francisco, California, USA.
Columbia University Medical Center, New York, New York, USA.
Pediatr Blood Cancer. 2021 Jun;68(6):e29023. doi: 10.1002/pbc.29023. Epub 2021 Mar 25.
The decision to initiate second-line treatment in children with immune thrombocytopenia (ITP) is complex and involves many different factors.
In this prospective, observational, longitudinal cohort study of 120 children from 21 centers, the factors contributing to the decision to start second-line treatments for ITP were captured. At study entry, clinicians were given a curated list of 12 potential reasons the patient required a second-line treatment. Clinicians selected all that applied and ranked the top three reasons.
Quality of life (QOL) was the most frequently cited reason for starting a second-line therapy. Clinicians chose it as a reason to treat in 88/120 (73%) patients, as among the top three reasons in 68/120 (57%), and as the top reason in 32/120 (27%). Additional factors ranked as the top reason to start second-line treatment included severity of bleeding (22/120, 18%), frequency of bleeding (19/120, 16%), and severity of thrombocytopenia (18/120, 15%). Patients for whom QOL (p = .006) or sports participation (p = .02) were ranked reasons were more likely to have chronic ITP, whereas those for whom severity (p = .003) or frequency (p = .005) of bleeding were ranked reasons were more likely to have newly diagnosed or persistent ITP. Parental anxiety, though rarely the primary impetus for treatment, was frequently cited (70/120, 58%) as a contributing factor.
Perceived QOL is the most frequently selected reason pediatric patients start second-line therapies for ITP. It is critical that studies of treatments for childhood ITP include assessments of their effects on QOL.
决定启动儿童免疫性血小板减少症 (ITP) 的二线治疗较为复杂，涉及许多不同的因素。
在这项针对 21 个中心的 120 名儿童进行的前瞻性、观察性、纵向队列研究中，记录了决定启动 ITP 二线治疗的因素。在研究开始时，临床医生获得了一份经过精心整理的 12 种患者可能需要二线治疗的潜在原因列表。临床医生选择所有适用的原因，并对前三个原因进行排名。
生活质量 (QOL) 是启动二线治疗最常被提及的原因。88/120（73%）名患者的临床医生选择它作为治疗的原因，68/120（57%）名患者将其列为前三个原因之一，32/120（27%）名患者将其列为首要原因。排名靠前的启动二线治疗的其他因素包括出血严重程度（22/120，18%）、出血频率（19/120，16%）和血小板减少严重程度（18/120，15%）。将 QOL（p=.006）或运动参与（p=.02）列为原因的患者更有可能患有慢性 ITP，而将出血严重程度（p=.003）或出血频率（p=.005）列为原因的患者更有可能患有新诊断或持续性 ITP。尽管父母焦虑很少成为治疗的主要动力，但它经常被提及（70/120，58%），作为一个促成因素。
感知到的 QOL 是儿科患者启动 ITP 二线治疗的最常选择的原因。对儿童 ITP 治疗的研究必须包括对其对 QOL 的影响的评估。
