Dana-Farber/Boston Children's Cancer and Blood Disorder Center, Boston, Massachusetts.
Texas Children's Hematology Center, Baylor College of Medicine, Houston, Texas.
Am J Hematol. 2018 Jul;93(7):882-888. doi: 10.1002/ajh.25110. Epub 2018 May 6.
Immune thrombocytopenia (ITP) is an acquired autoimmune bleeding disorder which presents with isolated thrombocytopenia and risk of hemorrhage. While most children with ITP promptly recover with or without drug therapy, ITP is persistent or chronic in others. When needed, how to select second-line therapies is not clear. ICON1, conducted within the Pediatric ITP Consortium of North America (ICON), is a prospective, observational, longitudinal cohort study of 120 children from 21 centers starting second-line treatments for ITP which examined treatment decisions. Treating physicians reported reasons for selecting therapies, ranking the top three. In a propensity weighted model, the most important factors were patient/parental preference (53%) and treatment-related factors: side effect profile (58%), long-term toxicity (54%), ease of administration (46%), possibility of remission (45%), and perceived efficacy (30%). Physician, health system, and clinical factors rarely influenced decision-making. Patient/parent preferences were selected as reasons more often in chronic ITP (85.7%) than in newly diagnosed (0%) or persistent ITP (14.3%, P = .003). Splenectomy and rituximab were chosen for the possibility of inducing long-term remission (P < .001). Oral agents, such as eltrombopag and immunosuppressants, were chosen for ease of administration and expected adherence (P < .001). Physicians chose rituximab in patients with lower expected adherence (P = .017). Treatment choice showed some physician and treatment center bias. This study illustrates the complexity and many factors involved in decision-making in selecting second-line ITP treatments, given the absence of comparative trials. It highlights shared decision-making and the need for well-conducted, comparative effectiveness studies to allow for informed discussion between patients and clinicians.
免疫性血小板减少症（ITP）是一种获得性自身免疫性出血性疾病，表现为孤立性血小板减少症和出血风险。虽然大多数 ITP 患儿在不接受药物治疗或接受药物治疗后迅速康复，但其他患儿的 ITP 持续存在或转为慢性。当需要时，如何选择二线治疗方法尚不清楚。ICON1 是在北美儿科 ITP 联盟（ICON）内进行的一项前瞻性、观察性、纵向队列研究，共纳入 21 个中心的 120 例开始接受二线 ITP 治疗的患儿，研究中考察了治疗决策。治疗医师报告了选择治疗方法的理由，并对前三种理由进行了排名。在倾向评分加权模型中，最重要的因素是患者/家长的偏好（53%）和治疗相关因素：副作用谱（58%）、长期毒性（54%）、给药便利性（46%）、缓解可能（45%）和预期疗效（30%）。医生、卫生系统和临床因素很少影响决策制定。在慢性 ITP 中，患者/家长的偏好被选为理由的情况更为常见（85.7%），而在新诊断的 ITP（0%）或持续性 ITP（14.3%）中则不常见（P = .003）。选择脾切除术和利妥昔单抗是因为它们有可能诱导长期缓解（P < .001）。选择口服药物（如艾曲波帕和免疫抑制剂）是因为其给药方便且预期的依从性好（P < .001）。在预计依从性较低的患者中，医师选择利妥昔单抗（P = .017）。治疗选择存在一定的医师和治疗中心偏倚。这项研究说明了在缺乏对照试验的情况下，选择二线 ITP 治疗方法的决策过程的复杂性和涉及的众多因素。它强调了共同决策的重要性，需要进行良好设计的、比较有效性的研究，以便在患者和临床医生之间进行知情讨论。
