Riera-Arnau J, Ballarín E, Llop R, Montané E, Hereu P, Vancells G, Padullés-Zamora N, Barriocanal A M, Cardona-Peitx G, Casasnovas C, Montoro J B, Nuñez M, Santacana Juncosa E, Selva-O'Callaghan A, Solanich X, Sabaté Gallego M
Department of Clinical Pharmacology, Vall d'Hebron Hospital Universitari, Vall Hebron Institut de Recerca (VHIR), Vall d'Hebron Barcelona Hospital Campus, Barcelona, Spain.
Department of Pharmacology, Therapeutics and Toxicology, Universitat Autònoma de Barcelona (UAB), Barcelona, Spain.
Front Pharmacol. 2024 Dec 16;15:1420682. doi: 10.3389/fphar.2024.1420682. eCollection 2024.
The increasing use of non-specific immunoglobulins (NSIGs) and their current shortage show a need for NSIGs' use prioritization. Data from a clinical perspective are necessary, mainly for pediatric patients.
The aim of the study was to assess the level of clinical evidence (LoE) of the indications that NSIGs are used for, the reasons for discontinuation, and the costs invested.
A retrospective multicentric study was conducted on NSIG incident users between September 2019 and December 2021 retrieved from the Registry of Patients and Treatments (RPT) from Catalonia (Spain). LoE was categorized as A) authorized indications, B) unauthorized with scientific support, C) unauthorized without support, and D) unknown (UNK), following local and the United Kingdom's guidelines as a sensitivity analysis. We also estimated overall spending and costs per patient visit.
A total of 400 patients were included (17.3% pediatric), with a mean follow-up of 122.1/person-years for adults. The most frequent indications were nervous system and blood diseases. Almost all pediatric patients (56; 81.2%) were treated under A-level indications, as for 217 (65.6%) adults. In the sensitivity analysis, the A-level usage rate decreased to one-third and the B-level usage rate increased by 2-3 times. Furthermore, 37.8% (151) of individuals discontinued. This was predominantly due to remission or no response. The total costs were 868,462.6€/year, with median spending per visit amounting to 1,500€ for adults and 700€ for pediatric patients.
NSIGs are used in clinical practice mainly for approved indications; however, non-approved indications are still an important issue. This could represent a significant economic burden on the healthcare system, focusing on the pediatric population and those at risk for discontinuation with alternative therapeutic options.
非特异性免疫球蛋白（NSIGs）的使用日益增加且目前供应短缺，这表明需要对NSIGs的使用进行优先级排序。从临床角度获取数据很有必要，尤其是针对儿科患者。
本研究旨在评估NSIGs使用指征的临床证据水平、停药原因以及所投入的成本。
对2019年9月至2021年12月期间从西班牙加泰罗尼亚患者与治疗登记处（RPT）检索到的NSIGs新使用者进行回顾性多中心研究。按照当地和英国的指南将临床证据水平分为A）批准的指征、B）有科学支持的未批准指征、C）无支持的未批准指征和D）未知（UNK），作为敏感性分析。我们还估算了总体支出以及每次患者就诊的费用。
共纳入400例患者（17.3%为儿科患者），成人患者的人均随访时间为122.1人年。最常见的指征是神经系统疾病和血液疾病。几乎所有儿科患者（56例；81.2%）接受的是A级指征治疗, 成人患者中有217例（65.6%）。在敏感性分析中, A级使用率降至三分之一，B级使用率增加了2至3倍。此外，37.8%（151例）的患者停药。这主要是由于病情缓解或无反应。每年的总费用为868,462.6欧元，成人每次就诊的中位费用为1500欧元，儿科患者为700欧元。
NSIGs在临床实践中主要用于批准的指征；然而，未批准的指征仍然是一个重要问题。这可能给医疗系统带来巨大的经济负担，尤其是儿科人群以及有停药风险且无替代治疗选择的人群。
