Electronic diaries in the management of haemophilia gene therapy: Perspective of an expert group from the German, Austrian and Swiss Society on Thrombosis and Haemostasis (GTH)

Abstract

Introduction Gene therapy (GT) is becoming a realistic treatment option for patients with haemophilia. Outside clinical trials, the complexity and potential complications of GT will pose unprecedented challenges to haemophilia care centres. Aim To explore the potential use of electronic tools to improve the delivery of GT under real‐world conditions. Methods Considering the hub‐and‐spoke model, the GTH working group on GT considered the entire patient pathway and reached consensus on requirements for an integrative software tool to secure documenting and sharing information between treaters, pharmacies and patients. Results Six steps of the gene therapy process were identified, each requiring completion of the previous step as a prerequisite for entry. The responsibilities of GT dosing and follow‐up treatment centres, read/write access rules, and the minimum data set were outlined. Data contributed by patients through mobile devices was also considered. Conclusion Important information needs to be shared between patients and treatment centres in a real‐world GT hub‐and‐spoke model. Collecting and sharing this information in well‐organised electronic applications will not only improve patient care but also enable national and international data collection in clinical registries.

Gentherapie der Hämophilie: Empfehlung der Gesellschaft für Thrombose- und Hämostaseforschung (GTH)

“…To date, tumor diseases have been occurred in four patients after gene therapy. 21 37 38 39 40 The comprehensive clinical, histopathologic, and molecular genetic reappraisal of these cases revealed no evidence of a causal relationship with the gene therapy. Similarly, a comprehensive molecular genetic reappraisal should be initiated in future cases of tumors after gene therapy.…”

“…Checklists can help to collect the required findings and other documentation at the referring center (spoke) before introduction of the patient to the dosing center (hub). 41 These are documented in the applica-tion as in a file and, with the patient's consent, shared with the hub center even before a presentation. In this way, unnecessary duplicate examinations and drives to the dispensing center can be reduced.…”

“…Electronic diaries are already common in the documentation of hemophilia treatment and are conscientiously kept by many patients. [39][40][41][42][43] Therefore, it makes sense to actively involve the patient in the documentation of all relevant data. In this way, especially during follow-up, information on bleeding and other PROs can be available to all physicians involved without loss of time and help to provide optimal advice to the patient.…”

“…Checklisten können helfen, vor einer Vorstellung des Patienten im Dosierungszentrum (Hub) die erforderlichen Befunde und sonstigen Unterlagen im zuweisenden Zentrum (Spoke) zu sammeln. 40 Register der Welt-Hämophilie-Organisation (WFH). 43 Neben der inhaltlichen Planung und Programmierung einer solchen elektronischen Plattform sind Datenschutz, Datensicherheit, Finanzierung und vertragliche Regelung zwischen den beteiligten Einrichtungen nicht unerhebliche Herausforderungen.…”

“…Checklisten können helfen, vor einer Vorstellung des Patienten im Dosierungszentrum (Hub) die erforderlichen Befunde und sonstigen Unterlagen im zuweisenden Zentrum (Spoke) zu sammeln. 40 Diese werden in der Anwendung wie in einer Akte dokumentiert und mit Einverständnis des Patienten auch schon vor einer Vorstellung im Hub-Zentrum mit diesem geteilt. So können unnötige Doppeluntersuchungen und Fahrten in das Dosierzentrum reduziert werden.…”