TY  - JOUR
A1  - Miesbach, Wolfgang
A1  - Eichler, Hermann
A1  - Holstein, Katharina
A1  - Holzhauer, Susanne
A1  - Klamroth, Robert
A1  - Knöfler, Ralf
A1  - Male, Christoph
A1  - Olivieri, Martin
A1  - Oldenburg, Johannes
A1  - Tiede, Andreas
T1  - 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)
T2  - Haemophilia
N2  - 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...
KW  - data management
KW  - electronic diaries
KW  - gene therapy
KW  - haemophilia treatment
KW  - treatment centres
Y1  - 2022
UR  - http://publikationen.ub.uni-frankfurt.de/frontdoor/index/index/docId/75592
UR  - https://nbn-resolving.org/urn:nbn:de:hebis:30:3-755928
SN  - 1365-2516
VL  - 28
IS  - 2
SP  - 264
EP  - 269
PB  - Wiley-Blackwell
CY  - Oxford [u.a.]
ER  -