Treatment regimens and outcomes in severe and moderate haemophilia A in the UK: The THUNDER study

Journal article


Scott M.J., Xiang, H., Hart, D.P., Palmer, B., Collins, P.W., Stephensen, D., Sima, C.S. and Hay, C.R.M. 2018. Treatment regimens and outcomes in severe and moderate haemophilia A in the UK: The THUNDER study. Haemophilia. https://doi.org/10.1111/hae.13616
AuthorsScott M.J., Xiang, H., Hart, D.P., Palmer, B., Collins, P.W., Stephensen, D., Sima, C.S. and Hay, C.R.M.
Abstract

Introduction: The THUNDER study provides an analysis of treatment patterns and outcomes in UK patients with severe or moderate haemophilia A (SHA/MHA) in 2015.

Methods: Patients with SHA or MHA registered with the UK National Haemophilia Database (NHD) were segregated by severity, inhibitor status and age. Haemophilia joint health score (HJHS) was derived from NHD records and treatment regimen and annualized bleed/joint‐bleed rate (ABR/AJBR) from Haemtrack (HT) in HT‐compliant patients.

Results: We report 1810 patients with SHA and 864 with MHA. Prophylaxis was used in 94.9% (n = 130/137) of HT‐compliant children <12 years with SHA, falling to 74.1% (n = 123/166) aged ≥40 years. Median ABR increased with age (1.0, IQR 0.0‐5.0, <12 years; 3.0 IQR, 1.0‐8.0, ≥40 years). Inhibitors were present in 159 (8.8%) SHA and 34 (3.9%) MHA. Median ABR increased from 2.0 (<12 years) to 21.(≥40 years) in SHA inhibitor patients using prophylaxis. Prophylaxis was used by 68.8% of HT‐compliant MHA patients (n = 106) (median FVIII baseline 0.01 IU/mL) associated with a median (IQR) ABR of 3.0 (1.0‐7.0). Median HJHS (n = 453) increased with age in SHA and MHA. Median (IQR) HJHS was higher in SHA inhibitor (17.0, 0.0‐64.5) than non‐ or past inhibitor patients (7.0, 0.0‐23.0).

Conclusions: Increasing ABR with age persists despite current prophylaxis regimens.SHA and MHA had similar ABR/AJBR and HJHS, leading to a suspicion that a subgroup of MHA may be relatively undertreated. More intensive prophylaxis may improve outcomes, but this requires further study.

KeywordsAnnualized bleed rate; FVIII inhibitors; Haemophilia A, ; Haemophilia joint health score; Prophylaxis
Year2018
JournalHaemophilia
PublisherWiley
ISSN1351-8216
1365-2516
Digital Object Identifier (DOI)https://doi.org/10.1111/hae.13616
Official URLhttps://onlinelibrary.wiley.com/doi/10.1111/hae.13616
Publication dates
Online08 Nov 2018
Publication process dates
Accepted08 Sep 2018
Deposited31 Mar 2022
Accepted author manuscript
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Output statusPublished
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https://repository.canterbury.ac.uk/item/90w87/treatment-regimens-and-outcomes-in-severe-and-moderate-haemophilia-a-in-the-uk-the-thunder-study

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