Abstract Number: PB0468
Meeting: ISTH 2021 Congress
Theme: Hemophilia and Rare Bleeding Disorders » Hemophilia - Basic
Background: Haemophilia is characterized by spontaneous hemarthrosis that often develop into chronic synovitis, pain, and eventually destruction of the joint.
Clinical metrics of joint morbidity, e.g., ‘Target joint’, are prevalent, accepted and relevant, though measures focused on bleeding activity are arguably becoming less sensitive to patient burden as current treatment strategies look to eradicate hemarthrosis.
‘Problem Joint’ (PJ) is a joint morbidity metric defined by an Expert Reference Group as having chronic joint pain and/or limited range of movement due to chronic synovitis and/or haemophilic arthropathy, with or without persistent bleeding.
Aims: Test the sensitivity of PJs as a patient relevant metric in children.
Methods: Data on PJs, demographics, clinical and socio-economic variables were captured within the ‘Cost of Haemophilia in Men: Socioeconomic Survey’ a family of datasets containing over 4,000 people with haemophilia. The paediatric European cohort (CHESS Paeds) comprised the focus of this piece. Statistical analysis explored the association of PJ count and location with respect to quality of life (EQ-5D-Y), and parent caregiving duties. Individuals with inhibitors were excluded.
Results: This cohort (N=198) recorded a mean age of 11.2. Around 19% had one or more PJs. Weight bearing PJs were more prevalent; 12% had 1+ weight bearing PJs vs. 8.5% non-weight bearing. EQ-5D and PJ count showed a negative trend (see Figure 1). Mean EQ-5D score was 0.71 for those with 0 PJs (N=160), 0.60 for 1 PJ (N=30) and 0.47 for 2+ PJs (N=8). Similarly, an increase in PJs lead to a greater caregiver burden: an average of 19.2 hours/week for those with 0 PJ (N=124) vs. 30.8 for 1+ PJs (N=29).
Conclusions: An increase in the number of PJs was associated with increasing humanistic burden in children. The PJ definition assesses from a patient relevant perspective. Further work will evaluate the appropriateness of the measure in a wider cohort.
To cite this abstract in AMA style:
Burke T, Rodriguez Santana I, Chowdary P, Curtis R, Khair K, Laffan M, McLaughlin P, Noone D, O'Mahony B, Pasi J, W Skinner M, O'Hara J. Examination and Validation of a Patient-centric Joint Metric: “PROBLEM JOINT”; Empirical Evidence from the CHESS Paediatrics Dataset [abstract]. Res Pract Thromb Haemost. 2021; 5 (Suppl 2). https://abstracts.isth.org/abstract/examination-and-validation-of-a-patient-centric-joint-metric-problem-joint-empirical-evidence-from-the-chess-paediatrics-dataset/. Accessed March 22, 2024.« Back to ISTH 2021 Congress
ISTH Congress Abstracts - https://abstracts.isth.org/abstract/examination-and-validation-of-a-patient-centric-joint-metric-problem-joint-empirical-evidence-from-the-chess-paediatrics-dataset/