RecruitingACTRN12621001397808

The Haemophilia Osteoporosis Registry (THOR): Identifying Mechanisms of Bone Loss in Haemophilia

Sponsor

Monash University

Enrollment

176 participants

Start Date

Jun 9, 2022

Study Type

Observational

Conditions

Haemophilia A Sarcopenia Osteoporosis Osteoarthritis

Summary

Treatment for haemophilia has improved immensely over the past 30 years which has increased the life expectancy in these patients. As such, comorbidities of ageing and their impact is now an area of importance – in particular musculoskeletal health. Reports have shown that fracture incidence is higher in patients with haemophilia, with disease severity effecting fracture risk. Currently the underlying mechanisms of increased fracture risk in patients with haemophilia is unknown, or what age the bone loss begins and whether or not this is different to the general population. Majority of existing studies have reported low areal bone mineral density (aBMD) measured with dual energy x-ray absorptiometry (DXA), however, other components of bone strength may be compromised, and may also contribute to the increased fracture risk. For example, the separate bone compartments (cortical vs trabecular), bone geometry (shape and size), microarchitecture (organisation of trabecular bone) and bone strength (buckling vs strain). With the advancements in bone imaging technology, these other components of bone strength can now be measured using cutting-edge high-resolution peripheral quantitative computed tomography (HRpQCT). Currently there are no guidelines on bone density screening or osteoporosis treatment for patients with haemophilia. As such, it is unknown when the best “window or age” for bone density screening would be most effective to prevent fracture. The findings from this study will indicate at which age bone density screening will be most beneficial for patients with haemophilia A. Therefore, The Haemophilia Osteoporosis Registry (THOR) is timely as it has been empirically designed and powered to identify the mechanisms of bone loss in patients with haemophilia.

Eligibility

Sex: MalesMin Age: 18 Yearss

Plain Language Summary

Simplified for easier understanding

Haemophilia A is a bleeding disorder where the blood doesn't clot properly. Thanks to major advances in treatment over the past few decades, people with haemophilia are living longer than ever. But a longer life means new challenges — including osteoporosis, a condition where bones become weak and fracture more easily. Research suggests people with haemophilia break bones more often than the general population, but the reasons why are not fully understood. This study — the Haemophilia Osteoporosis Registry (THOR) — uses advanced bone imaging technology to look in detail at the structure, density, and strength of bones in men with moderate to severe haemophilia A, compared to healthy men without haemophilia. The goal is to understand when and how bone loss starts in people with haemophilia, so that future screening guidelines can be created. You may be eligible if you are an adult male, weigh under 160 kg, and have been diagnosed with moderate to severe haemophilia A. Healthy men without major medical conditions may also join as controls. Participants need to be able to lie still during imaging scans and communicate effectively in English. The study is conducted at a hospital-based research centre and involves bone density scans and a research visit.

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Interventions

This is a prospective longitudinal observational study, recruiting men with haemophilia A and age-matched controls residing in Victoria, Australia, with a total of 176 participants. Stratified sampling by age will be used to ensure equal distribution across the four age-bands, namely 18-30, 31-40, 41-50, 51+ years. Recruitment will include: patients with haemophilia A will be approached during clinical consultations (in person or via telehealth) at Alfred Health. Each patient who is approached to join the study will be recorded in the Log Book. If patients consent to passing on their contact details, the consultant will refer the patients to the team at Monash Medical Centre to discuss the study in further detail. Potential participants will undergo screening against criteria to determine their eligibility for participation. The age-matched controls will be recruited through: - Posters displayed throughout Monash Medical Centre and Monash University Clayton Campus, and; - Email lists, newsletters, social media, recruitment registry, community organisations, local social and sports clubs will also be employed to advertise recruitment Participants will come into Monash Medical Centre for a study visit at baseline and follow-up 1, with an interval of 12 months between study visits. Data collected will include: - Questionnaires: general demographics, medical history, including medication use and co-morbidities at baseline (General Questionnaire); health changes during the study will be collected at follow-up (General Medical Hx Update); Patient Reported Outcomes Burdens and Experiences (PROBE) questionnaire (containing the EQ-5D-5L and VAS); Bone-specific Physical Activity Questionnaire (BPAQ or BSPA in REDCap); International Physical Activity Questionnaire (IPAQ); Gilbert Joint Health Score questionnaire (Gilbert Score). - Anthropometry: height, weight and sidedness - Bloods: fasting blood samples will be collected to measure procollagen type 1 N propeptide (P1NP), C-terminal telopeptide of type 1 collagen (CTX), calcium, phosphate, vitamin D, and liver function tests (bilirubin, albumin, GGT, ALP, AST, ALT). - Bone health: dual energy x-ray absorptiometry (DXA) and high resolution peripheral quantitative computed tomography (HR-pQCT) - Joint health: HR-pQCT will scan the ankle and knee joints - Body composition: DXA - Muscle strength: hand grip strength and jumping mechanography These will all be collected at both study visits by the research team (including research assistant, Research Fellow, Students (ie PhD, Honours, Medical students). The anticipated duration of each study visit is 1.5hrs.