The impact of home therapy interventions on treatment adherence and patient independence in haemophilia management in Assam, India

The study included patients diagnosed with haemophilia A or B (mild, moderate, or severe), aged ≥2 years, previously dependent on hospital-based infusion therapy, and willing to provide informed consent. Patients were excluded if they had active inhibitors against clotting factors, cognitive or physical disabilities preventing self-infusion, or severe comorbid conditions limiting home-based care. The sample size was determined based on prior pilot data, assuming a 50% improvement in adherence, with a 95% confidence interval and 80% power.

The study employed a clearly defined model of structured home therapy, involving a planned, multi-component intervention designed to enable PWH and their caregivers to independently administer clotting factor concentrates at home with competence and confidence. This model encompassed four core elements: patient education, practical infusion training, supervised transition to home-based care, and continuous follow-up and support. Implementation began following the baseline survey conducted in February 2023, which assessed patient demographics, disease severity, treatment history, and barriers to home therapy. Eligible patients were enrolled between February and May 2023, each participating in the program for six months.

The training program (Table 1) was delivered by trained haemophilia nurses under the supervision of the treating physician, and guided by internal standard operating procedures (SOPs) developed at Assam Medical College. Training included:

Monthly self-infusion workshops held at the treatment centre, where patients practiced venipuncture using arm mannequins and vein-locator devices;

To support the transition to home therapy, nurse-led home visits were conducted at least once every month, during which patients demonstrated the infusion process under observation. Nurses offered real-time correction of technique, reinforced dosing reminders, and addressed any infusion-related concerns. During implementation, the team encountered logistical challenges, particularly in scheduling visits to remote outstation locations, and occasional interruptions in factor supply, which required coordination with government procurement agencies. Some patients initially expressed apprehension regarding venous access or needle phobia; these were mitigated through repeated counselling and caregiver involvement. This structured approach ensured that home therapy was not only introduced safely but was also sustained through regular reinforcement, supervision, and capacity-building at the household level.

Nurses played a pivotal role as educators and care coordinators in the structured home therapy program. They began with conducting hands-on training sessions, where study participants and caregivers were taught proper self-infusion techniques using arm mannequins and vein-locator devices. The nurses also facilitated video-based education on factor preparation, administration, and storage; following training, they undertook monthly home visits to monitor adherence to treatment, assess technique accuracy, and provide real-time feedback. During these visits, they reinforced dose timing, addressed complications, and documented progress. They also provided a point of liaison between the patients and the treatment centre, promptly reporting clinical concerns to the haematologist, coordinating factor availability, and ensuring continuity of care. Their ongoing support was central to building patient confidence and sustaining safe home therapy practices.

Throughout the study, participants completed two follow-up surveys: a mid-home therapy (HT) survey in October 2023 to assess initial improvements in adherence and satisfaction, and a post-HT survey in December 2023, evaluating treatment compliance, self-infusion proficiency, and quality of life changes. The intervention specifically addressed key barriers such as long travel distances (enabled home therapy), unavailability of clotting factors (ensured supply), and lack of awareness (provided counselling and education). The intervention strategies included clinical support from nurses, educational materials such as training videos and patient manuals, and technology-driven adherence tools like SMS reminders for dosing and factor administration.

All PWH were assessed for annual bleed rate, joint health, functional state and QoL, before and after implementation of home therapy, using standard validated methods including Haemophilia Joint Health Score (HJHS), Functional Independence Score in Haemophilia (FISH) and EQ-5D-5L. Adherence to therapy and patient satisfaction were assessed using structured questionnaires developed by the patient support program (PSP) team, based on WHO adherence frameworks and patient-reported outcome measures. Though not externally validated, these tools were piloted for clarity and cultural relevance. Data were collected at three time points — pre-therapy (February 2023), mid-therapy (October 2023), and post-therapy (December 2023) — via face-to-face interviews during clinic visits and nurse-led home visits (Table 2).

Statistical analysis was conducted using descriptive statistics (mean, standard deviation, proportions, and percentages) to summarise participant characteristics. Comparative analyses included paired t-tests to evaluate pre- and post-intervention changes in self-infusion rates and adherence. The effect size (Cohen's d) was calculated to assess the magnitude of change following the intervention, and multivariate regression was used to identify predictors of improved adherence.

The study adhered to strict ethical guidelines. Ethical approval was obtained from the Institutional Ethics Committee (H), Assam Medical College, Dibrugarh (Reg no. ECR/636/Inst/AS/2014) No. AMC/EC/5876 dated 3rd June 2021. Informed consent was collected from all adult participants and guardians of minors. Patient confidentiality was maintained by anonymising data and securing records. The study was independently conducted without pharmaceutical sponsorship, ensuring no conflict of interest.

In our cohort, the proportion of patients capable of performing self-infusion rose significantly from 38% at baseline to 75% post-intervention. This improvement is clinically meaningful, as self-management – the ability of PWH to actively manage their daily health and healthcare – supports better bleed management, which is vital in haemophilia care ^[4]. This progress was achieved through the dedicated efforts of haemophilia nurses, who empowered patients by providing sustained training and conducting regular home visits.

The importance of the nurse role in empowering PWH to self-manage their condition has been recognised and continues to be developed elsewhere, with nurses providing education and support in all aspects of self-management, including bleed recognition, prevention and management, and home treatment ^[12,13]. In the UK, the Haemophilia Nurses Association's core competency framework for haemophilia nurses includes a range of competencies linked to communicating with and supporting patients and their families. This includes teaching and reviewing self-management skills, enabling PWH to manage bleeding symptoms independently, and delivering patient education ^[14].

Our findings reinforce the vital contribution of the haemophilia nurse and the need for a structured learning module to advance haemophilia care. Integrating telehealth-based mentoring, digital infusion logs, and remote counselling can further enhance skill retention and promote self-efficacy among PWH—an approach particularly valuable for those in rural and semi-urban settings.

Our data revealed a 56% increase in the uptake of prophylactic therapy following the structured nursing intervention. Prophylaxis remains the gold standard for preventing spontaneous bleeds and preserving long-term joint function ^[11,15]. However, suboptimal compliance remains a widespread challenge in many low- and middle-income countries ^[16]. Evatt underscores the need for sustainable decentralised models to overcome systemic barriers and improve patient outcomes worldwide ^[13]. Our findings reaffirm that accessible, context-sensitive nursing support is pivotal for expanding prophylaxis uptake in India.

Adherence to the recommended dosing frequency is vital for bleed prevention and long-term joint health. In our cohort, adherence to the prescribed twice-weekly dosing schedule improved from 76% to 83%, while infrequent dosing patterns decreased. Counselling has been shown to be an effective approach that nurses can use to supplement other methods, building a multifaceted strategy to enhance adherence; tackling non-adherence seems to demand continuous efforts and follow-up ^[17]. Khair highlights how proactive nursing engagement fosters trust and reinforces treatment routines ^[12].Furthermore, consistent prophylaxis initiated early in life can prevent joint disease, justifying continued investments in adherence support ^[15].

Improved mobility and pain reduction were reported by 93% and 82% of patients, respectively, following the intervention. These self-reported gains align with existing literature showing that well-managed prophylaxis and timely infusions effectively preserve joint function and reduce disability ^[15,18]. García-Dasí et al. demonstrate a clear correlation between adherence to prophylaxis and better QoL scores in paediatric patients, highlighting the broader psychosocial benefits of optimal disease control ^[9]. Integrating adjunct interventions — such as remote physiotherapy and structured pain education — has further potential to maximise functional outcomes.

One of the most significant outcomes in our study was a 55% reduction in reliance on hospital-administered infusions, illustrating a successful transition to patient-managed or caregiver-administered treatment. This shift is consistent with global efforts to decentralise haemophilia care, thereby reducing the burden on tertiary care infrastructure and reallocating resources to patients with complex needs ^[11]. Home therapy has been shown to not only reduce hospitalisation costs but also to improve cost-effectiveness by preventing expensive complications associated with untreated bleeds ^[11,19].

This study provides important regional evidence supporting structured nursing interventions and home therapy in an Indian context. By implementing a tailored educational programme complemented by telehealth tools, we have demonstrated that sustainable, patient-centred models are feasible even in resource-limited settings. Future programmes should consider broader digital integration for real-time dose tracking and remote monitoring, as suggested by recent trials ^[20]. Additionally, training caregivers — particularly in paediatric cases — can ensure equitable access to home-based therapy for younger patients who cannot self-infuse independently ^[21].

Despite these encouraging outcomes, the study has some limitations. The sample size was modest and derived from a single institution, meaning the results may not be generalisable. The sample size was determined based on feasibility and resource availability within the defined program duration, rather than formal power calculations based on effect size assumptions. However, a significant proportion of PWH at the treatment centre were already on home therapy at baseline, meaning the study could specifically targeted those patients who faced significant barriers to adopting or sustaining home-based care, as identified through the baseline survey. Therefore, while the small sample size may limit generalisability, the study provides valuable insights into real-world implementation challenges and practical solutions in a resource-constrained setting, forming a basis for larger multicentre or regional studies in the future.