Severe forms of hemophilia and sickle cell disease are diagnosed in childhood and treated for the rest of a patient's life, with most of that treatment happening at home, administered by the patient directly or a caregiver.
The care system meets these patients in episodes: there are a few appointments a year, occasional admissions in acute circumstances, and a handover from pediatric to adult services when patients come of age. Throughout such an extended care journey, what holds the span together is a continuous record for visibility and better clinician-informed decisions, although usually there is not one.
A lifetime of treatment with no continuous record
Record-keeping of bleeds and treatment is a named component of self-management in professional guidance, and those records inform treatment decisions and long-term management.¹
Importantly, the clinical picture in these conditions is largely what the patient and caregiver report. However, much of it is never written down or documented. Patients with sickle cell disease report managing an average of seven painful crises a year without medical help, preferring home to hospital care they have found poor.² Those events shape the course of the disease and leave no trace in that record.
The handover from pediatric to adult services is also where the span breaks visibly. As an example, young adults with sickle cell disease who waited six months or more between their last pediatric and first adult appointment went on to twice the rate of hospitalization over the following eight years, compared with those who transferred within two months. ³
In the end, what families are given to work with is fragmented. Caregivers and clinicians of children with hemophilia describe the available information as incomplete and lacking structure, and want it standardized and centralized in digital form. ⁴ Care plans themselves are often held on paper and used inconsistently between settings. ⁵
How Dawn Health holds the record: different condition, same model
Ekiva PNH, Dawn Health's Therapy Companion for paroxysmal nocturnal hemoglobinuria, built with Novartis, is not a hemophilia or sickle cell product (dawnhealth.com/ekiva-pnh). What it shows is the same model running in rare hematology today: disease-specific symptom and lab logging feeding reports a care team can use.
The available product suite tier is designed for this pattern of care:
- Symptoms and lab values logging: bleeds and infusions recorded alongside lab results, at the time they happen.
- Quality of life logging: the impact treatment has on daily life, tracked continuously through defined sets of questionnaires.
- Personal reports and consultation support: patients arrive with a structured treatment and health history, which matters most when the person across the desk is new to them.
- Educational content and support: standardized, condition-specific information of the kind families say they cannot currently find in one place.⁴
The commercial case for pharma
In hemophilia, adherence tracks with fewer bleeds and healthier joints, so a companion built to support it works on the outcome the therapy exists to produce.⁶
A product used from childhood into adult life holds a relationship across decades, and across every therapy the patient takes in that time. Where several therapies share similar mechanisms, the support wrapped around them is one of the few remaining points of difference.
The record has commercial weight of its own: bleed rates and quality-of-life trajectories collected continuously feed the real-world evidence base that small, dispersed populations depend on for reimbursement.
Dawn Health designs patient support solutions to extend it.
References
- Srivastava A, Santagostino E, Dougall A, et al. WFH guidelines for the management of hemophilia, 3rd edition. Haemophilia. 2020;26(Suppl 6):1-158. doi:10.1111/hae.14046.
- NHS Race and Health Observatory. Sickle cell patients face unequal care: new report highlights. June 2025. Research conducted by Imperial College London. nhsrho.org.
- Howell KE, Kayle M, Smeltzer MP, et al. Gaps during pediatric to adult care transfer escalate acute resource utilization in sickle cell disease. Blood Advances. 2024;8(14):3679-3685. doi:10.1182/bloodadvances.2023011268.
- Mussert CMA, Kamminga NCW, van Hoorn ES, et al. Experiences and needs regarding information provision in children with haemophilia: a qualitative study on caregivers' and healthcare providers' perspectives. Haemophilia. 2025. doi:10.1111/hae.70063.
- NHS Race and Health Observatory and Public Digital. Digital care plans can transform NHS services for sickle cell patients. January 2023. nhsrho.org.
- Khanji C, Nuabor W, Gould TJ, et al. Systematic literature review of outcomes associated with adherence to haemophilia drug therapy. Haemophilia. 2025. doi:10.1111/hae.15153.
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