Benchmarking Paediatric Sickle Cell Care in Australia

Background: Sickle cell disease (SCD) is a severe, multisystem vasculopathy, associated with significant reduction in life expectancy, and high burden of care for patients/families and clinicians. Haematologists in Australia have observed an increase in numbers of children presenting for care with SCD in recent years that disproportionately affects migrant families with socio-economic challenge. Models of care for these patients in Australia are still evolving and there is a need for service benchmarking to define consensus best practice for service delivery. Key issues include access to affordable Hydroxyurea (HU) paediatric formulations, red cell exchange (RCE), antenatal/newborn screening policy and consideration of expanding indications for curative therapies (bone marrow transplantation).

Aim: To survey aspects of health care service delivery for paediatric SCD at six specialist centres in Australia.

Method: Lead clinicians at the six centres were surveyed to determine: basic demographics, availability of current treatment modalities, transfusion practices, service integration, care pathways and access to support services and transition services. Current challenges to optimal health care delivery were identified.

Results: A total of 293 cases were identified. Variations in care included: inequity of funding for HU formulation, access to RCE as recommended transfusion method, screening protocols for stroke prevention, disease specific pain management pathways. Identified challenges to optimal care delivery were consistent across the centres, with common themes identified:

• difficulty accessing adequate support services (social work, mental health) and care co-ordination

• inability to provide comprehensive care such as educational resources/neuropsychology testing (as is best practice in international centres)

• financial restrictions (variations in funding for paediatric formulations where HU is the only disease modifying therapy DMT available in Australia, liquid Hydroxyurea mixture is a special access product often requiring patient co-payment despite PBS coverage of capsules)

• limited networks of clinicians to provide multi-disciplinary care models

• lack of designated transition pathways for young adults in some locations.

Conclusion - This survey has identified key challenges in access to RCE, affordable HU formulations for children, as well as allied health support/care co-ordination for this disadvantaged population. We recommend the need for the development of consensus recommendations for standards of care for sickle cell services in Australia. These standards will provide a useful benchmark for advocacy and interactions with policymakers.

No relevant conflicts of interest to declare.