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Strensiq – a drug treatment for severe hypophosphatasia – is now available by a managed access agreement through NHS England and NICE.


Here are some of the resources we have gathered together to assist you in diagnosis and treatment:


Announcements

NHS Strensiq announcement

Managed Access Agreement (containing patient eligibility, stopping rules (sheet 21 onwards) and the consent form (sheet 11)

Current NICE guidance on asfotase alfa (Strensiq) for treating paediatric-onset hypophosphatasia in adults and children


Background

The hallmark of the condition is a persistently low alkaline phosphatase (ALP) level. Diagnostic delay of over 10 years is common because the significance of low ALP is often not recognised. Patients usually present with predominantly musculoskeletal symptoms, including:

  • Chronic pain

  • CPPD 'pseudogout'

  • Early-onset osteoarthritis

  • Calcific peritendonitis – mimics spondyloarthropathy and Diffuse Idiopathic Skeletal Hyperostosis (DISH)

  • Fractures

  • Dental loss


If you see a patient with an unexplained persistently low ALP level, please consider a referral to a specialist centre for an assessment. These are:


Adults

  • Addenbrooke’s Hospital, Cambridge

  • Queen Elizabeth Hospital, Birmingham

  • Manchester Royal Infirmary

  • Royal National Orthopaedic Hospital (Stanmore)

  • St. George’s Hospital, London

  • Oxford University Hospital

  • Norfolk & Norwich Hospital

  • Sheffield Northern General Hospital


Paediatrics

  • Sheffield Children’s Hospital

  • Manchester Children’s Hospital

  • Birmingham Children’s Hospital