International Guidelines for the Diagnosis and Management of Pyruvate Kinase Deficiency

Living With PKD

People living with PK deficiency, their caregivers and families report that living with PK deficiency negatively impacts the person with PKD’s quality of life including their physical, emotional, and functional well-being. Improved resources are needed to support the PK deficiency community, with a need for better understanding of how the disease-related physical and psychosocial experiences change over the course of the affected person’s lifetime.

Diagnosis and Genetics of PK Deficiency

The rarity of PK deficiency and wide variability of symptoms make it difficult to diagnose resulting in misdiagnosis and underdiagnosis. Without the right diagnosis, people with PK deficiency can’t get the care they need.

Understanding Pyruvate Kinase Deficiency

Pyruvate kinase deficiency (also called PK deficiency or PKD) is a red blood disease that affects people whose bodies don’t make enough of the enzyme pyruvate kinase. People with this rare blood condition are often born with it, inheriting a damaged gene from each of their parents in an autosomal recessive pattern. It is classified as a rare, inherited anemia and causes iron overload that can cause long-term damage to the body.

It is estimated that PK deficiency affects 1 in 100,000 to 1 in 300,000 people worldwide and is pan-ethnic.

Healthcare Professionals

disease prevalence

disease prevalence

Pyruvate kinase deficiency is the most important common cause of chronic congenital non-spherocytic hemolytic anemia worldwide, affecting at least one in 100,000 to one in 300,000 individuals.