Table 2 Key challenges in CHI
Challenge | Impact | Possible solution |
|---|---|---|
Early diagnosis and intervention | Delayed diagnosis leads to potential hypoglycemia-related neurological damage and developmental delays Neurocognitive impairment has a significant impact on the lives of patients and their families | Early screening for severe recurring and prolonged hypoglycemia from birth |
Lack of awareness of signs associated with CHI in neonatal primary care providers | Signs and symptoms of CHI are missed or misinterpreted, leading to misdiagnosis or delayed diagnosis | Increasing awareness of the signs of CHI in all professions caring for neonates and infants |
Limited availability of treatment options | Limited repertoire of treatment options for a very heterogeneous patient population, making medical management difficult for many patients | Development of new treatment options |
Lack of responsiveness to available treatment options | For those patients who are unresponsive or only partially responsive to available treatment options, medical management is difficult Patients for whom medical therapy is failing require subtotal pancreatectomy, which is often associated with postsurgical diabetes mellitus | Development of new treatment options |
Adverse events with available treatment options | Medical therapy is associated with concerning adverse events in some patients, including pulmonary hypertension with diazoxide therapy and necrotizing enterocolitis with somatostatin analogue therapy Other adverse events, such as hypertrichosis with diazoxide therapy, can be very distressing for patients and families, even though not of a life-limiting nature | Development of new treatment options |
Glycemic monitoring | The continuous need for glycemic monitoring is very demanding on patients and families Measurements with blood glucose meters may not be performed frequently enough to detect all episodes of hypoglycemia The utility of CGM devices has not yet been established in CHI | Development of new treatment options that minimize the need for constant glycemic monitoring Rigorous evaluation of CGM meters in CHI |