PERSISTENT HYPERINSULINEMIC HYPOGLYCEMIA OF INFANCY (PHHI): PARTIAL AND COMPLETE CLINICAL REMISSION AFTER LONG-TERM OCTREOTIDE TREATMENT

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Moderate to severe PHHI usually requires near-total pancreatectomy to avoid hypoglycemia induced brain damage. We treated 13 such patients with octreolide over the last 6 y. Seven subsequently underwent partial pancreatectomy: 2 had inadequate response due to sepsis, 1 failed to respond to combined octreotide/glucagon/diazoxide and 4 because their family situation did not permit chronic octreotide treatment. Data on the remaining 6 pts on chronic octreotide treatment without pancreatectomy are reported here. Frequent feedings with high glucose intake (11-13 mg/kg/min) and raw cornstarch at night were required in all patients, whereas 2 also required gastrostomy due to poor feeding. Octreotide was started immediately in 5 of 6 patients since previous experience indicates that diazoxide is rarely effective in severe disease. Octreotide was given in 3-4 daily sc injections in 3 patients, and continuous sc infusion (Medix insulin infuser) in 3. All patients had acute G1 symptoms (vomiting, abdominal distcntion, steatorrhea) and weight loss after beginning the drug. This responded partially to oral pancreatic enzyme treatment, and remitted after 2-4 weeks. Asymptomatic gallstones were discovered at routine ultrasound in 1 patient after 1 y of treatment. Growth rate decreased in all during the first 2-6 months, but normalized subsequently as did body weight. All patients have normal psychomotor development for age, however 2 are <1 year old. Every 6-12 months an attempt was made to stop octreotide treatment or switch to diazoxide. Five patients stopped octreotide after 8.5 m to 5.5 y; 1 switched to diazoxide (3y), 2 required per-cutaneous gastrostomy, and 1 (5.5 y old) required no further treatment. The remaining 2 (age 4M-1.5y) are still treated with octreotide. We conclude that with octreotide treatment pancreatectomy can be avoided in some patients. However, efficacy is partial, and close follow-up with repeated blood glucose determinations, frequent feedings, gastrostomy and hospital admissions during acute illnesses may still be necessary. Most can eventually stop octreotide and enter complete or partial remission.