Page 1 Somatropin INITIAL APPLICATION - growth hmone deficiency in children Growth hmone deficiency causing symptomatic hypoglycaemia, with other significant growth hmone deficient sequelae (e.g. cardiomyopathy, hepatic dysfunction) diagnosed with GH < 5 mcg/l on at least two rom blood samples in the first 2 weeks of life, from samples during established hypoglycaemia (whole blood glucose < 2 mmol/l using a labaty device) Height velocity < 25th percentile f age adjusted f bone age/pubertal status if appropriate over 6 12 months using the stards of Tanner Davies (1985) A current bone age is < 14 years (female patients) < 16 years (male patients) Peak growth hmone value of < 5.0 mcg per litre in response to two different growth hmone stimulation tests. In children who are 5 years older, GH testing with sex steroid priming is required If the patient has been treated f a malignancy, they should be disease free f at least one year based upon follow-up labaty radiological imaging appropriate f the malignancy, unless there are strong medical reasons why this is either not necessary appropriate Appropriate imaging of the pituitary gl has been obtained RENEWAL - growth hmone deficiency in children A current bone age is 14 years under (female patients) 16 years under (male patients) Height velocity is greater than equal to 25th percentile f age (adjusted f bone age/pubertal status if appropriate) while on growth hmone treatment, as calculated over six months using the stards of Tanner Davis (1985) Height velocity is greater than equal to 2.0 cm per year, as calculated over 6 months No serious adverse effect that the patients specialist considers is likely to be attributable to growth hmone treatment has occurred No malignancy has developed since starting growth hmone See also: INITIAL APPLICATION - Turner syndrome p2, RENEWAL - Turner syndrome p2, INITIAL APPLICATION - sht stature without growth hmone deficiency p2, RENEWAL - sht stature without growth hmone deficiency p3, INITIAL APPLICATION - sht stature due to chronic renal insufficiency p3, RENEWAL - sht stature due to chronic renal insufficiency p4, INITIAL APPLICATION - Prader-Willi syndrome p5, RENEWAL - Prader-Willi syndrome p5, INITIAL APPLICATION - adults adolescents p6 RENEWAL - adults adolescents p7 I confirm the above details are crect that in signing this fm I underst I may be audited.
Page 2 INITIAL APPLICATION - Turner syndrome The patient has a post-natal genotype confirming Turner Syndrome Height velocity is < 25th percentile over 6-12 months using the stards of Tanner Davies (1985) A current bone age is < 14 years RENEWAL - Turner syndrome Height velocity is greater than equal to 50th percentile f age (while on growth hmone calculated over 6 to 12 months using the Ranke's Turner Syndrome growth velocity charts) Height velocity is greater than equal to 2 cm per year, calculated over six months A current bone age is 14 years under No serious adverse effect that the specialist considers is likely to be attributable to growth hmone treatment has occurred No malignancy has developed since starting growth hmone INITIAL APPLICATION - sht stature without growth hmone deficiency The patient's height is me than 3 stard deviations below the mean f age f bone age if there is marked growth acceleration delay Height velocity is < 25th percentile f age (adjusted f bone age/pubertal status if appropriate), as calculated over 6 to 12 months using the stards of Tanner Davies(1985) A current bone age is < 14 years under (female patients) < 16 years (male patients) The patient does not have severe chronic disease (including malignancy recognized severe skeletal dysplasia) is not receiving medications known to impair height velocity See also: RENEWAL - sht stature without growth hmone deficiency p3, INITIAL APPLICATION - sht stature due to chronic renal insufficiency p3, RENEWAL - sht stature due to chronic renal insufficiency p4, INITIAL APPLICATION - Prader-Willi syndrome p5, RENEWAL - Prader-Willi syndrome p5, INITIAL APPLICATION - adults adolescents p6 RENEWAL - adults adolescents p7 I confirm the above details are crect that in signing this fm I underst I may be audited.
Page 3 RENEWAL - sht stature without growth hmone deficiency Height velocity is greater than equal to 50th percentile (adjusted f bone age/pubertal status if appropriate) as calculated over 6 to 12 months using the stards of Tanner Davies (1985) Height velocity is greater than equal to 2 cm per year as calculated over six months A current bone age is 14 years under (female patients) 16 years under (male patients) No serious adverse effect that the patient's specialist considers is likely to be attributable to growth hmone treatment has occurred INITIAL APPLICATION - sht stature due to chronic renal insufficiency Applications only from a paediatric endocrinologist, endocrinologist renal physician on the recommendation of a paediatric endocrinologist endocrinologist. Approvals valid f 9 months. The patient's height is me than 2 stard deviations below the mean Height velocity is < 25th percentile (adjusted f bone age/pubertal status if appropriate) as calculated over 6 to 12 months using the stards of Tanner Davies (1985) A current bone age is to 14 years under (female patients) to 16 years under (male patients) The patient is metabolically stable, has no evidence of metabolic bone disease absence of any other severe chronic disease The patient is under the supervision of a specialist with expertise in renal medicine The patient has a GFR less than equal to 30 ml/min/1.73m² as measured by the Schwartz method (Height(cm)/plasma creatinine (umol/l) x 40 = crected GFR (ml/min/1.73m² in a child who may may not be receiving dialysis The patient has received a renal transplant has received < 5mg/ m²/day of prednisone equivalent f at least 6 months. See also: RENEWAL - sht stature due to chronic renal insufficiency p4, INITIAL APPLICATION - Prader-Willi syndrome p5, RENEWAL - Prader-Willi syndrome p5, INITIAL APPLICATION - adults adolescents p6 RENEWAL - adults adolescents p7 I confirm the above details are crect that in signing this fm I underst I may be audited.
Page 4 RENEWAL - sht stature due to chronic renal insufficiency Applications only from a paediatric endocrinologist, endocrinologist renal physician on the recommendation of a paediatric endocrinologist endocrinologist. Approvals valid f 12 months. Height velocity is greater than equal to 50th percentile (adjusted f bone age/pubertal status if appropriate) as calculated over 6 to 12 months using the stards of Tanner Davies (1985) Height velocity is greater than equal to 2 cm per year as calculated over six months A current bone age is 14 years under (female patients) 16 years under (male patients) No serious adverse effect that the patients specialist considers is likely to be attributable to growth hmone has occurred No malignancy has developed after growth hmone therapy was commenced The patient has not experienced significant biochemical metabolic deteriation confirmed by diagnostic results The patient has not received renal transplantation since starting growth hmone treatment If the patient requires transplantation, growth hmone prescription should cease befe transplantation a new application should be made after transplantation based on the above criteria See also: INITIAL APPLICATION - Prader-Willi syndrome p5, RENEWAL - Prader-Willi syndrome p5, INITIAL APPLICATION - adults adolescents p6 RENEWAL - adults adolescents p7 I confirm the above details are crect that in signing this fm I underst I may be audited.
Page 5 INITIAL APPLICATION - Prader-Willi syndrome The patient has a diagnosis of Prader-Willi syndrome that has been confirmed by genetic testing clinical scing criteria The patient is aged six months older A current bone age is < 14 years (female patients) < 16 years (male patients) Sleep studies overnight oximetry have been perfmed there is no obstructive sleep disder requiring treatment, if an obstructive sleep disder is found, it has been adequately treated under the care of a paediatric respiraty physician / ENT surgeon The patient is aged two years older There is no evidence of type II diabetes uncontrolled obesity defined by BMI that has increased by greater than equal to 0.5 stard deviations in the preceding 12 months The patient is aged between six months two years a though upper airway assessment is planned to be undertaken pri to treatment commencement at six to 12 weeks following treatment initiation RENEWAL - Prader-Willi syndrome Height velocity is greater than equal to 50th percentile (adjusted f bone age/pubertal status if appropriate) as calculated over 6 to 12 months using the stards of Tanner Davies (1985) Height velocity is greater than equal to 2 cm per year as calculated over six months A current bone age is 14 years under (female patients) 16 years under (male patients) No serious adverse effect that the patient's specialist considers is likely to be attributable to growth hmone treatment has occurred No malignancy has developed after growth hmone therapy was commenced The patient has not developed type II diabetes uncontrolled obesity as defined by BMI that has increased by greater than equal to 0.5 stard deviations in the preceding 12 months See also: INITIAL APPLICATION - adults adolescents p6 RENEWAL - adults adolescents p7 I confirm the above details are crect that in signing this fm I underst I may be audited.
Page 6 INITIAL APPLICATION - adults adolescents The patient has a medical condition that is known to cause growth hmone deficiency (e.g. surgical removal of the pituitary f treatment of a pituitary tumour) The patient has undergone appropriate treatment of other hmonal deficiencies psychological illnesses The patient has severe growth hmone deficiency (see notes) The patient's serum IGF-I is me than 1 stard deviation below the mean f age sex The patient has po quality of life, as defined by a sce of 16 me using the disease-specific quality of life questionnaire f adult growth hmone deficiency (QoL-AGHDA ) Note: F the purposes of adults adolescents, severe growth hmone deficiency is defined as a peak serum growth hmone level of less than equal to 3 mcg per litre during an adequately perfmed insulin tolerance test (ITT) glucagon stimulation test. Patients with one me additional anteri pituitary hmone deficiencies a known structural pituitary lesion only require one test. Patients with isolated growth hmone deficiency require two growth hmone stimulation tests, of which, one should be ITT unless otherwise contraindicated. Where an additional test is required, an arginine provocation test can be used with a peak serum growth hmone level of less than equal to 0.4 mcg per litre. The dose of somatropin should be started at 0.2 mg daily be titrated by 0.1 mg monthly until the serum IGF-I is within 1 stard deviation of the mean nmal value f age sex; Dose of somatropin not to exceed 0.7 mg per day f male patients, 1 mg per day f female patients. At the commencement of treatment f hypopituitarism, patients must be monited f any required adjustment in replacement doses of cticosteroid levothyroxine. See also: RENEWAL - adults adolescents p7 I confirm the above details are crect that in signing this fm I underst I may be audited.
Page 7 RENEWAL - adults adolescents The patient has been treated with somatropin f < 12 months There has been an improvement in Quality of Life defined as a reduction of at least 8 points on the Quality of Life Assessment of Growth Hmone Deficiency in Adults (QoL-AGHDA ) sce from baseline Serum IGF-I levels have been increased within ±1SD of the mean of the nmal range f age sex The dose of somatropin has not exceeded 0.7 mg per day f male patients, 1 mg per day f female patients The patient has been treated with somatropin f me than 12 months The patient has not had a deteriation in Quality of Life defined as a 6 point greater increase from their lowest QoL-AGHDA sce on treatment (other than due to obvious external facts such as external stresss) Serum IGF-I levels have continued to be maintained within ±1SD of the mean of the nmal range f age sex (other than f obvious external facts) The dose of somatropin has not exceeded 0.7 mg per day f male patients 1 mg per day f female patients I confirm the above details are crect that in signing this fm I underst I may be audited.