Quality of life in children and adolescents with growth hormone deficiency and their caregivers: an Italian survey.

PURPOSE: The aim of this study was to produce evidence on quality of life (QoL) among Italian growth hormone deficiency (GHD) children and adolescents treated with growth hormone (GH) and their parents. METHODS: A survey was conducted among Italian children and adolescents aged 4-18 with a confirmed diagnosis of GHD and treated with GH therapy and their parents. The European Quality of Life 5 Dimensions 3 Level Version (EQ-5D-3L) and the Quality of Life in Short Stature Youth (QoLISSY) questionnaires were administered between May and October 2021 through the Computer-Assisted Personal Interview (CAPI) method. Results were compared with national and international reference values. RESULTS: The survey included 142 GHD children/adolescents and their parents. The mean EQ-5D-3L score was 0.95 [standard deviation (SD) 0.09], while the mean visual analogue scale (VAS) score was 86.2 (SD 14.2); the scores are similar to those of a reference Italian population aged 18-24 of healthy subjects. As for the QoLISSY child-version, compared to the international reference values for GHD/ idiopathic short stature (ISS) patients, we found a significantly higher score for the physical domain, and lower scores for coping and treatment; compared to the specific reference values for GHD patients, our mean scores were significantly lower for all domains except the physical one. As for the parents, we found a significantly higher score for the physical domain, and a lower score for treatment; compared to reference values GHD-specific, we found lower score in the social, emotional, treatment, parental effects, and total score domains. CONCLUSIONS: Our results suggest that the generic health-related quality of life (HRQoL) in treated GHD patients is high, comparable to that of healthy people. The QoL elicited by a disease specific questionnaire is also good, and comparable with that of international reference values of GHD/ISS patients.

[Male osteoporosis: current treatments and future options]. / L'osteoporosi maschile: possibilità terapeutiche attuali e prospettive future.

PURPOSE: To provide indications for currently approved treatments and future options for male osteoporosis, based on the pathogenetic mechanisms of bone loss in the male sex. DESIGN: Review of the most significant data reported in the literature. RESULTS: Unhealthy lifestyle habits should be modified. Testosterone replacement is indicated only in patients with diagnosis of hypogonadism. Based on the demonstrated pathogenetic role of estrogen lack in bone loss in men, either low doses of this hormone or selective androgen receptor modulators have been proposed for the treatment of male osteoporosis. Bisphosphonates are the only medications approved by Food and Drug Administration for idiopathic and glucocorticoid-induced osteoporosis in men. As far as anabolic treatments are concerned, there is not agreement on clinical utility of sodium fluoride. Short-term treatment with parathyroid hormone (PTH) seems to be safe and effective in patients with idiopathic osteoporosis, due to its anabolic action. Therapeutic use of growth hormone (GH) and insulin-like growth factor type I (IGF-I), both considered as potential anabolic agents, is still limited because of the high incidence of side effects and relatively transient efficacy, particularly of IGF-I. CONCLUSIONS: Treatments should be selected on the basis of anti-fracture efficacy of various medications, which has been demonstrated so far only for alendronate and risedronate. Although anabolic agents produce noticeable increase of bone mineral density, is still debated if they also reduce fracture incidence in males with osteoporosis.

Latissimus dorsi transfer for primary treatment of irreparable rotator cuff tears.

Transfer of the musculotendinous unit of the latissimus dorsi was performed in seven patients (5 men and 2 women, with a mean age of 57 years) with irreparable rotator cuff tear who had had no previous surgery for cuff repair. Preoperatively, the mean active shoulder motion was 86° in flexion, 74° in abduction and 22° in external rotation. One patient had a positive lift-off test. The average preoperative Constant and Murley score was 44%. Diagnosis of irreparability of the cuff leasion was made preoperatively only in one case. In the remaining patients, the preoperative data only led to suspect that the tear was irreparable. At surgery, all patients had an irreparable tear of the superoinferior portion of the cuff and one patient also had a tear of the subscapularis tendon. In all cases the latissimus dorsi tendon was inserted to the greater tuberosity and, in four cases, to the subscapularis tendon; in three patients it was sutured to the bicipital tendon. Postoperatively all patients had relief of shoulder pain. The mean improvement in active flexion, abduction and external rotation was, respectively, 39°, 29° and 10°: At the latest follow-up, the average Constant and Murley score was 64%. The results of surgery were rated as excellent in three cases, good in two, fair in one and poor in one. All patients but one returned to preoperative work. Transfer of the latissimus dorsi muscle is an effective procedure for patients in middle or early elderly age who have an irreparable tear of the supraspinatus and infraspinatus tendons.

Increased serum levels of carboxyterminal propeptide of type 1 collagen (PICP) in hyperthyroidism.

Type 1 collagen is the major organic constituent of the bone: its synthesis is reflected by the serum levels of type 1 procollagen C-terminal propeptide (PICP), which is therefore considered an index of osteoblastic activity. Serum PICP along with other serum and urinary markers of bone metabolism were measured in 16 untreated premenopausal females affected by Graves' disease and also in 7 of them after attainment of euthyroidism by methimazole treatment. Before treatment PICP was higher than sex and age-matched controls (324.19 +/- 101.74 vs. 131.44 +/- 26.25 micrograms/l, p < 0.001). Osteocalcin, alkaline phosphatase, serum calcium and urinary excretions of calcium and hydroxyproline were significantly increased with respect to controls, whereas parathormone was lower. Treatment induced a significant decrease of PICP, as well as calcemia, calciuria and hydroxyprolinuria compared to pretreatment values, while osteocalcin and alkaline phosphatase did not significantly differ. Non parametric correlation analysis showed positive correlation of free T3 and PICP (rs = 0.73, p < 0.005), osteocalcin and alkaline phosphatase; PICP was also significantly correlated with osteocalcin and alkaline phosphatase. Our data suggest that hyperthyroidism due to Graves' disease causes an increase of serum concentrations of PICP, which decrease after attainment of euthyroidism. The differences between PICP and BGP as markers of bone synthesis need to be further clarified.

Clodronate treatment increases serum osteocalcin in normocalcemic osteolytic bone metastases.

28 consecutive patients with multiple osteolytic bone metastases due to various types of cancer were treated with 300 mg/day of sodium clodronate given intravenously as a 3-hour infusion, 16 patients were treated for 10 days and 12 for 20 days. Our findings showed that clodronate administration causes an increase in serum osteocalcin (sBGP) in the group of patients treated for 20 days (p less than 0.05), a reduction in serum Ca in both groups of patients (p less than 0.01), an increase in serum alkaline phosphatase (p less than 0.05 and p less than 0.01), a reduction in urinary Ca (p less than 0.01) and a reduction in uOHP (p less than 0.05). The raise in sBGP may be attributed to the reduction of sCa and to the consequent secondary hyperparathyroidism. The increase in sBGP can be considered as the expression of osteoblasts stimulation following an adequate period of therapy with sodium clodronate.

[Serum osteocalcin in hyperthyroidism]. / L'osteocalcina sierica nell'ipertiroidismo.

Several bone metabolism biochemical parameters were measured to evaluate the increased serum osteocalcin (sBGP) in hyperthyroidism. Twenty patients (19 women and 1 man, aged 22-69) and 20 age and sex-matched healthy subjects were examined. The following serum measurements were performed: calcium, phosphate, mid-molecule PTH, calcitonin, 25OH vitD, alkaline phosphatase, total and free thyroid hormones; urinary excretion of calcium, hydroxyproline and creatinine was also measured. The results (mean +/- standard error) show significant increases of sBGP (16.4 +/- 1.02 ng/mL; p less than 0.001), serum calcium (10.1 +/- 0.1 mg/dL; p less than 0.001), alkaline phosphatase (144.0 +/- 11.7 UI/L; p less than 0.001), urinary calcium (315.6 +/- 48.5 mg/g urinary creatinine; p less than 0.001) and hydroxyproline (43.0 +/- 6.1 mg/g urinary creatinine; p less than 0.001). A significant correlation between total and free thyroid hormones and sBGP was found. Accelerated bone turnover in hyperthyroid patients is therefore confirmed: both osteoclastic bone resorption, as suggested by increased serum and urinary calcium and urinary hydroxyproline, and osteoblastic bone formation, as suggested by increased serum osteocalcin and alkaline phosphatase, are stimulated.

Serum bone GLA-protein in hypercalcemia of primary hyperparathyroidism and malignancy.

Serum bone GLA-protein, a modern and sensitive marker of bone turnover, was measured in 15 patients with primary hyperparathyroidism, 18 patients with hypercalcemia of malignancy, 41 patients with bone metastasis without hypercalcemia, and 29 healthy subjects. Serum bone GLA-protein was increased in primary hyperparathyroidism (17.6 +/- 3.9 ng/ml) and normal in hypercalcemia of malignancy (5.2 +/- 2.8 ng/ml; p less than 0.001 vs hyperparathyroidism) and in normocalcemic patients with bone metastases. In primary hyperparathyroidism parathyroid hormone correlated positively with urinary calcium excretion (p less than 0.05) and with urinary hydroxyproline excretion (p less than 0.001). The sensitivity of serum bone GLA-protein measurements in differentiating between primary hyperparathyroidism and hypercalcemia of malignancy was 91% and the specificity 84%. Thus this marker appears to be a useful tool for the differential diagnosis of hypercalcemias.

Hypercalcemia in malignancies: treatment with dichloromethylene diphosphonate (Cl2MDP).

Eleven hypercalcemic patients affected by various kinds of cancer were treated with dichloromethylene diphosphonate (Cl2MDP), 400 mg diluted in 500 ml of saline solution, i.v., during 3 h. Drug administration normalized plasma calcium levels in all treated patients, within 72 h for patients with lung cancer; within a longer period for other kinds of cancer. Moreover, Cl2MDP also reduced calciuria in treated patients. The use of the drug was not followed by side effects and was also well tolerated in patients with kidney insufficiency.