Context
Hypoparathyroidism is a rare disease and as such, its natural history, long term complications and correct clinical management remain unclear.
Objective
To describe the natural history and clinical characteristics of the disease.
Design and setting
Topresent a retrospective observational analysis from seven specialized centers in Buenos Aires, Argentina.
Patients
chronic hypoparathyroid patients followed up between 1985 and December 2018.
Main Outcome Measures
data on demographics, etiology, clinical complications, biochemical parameters, DXA values and treatment doses were collected.
Results
322 subjects with chronic hypoparathyroidism were included, 85.7 % were female. Mean age was 55.2 ± 16.8 years and mean age at diagnosis was 43.8 ± 16.8. Prevalence of surgical hypoparathyroidism was 90.7 %, most common causes being thyroid carcinoma and benign thyroid disease. A history of hypocalcemia requiring hospitalization was present in 25.7 % and 4.3 % had a history of seizures. Overall, 40.9 % had reported at least one neuromuscular symptom. Renal insufficiency was present in 22.4 % and was significantly associated with age (p<0.0001). Hyperphosphatemia was present in 42 %. A history of severe hypocalcemia, paresthesias, tetany, ganglia calcifications, seizures and cataracts was significantly higher in nonsurgical patients.
Conclusion
Although these patients were followed up by experienced physicians, clinical management was heterogeneous and probably insufficient to assess all the potential complications of this chronic disease. Almost 70 % of this group of patients met the experts´ indications for considering the use of rhPTH 1-84. Being aware of this fact is the first step to improve our medical management of this disease in the future.
Objective: To describe the long term safety and efficacy of pegvisomant (PEGV), and the predictors of treatment response in patients with acromegaly in the real life setting. Subjects and methods: We retrospectively reviewed the clinical, hormonal and radiological data of acromegalic patients treated with PEGV in 17 Argentine centers. Results: Seventy-five patients (age range 22-77, 51 females) with acromegaly have been treated with PEGV for up to 118 months (median 27 months). Before PEGV, 97.3% of patients had been treated with medical therapy, surgery and/or radiotherapy, two patients had no previous treatment. At that time, all patients had an IGF-1 above the upper normal limit (ULN) (mean 2.4 x ULN ± 0.98, range 1.25-7). At diagnosis of acromegaly 84% presented macroadenomas, prior to PEGV only 23,5% of patients remained with tumor remnant > 1 cm, the remaining showed normal or less than 1 cm images. Disease control (IGF-1 ≤ 1.2 x ULN) was achieved in 62.9% of patients with a mean dose of 11.8 mg/day. Thirty-four patients (45%) received PEGV monotherapy, while 41 (55%) received combined therapy with either somatostatin analogues and/or cabergoline. Adverse events related to PEGV were: local injection site reaction in 5.3%, elevated liver enzymes in 9.3%, and tumor size growth in 9.8%. Pre-PEGV IGF-I level was the only predictor of treatment response: 2.1 x ULN vs 2.8 x ULN in controlled and uncontrolled patients respectively (p < 0.001). Conclusion: this long term experience indicates PEGV treatment was highly effective and safe in our series of Argentine patients with acromegaly refractory to standard therapies. Arch Endocrinol Metab.
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