Teriparatide (rhPTH 1–34) treatment in the pediatric age: long-term efficacy and safety data in a cohort with genetic hypoparathyroidism
Hypoparathyroidism is characterized by the absence or inadequately low circulating concentrations of the parathyroid hormone, resulting in hypocalcemia, hyperphosphatemia, and elevated fractional excretion of calcium in the urine. The use of activated vitamin D analogs and calcium supplements represent conventional therapy. Subcutaneous recombinant human parathormone [rhPTH(1–34)] has been proposed as a substitutive treatment, even to avoid side effects of vitamin D and calcium.
To assess the long-term safety and efficacy of rhPTH(1–34) in a pediatric cohort of patients with genetic hypoparathyroidism.
The study is a 9.2-year self-controlled study of six pediatric patients (four males and two females, aged 9.4 ± 5.2) with DiGeorge, hypoparathyroidism-deafness-renal dysplasia (HDR) or autoimmune-candidiasis-polyendocrinopathy-ectodermal-dysplasia (APECED) syndrome, associated with autoimmune intestinal malabsorption in a patient. The presence of clinical signs of hypocalcemia and biochemical parameters, such as calcium, phosphate, alkaline phosphatase in the blood and calcium–creatinine ratio in urine, were compared during conventional treatment and rhPTH(1–34) (teriparatide, 12.5 μg twice daily).
The rhPTH(1–34) treatment allowed a reduction, although not always a complete suspension, of calcium supplementation and a slight reduction of calcitriol therapy. The number of tetanic episodes was reduced in four patients during the rhPTH(1–34) treatment. Mean blood calcium, alkaline phosphatase, and phosphate did not significantly change, while a significant reduction of the urinary calcium-to-creatinine ratio (0.55 ± 0.32 vs 0.16 ± 0.09, p = 0.03) was obtained. Renal ultrasound examination showed a worsening in three patients, while it did not change in the remaining three subjects during the follow-up.
In children with syndromic hypoparathyroidism presented here, replacement therapy with rhPTH(1–34) allowed to maintain adequate levels of the calcium and phosphate in the blood, normalize urinary calcium excretion, and reduce tetanic episodes. In patients with low compliance to conventional therapy or intestinal malabsorption, the use of rhPTH(1–34) could be considered, also to reduce the side effects of treatment with vitamin D and calcium.
KeywordsTeriparatide Children Hypoparathyroidism
All authors whose names appear on the submission have contributed sufficiently to the scientific work and therefore share collective responsibility and accountability for the results (G.T., R.B. and L.D.S. contributed to the study design, the manuscript writing and final revisions; D.T., S.E., P.M. contributed to the study design and the conceptual part of this manuscript)
Compliance with ethical standards
Conflict of interest
The authors declare that they have no conflict of interest.
All procedures performed in studies involving human participants were in accordance with the ethical standards of the institutional and/or national research committee and with the 1964 Helsinki declaration and its later amendments or comparable ethical standards. This article does not contain any studies with animals performed by any of the authors.
Informed consent was obtained from all parents of the individual participants included in the study.
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