Bisphosphonate treatment on bone metabolism in children with osteogenesis imperfecta

Doctoral Dissertation uoadl:2778778 285 Read counter

Τομέας Υγείας - Μητέρας - Παιδιού
Library of the School of Health Sciences
Deposit date:
Papaphylactou Maria
Dissertation committee:
Παπαευαγγέλου Βασιλική, Καθηγήτρια, Ιατρική, ΕΚΠΑ
Κανακά-Gantenbein Χριστίνα, Καθηγήτρια, Ιατρική, ΕΚΠΑ
Παπαδημητρίου Αναστάσιος, Καθηγητής, Ιατρική, ΕΚΠΑ
Πρίφτης Κωνσταντίνος, Αναπληρωτής Καθηγητής, Ιατρική, ΕΚΠΑ
Καραβανάκη Κυριακή, Αναπληρώτρια Καθηγήτρια, Ιατρική, ΕΚΠΑ
Αττιλἀκος Αχιλλέας, Επίκουρος Καθηγητής, Ιατρική, ΕΚΠΑ
Κασίμος Δημήτριος, Επίκουρος Καθηγητής, Ιατρική, ΕΚΠΑ
Original Title:
Επίδραση της θεραπείας με διφωσφονικά στον οστικό μεταβολισμό σε παιδιά με ατελή οστεογένεση
Translated title:
Bisphosphonate treatment on bone metabolism in children with osteogenesis imperfecta
Osteogenesis imperfecta (OI) is a phenotypically and molecularly heterogenous group of inherited connective tissue disorders that share similar skeletal abnormalities causing bone fragility and deformity, leading to growth deficiency.
Bisphosphonates are potent antiresorptive agents that inhibit osteoclast function.
We aimed to assess in children with OI the effects of treatment with bisphosphonates on the number of fractures, bone mass density (BMD), markers of bone metabolism and receptor activator of nuclear factor-κB (sRANKL) and osteoprotegerin (OPG) levels.
Ten children with a clinical diagnosis of OI, median age 8.12 years, were included in the study. The patients received pamidronate infusions on two consecutive days, administered at age-dependent intervals of eight to sixteen weeks or zoledronic acid once every six months, for a period of twelve months. Clinical changes were evaluated regularly during treatment and changes of BMD were assessed after twelve months of treatment. Serum levels of biochemical markers (Ca, iCa, P, Cr, PTH, 25-OH-D), markers of bone formation (total ALP, bALP), markers of bone resorption (CTX, uCa, PYD, DPD) and sRANKL and OPG were measured before and 48 hours after each bisphosphonate course.
Bisphosphonate administration was safe and well tolerated by the patients. Patients reported increase in appetite, decrease in sweating, relief of chronic pain and fatigue and increase in functional capacity.
Anthropometric measurements did not change. New fractures did not occur, except in one patient.
During bisphosphonate treatment vertebral shape and size increased and BMD increased significantly.
Both markers of bone resorption and bone formation decreased, while serum levels of sRANKL and OPG remained unchanged.
In conclusion, in children with OI administration of bisphosphonates for a period of twelve months had a positive impact on skeletal health.
Main subject category:
Health Sciences
Osteogenesis imperfecta, Bisphosphonates, Bone metabolism
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