The purpose of this pilot study is to investigate the safety and preliminarily assess efficacy of early intervention with zoledronate in high risk pediatric hematopoietic stem cell transplantation (HSCT) patients to prevent the development of bone disease and fractures and reduce potential pain and suffering.
Patients ≥5 and ≤18 years old who are preparing for HSCT with a height-for-age corrected DXA Z-score of <-2.0 and admitted to a CCHMC inpatient unit.
Patients ≥5 and ≤18 years old recovering from HSCT and who have developed de novo acute or chronic GVHD and are admitted to a CCHMC inpatient unit.
Age <5 years and >18 years
Patients with Fanconi anemia or other radiation-sensitive syndromes with increased malignancy risk
history of prior bisphosphonate use
low 25-OH vitamin D levels (<20 ng/mL)
Single group
Zoledronate
DrugZoledronate is in the class of drugs entitled bisphosphonates which act to inhibit bone resorption by inhibiting osteoclast activity therefore reducing bone turnover.
Safety of early intervention Zoledronate
Calcium levels following infusion of zoledronate
Feasibility of early intervention Zoledronate
Number of days required for inpatient stay beyond what is necessary for the patient's admission
Bone health efficiency
DXA scans will be measured with a z-score comparing bone density to averages for patient age and height
Bone substrate turnover
c-terminal telopeptide (CTX) and procollagen type 1 n-terminal propeptide (P1NP) measured in pg/mL
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