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Vitamin D and Bisphosphonates in the Treatment of Sickle Cell Disease

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ClinicalTrials.gov Identifier: NCT02972138
Recruitment Status : Completed
First Posted : November 23, 2016
Last Update Posted : October 6, 2017
Sponsor:
Information provided by (Responsible Party):
Società Italiana Talassemie ed Emoglobinopatie

Study Description
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Brief Summary:
Sickle cell disease (SCD) is a worldwide distributed hereditary red cell disorder, which affects approximately 75,000 individuals in the United States and almost 20,000- 25,000 subjects in Europe, this latter mainly related to the immigration fluxes from endemic areas such as Sub-Saharian Africa to European countries. Studies of global burden disease have pointed out the invalidating impact of SCD on patient quality of life. This requires the development of new therapeutic options to treat sickle cell related acute and chronic complications. SCD is caused by a point mutation in the β-globin gene resulting in the synthesis of pathological hemoglobin S (HbS). HbS displays peculiar biochemical characteristics, polymerizing when deoxygenated with associated reduction in cell ion and water content (cell dehydration), increased red cell density and further acceleration of HbS polymerization. Pathophysiological studies have shown that dense, dehydrated red cells play a central role in acute and chronic clinical manifestations of SCD, in which intravascular sickling in capillaries and small vessels leads to vaso-occlusion and impaired blood flow with ischemic/reperfusion injury. In microcirculation, vaso-occlusive events (VOC) result from a complex and still partially known scenario, involving the interactions between different cell types, including dense red cells, reticulocytes, abnormally activated endothelial cells, leukocytes, platelets and plasma factors. Target organs, such as bone or lung, are involved in both acute and chronic clinical manifestations of SCD, related to their peculiar anatomic organization mainly characterized by sluggish circulation and relative local hypoxia. VOCs combined with marrow hyperplasia and inflammation has been suggested to contribute to the development of sickle bone disease (SBD). Recently, it has been proposed a possible role of vitamin D deficiency in SBD, which appears to be subordinated to the primary defect in bone homeostasis. In a humanized mouse model for SCD, we recently reported that SBD is due to imbalance between osteoblast/osteoclast activity induced by recurrent VOCs. In addition, we show that zoledronic acid prevents bone impairment related to SCD, reducing osteoclast activity and improving osteoblast performance.

Condition or disease
Sickle Cells Disease

Detailed Description:

This is a retrospective study aimed to evaluate sickle bone disease (SBD) in a population of young adult patients with sickle cell disease treated with vitamin D supplementation and anti-resorptive therapy (bisphosphonates). We plan to analyze data from 1 January 2010 to 31 December 2015.

In addition to the standard hematological analysis, the following parameters and radiologic exams will be evaluated:

  • Serum levels of Ca2+, P, Vitamin D, parathormone , creatinine, blood urea nitrogen , Na, K, Cl
  • Bone turnover markers: C-terminal telopeptide (CTX), N-terminal propeptide of type I procollagen (PINP)
  • Bone densitometry (DXA) at lumbar spine and proximal femur
  • Standard X-Ray at dorsal-lumbar spine in LL projection for morphometric analysis
Study Design
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Study Type : Observational
Actual Enrollment : 80 participants
Observational Model: Cohort
Time Perspective: Retrospective
Official Title: Vitamin D Supplementation and Anti-resorptive Therapy (Bisphosphonates) Treatment of Young Adult Patients With Sickle Cell Disease
Study Start Date : January 2010
Actual Primary Completion Date : December 2015
Actual Study Completion Date : September 2017

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Vitamin D
Drug Information available for: Vitamin D

Groups and Cohorts
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Outcome Measures
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Primary Outcome Measures :
  1. Incidence of vertebral fracture [ Time Frame: 5 years follow up ]

Secondary Outcome Measures :
  1. severity of vertebral fracture [ Time Frame: 5 years follow up ]
  2. Incidence of non-vertebral fracture [ Time Frame: 5 years follow up ]

Eligibility Criteria
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Ages Eligible for Study:   18 Years to 50 Years   (Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population
Patients with sickle cell disease older than 18 years of age and younger than 50 years of age
Criteria

Inclusion Criteria:

  • Young adult patients with sickle cell disease (older than 18 years of age) and younger than 50 years of age

Exclusion Criteria:

  • Women with positive pregnant test, patients with history of heart, renal and liver failure, patients taking drugs influencing bone metabolisms within the two years before the beginning of the study (i.e.: glucocorticoids, hormonal replacement)
  • Patients in meonopause, patients with traumatic vertebral fracture
  • Patients with hypo/hyperthyroidism
  • Patients with hyperparathyroidism
  • Patients with osteomalacia, patients with history of Paget disease
  • Patients with Cushing syndrome
  • Patients with malabsorption diseases (i.e.: caeliac disease)
  • Patients with history of cancer
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02972138


Locations
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Italy
Ospedali Galliera - S.S.D. Microcitemia, anemie congenite e dismetabolismo del ferro
Genova, Italy, 16128
Ospedali Galliera - S.S.D. Ortogeriatria per intensità di cure
Genova, Italy, 16128
Università degli Studi di Verona
Verona, Italy, 37129
Sponsors and Collaborators
Società Italiana Talassemie ed Emoglobinopatie
Investigators
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Principal Investigator: Luca G Dalle Carbonare, MD Università degli studi di Verona
More Information
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Publications:

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Responsible Party: Società Italiana Talassemie ed Emoglobinopatie
ClinicalTrials.gov Identifier: NCT02972138    
Other Study ID Numbers: Vit.D-Bisph-SCD
First Posted: November 23, 2016    Key Record Dates
Last Update Posted: October 6, 2017
Last Verified: December 2015
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Undecided
Keywords provided by Società Italiana Talassemie ed Emoglobinopatie:
Sickle-cells Disease
bisphosphonates
Vitamine D
Additional relevant MeSH terms:
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Anemia, Sickle Cell
Anemia, Hemolytic, Congenital
Anemia, Hemolytic
Anemia
 Hematologic Diseases
Hemoglobinopathies
Genetic Diseases, Inborn