Haemophilia. 2019;00:1–8. wileyonlinelibrary.com/journal/hae | 1 © 2019 John Wiley & Sons Ltd
Received: 10 August 2019
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Revised: 25 November 2019
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Accepted: 3 December 2019
DOI: 10.1111/hae.13917
ORIGINAL ARTICLE
Effect of low-dose factor VIII prophylaxis therapy on bone
mineral density and 25(OH) vitamin D level in children with
severe haemophilia A
Nevine Gamal Andrawes
1
| Manal Hashem Fayek
2
| Nouran Salah El-Din
1
|
Raguia Atef Mostafa
3
1
Pediatrics Department, Ain Shams
University, Cairo, Egypt
2
Clinical Pathology Department, Ain Shams
University, Cairo, Egypt
3
Resident of Pediatrics, El Agouza
Specialized Hospital, Cairo, Egypt
Correspondence
Nouran Salah El Din, Pediatrics Department,
Ain Shams University, 25 korash street, Nasr
city, Cairo, Egypt.
Email: niron85@hotmail.com
Abstract
Background: Decreased bone mineral density (BMD) is a significant morbidity in hae-
mophilia. Vitamin D is important for the bone health of people with haemophilia.
Regular factor VIII prophylaxis can prevent bleeding and arthropathy.
Aim: To determine the 25(OH) vitamin D level in severe haemophilia A patients and
correlate it to their Hemophilia Joint Health Score (HJHS) and dual-energy X-ray ab-
sorptiometry (DEXA). We also compared the 25(OH) vitamin D and DEXA in hae-
mophilia A and healthy children and in haemophilia A children on prophylaxis versus
on-demand therapy.
Methods: Fifty severe haemophilia A patients were compared to 50 age-matched
healthy boys. Patients were recruited from the Pediatric Hematology Clinic, Ain
Shams University from May 2017 to April 2018. Full medical history was taken with
emphasis on frequency of bleeding episodes, duration and amplitude of pain as-
sessed by the pain score. Weight, height, body mass index and HJHS were assessed.
25(OH) vit-D3, calcium, phosphorus and alkaline phosphatase were measured. BMD
was assessed using Lunar DEXA, paediatric software.
Results: People with haemophilia had significantly lower 25(OH) vit-D3 (P < .001) and
DEXA z-score (P < .001) than controls. Seventy per cent of patients were on factor
VIII prophylaxis twice weekly (15U/kg/dose). Significant difference was found re-
garding DEXA z-score (P = .012), 25(OH) vit-D3 (P = .033) and HJHS (P = .022) among
patients on prophylaxis and on-demand therapy.
Conclusion: Severe haemophilia A patients showed significantly lower 25(OH) vit-D3
and DEXA than controls. Hence, vitamin D deficiency should be tested in all people
with haemophilia for early diagnosis and treatment. Low-dose prophylaxis in severe
haemophilia preserves BMD and increases vitamin D. Further studies are required
to evaluate the effect of different prophylaxis protocols on BMD and haemophilic
arthropathy.
KEYWORDS
bone mineral density, Factor VIII, prophylaxis therapy, severe haemophilia A, vitamin D