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Is Low Dose a New Dose to Initiate Hemophilia A Prophylaxis? – A Systematic Study in Eastern India

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Abstract

Objectives

To investigate the effectiveness of low dose secondary/tertiary prophylaxis in severe Hemophilia A children and determine improvements in their daily life.

Methods

Thirty Hemophilia A children (≤ 12 y) with factor VIII <2% and less than two joint bleeds without inhibitors, were given prophylaxis with recombinant Fc fusion long acting factor VIII (ELOCTATE) at 10 IU.kg−1 twice weekly for 1 y. Earlier, patients received on-demand FVIII for a minimum of six months. Outcome was measured in terms of annual bleeding rate, Hemophilia Joint Health Score (HJHS) and child activity/participation was measured in terms of school absenteeism, School Activity Participation Score and Daily Activity Score according to Beijing Children Hospital assessment scale.

Results

A total of 30 children were included in the study. There was reduction in annual joint bleeds by 85.76% (14.5 to 2.2) and school absenteeism (days/month) by 86% (17.38 to 2.42) before and after prophylaxis respectively. Majority (43%) showed moderate improvement in daily activity score. Mean HJHS score was 8.3. There was mild improvement in School Activity Participation Score in 57%. Mean annual hospitalization rate reduced from 8.7 to 1.1 with improvement in joint scores. Mean annual factor consumption decreased from 1944.2 IU.kg−1 to 1560.3 IU.kg−1.

Conclusions

With low dose secondary/tertiary prophylaxis, there is significant reduction in the annual joint bleed rate with improvement in joint health and child activity. As factor consumption is reduced, this has a positive effect on cost benefit; and is a very feasible option in developing countries.

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Authors and Affiliations

  1. Department of Hematology, NRS Medical College, Kolkata, 700014, India

    Shazia Gulshan, Prakas Kumar Mandal, Abhijit Phukan, Shuvraneel Baul, Rajib De, Tuphan Kanti Dolai & Prantar Chakrabarti

Authors
  1. Shazia Gulshan
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  2. Prakas Kumar Mandal
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  3. Abhijit Phukan
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  4. Shuvraneel Baul
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  5. Rajib De
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  6. Tuphan Kanti Dolai
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  7. Prantar Chakrabarti
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Contributions

SG, PKM, PC: Concept, design, literature search, clinical studies, acquisition, analysis or interpretation of data, statistical analysis, manuscript preparation, manuscript editing and review; AP, SB, RD, TKD: Literature search, clinical studies, manuscript editing and review. PC will act as guarantor for this paper.

Corresponding author

Correspondence to Prakas Kumar Mandal.

Ethics declarations

Informed Consent

Informed consent was obtained from all individual participants and/or their legal guardians included in the study.

Conflict of Interest

None.

Source of Funding

The study was conducted with long acting recombinant FVIII Fc fusion (ELOCTATE, Biogen Inc., Cambridge, MA 02142) products from World Federation of Hemophilia (WFH) Humanitarian Aid.

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Gulshan, S., Mandal, P.K., Phukan, A. et al. Is Low Dose a New Dose to Initiate Hemophilia A Prophylaxis? – A Systematic Study in Eastern India. Indian J Pediatr 87, 345–352 (2020). https://doi.org/10.1007/s12098-019-03179-w

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  • DOI: https://doi.org/10.1007/s12098-019-03179-w

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