First-year results of an expanded humanitarian aid programme for haemophilia in resource-constrained countries

G. F. Pierce, A. Haffar, G. Ampartzidis, F. Peyvandi, S. Diop, M. El-Ekiaby, H. M. van den Berg

Research output: Contribution to journalArticle

4 Citations (Scopus)

Abstract

Introduction: The gaps in haemophilia treatment around the world are enormous; approximately 60% of an estimated 475 000 individuals are not identified. Of the 187 000 diagnosed, 30% (57 000) access clotting factor replacement therapy. Since 1996, humanitarian aid distributed by the World Federation of Hemophilia (WFH) has played a minor, yet vital role providing life-saving clotting factor to countries in emergency situations. Donated amounts have been small and sporadic, often salvaging short-dated products, providing little opportunity to leverage donations with governments. In 2015, a prospective donation programme of >100 million IU per year of extended half-life factor VIII and IX over 10 years was established, necessitating the development of new logistics and training programmes. Aim: To measure the impact of a greatly expanded hemophilia humanitarian aid program. Materials and methods: In 2016, the first full year of the expanded programme, WFH, distributed products to 58 countries with factor VIII usage <1 I.U. per capita, a level incompatible with long-term survival and far below the 4 I.U. FVIII per capita minimum established in Europe. Results: The scope of the programme and utilization data for 2016 indicate primarily use for acute bleeding, orthopaedic and emergency surgeries. Compared to 2014, 2016 data showed substantial increases in patients served (5.9-fold, from 2119 to 14 579), surgeries performed (37-fold) and bleeds treated (6.9-fold). Patients on prophylaxis rose from 0 to 852, including 458 children under 10 years old. Discussion: The expanded humanitarian aid programme impacts an estimated 10% of individuals with haemophilia previously unable to access treatment. Conclusion: This programme represents an unprecedented public-private partnership to deliver medicines to individuals with no access. Further, the programme offers the prospective opportunity to engage governments to take more responsiblity for increasing training, medical management, and product supply in 58 resource constrained countries.

Original languageEnglish
Pages (from-to)229-235
JournalHaemophilia
Volume24
Issue number2
DOIs
Publication statusPublished - 2018

Fingerprint

Hemophilia A
Blood Coagulation Factors
Factor VIII
Emergencies
Public-Private Sector Partnerships
Factor IX
Orthopedics
Half-Life
Therapeutics
Hemorrhage
Education
Survival

Keywords

  • Clotting factor
  • Donation
  • Factor VIII
  • Hemophilia
  • Hemophilic arthropathy
  • Humanitarian aid
  • Public-private partnership

ASJC Scopus subject areas

  • Hematology
  • Genetics(clinical)

Cite this

Pierce, G. F., Haffar, A., Ampartzidis, G., Peyvandi, F., Diop, S., El-Ekiaby, M., & van den Berg, H. M. (2018). First-year results of an expanded humanitarian aid programme for haemophilia in resource-constrained countries. Haemophilia, 24(2), 229-235. https://doi.org/10.1111/hae.13409

First-year results of an expanded humanitarian aid programme for haemophilia in resource-constrained countries. / Pierce, G. F.; Haffar, A.; Ampartzidis, G.; Peyvandi, F.; Diop, S.; El-Ekiaby, M.; van den Berg, H. M.

In: Haemophilia, Vol. 24, No. 2, 2018, p. 229-235.

Research output: Contribution to journalArticle

Pierce, GF, Haffar, A, Ampartzidis, G, Peyvandi, F, Diop, S, El-Ekiaby, M & van den Berg, HM 2018, 'First-year results of an expanded humanitarian aid programme for haemophilia in resource-constrained countries', Haemophilia, vol. 24, no. 2, pp. 229-235. https://doi.org/10.1111/hae.13409
Pierce, G. F. ; Haffar, A. ; Ampartzidis, G. ; Peyvandi, F. ; Diop, S. ; El-Ekiaby, M. ; van den Berg, H. M. / First-year results of an expanded humanitarian aid programme for haemophilia in resource-constrained countries. In: Haemophilia. 2018 ; Vol. 24, No. 2. pp. 229-235.
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AU - Haffar, A.

AU - Ampartzidis, G.

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AU - Diop, S.

AU - El-Ekiaby, M.

AU - van den Berg, H. M.

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N2 - Introduction: The gaps in haemophilia treatment around the world are enormous; approximately 60% of an estimated 475 000 individuals are not identified. Of the 187 000 diagnosed, 30% (57 000) access clotting factor replacement therapy. Since 1996, humanitarian aid distributed by the World Federation of Hemophilia (WFH) has played a minor, yet vital role providing life-saving clotting factor to countries in emergency situations. Donated amounts have been small and sporadic, often salvaging short-dated products, providing little opportunity to leverage donations with governments. In 2015, a prospective donation programme of >100 million IU per year of extended half-life factor VIII and IX over 10 years was established, necessitating the development of new logistics and training programmes. Aim: To measure the impact of a greatly expanded hemophilia humanitarian aid program. Materials and methods: In 2016, the first full year of the expanded programme, WFH, distributed products to 58 countries with factor VIII usage <1 I.U. per capita, a level incompatible with long-term survival and far below the 4 I.U. FVIII per capita minimum established in Europe. Results: The scope of the programme and utilization data for 2016 indicate primarily use for acute bleeding, orthopaedic and emergency surgeries. Compared to 2014, 2016 data showed substantial increases in patients served (5.9-fold, from 2119 to 14 579), surgeries performed (37-fold) and bleeds treated (6.9-fold). Patients on prophylaxis rose from 0 to 852, including 458 children under 10 years old. Discussion: The expanded humanitarian aid programme impacts an estimated 10% of individuals with haemophilia previously unable to access treatment. Conclusion: This programme represents an unprecedented public-private partnership to deliver medicines to individuals with no access. Further, the programme offers the prospective opportunity to engage governments to take more responsiblity for increasing training, medical management, and product supply in 58 resource constrained countries.

AB - Introduction: The gaps in haemophilia treatment around the world are enormous; approximately 60% of an estimated 475 000 individuals are not identified. Of the 187 000 diagnosed, 30% (57 000) access clotting factor replacement therapy. Since 1996, humanitarian aid distributed by the World Federation of Hemophilia (WFH) has played a minor, yet vital role providing life-saving clotting factor to countries in emergency situations. Donated amounts have been small and sporadic, often salvaging short-dated products, providing little opportunity to leverage donations with governments. In 2015, a prospective donation programme of >100 million IU per year of extended half-life factor VIII and IX over 10 years was established, necessitating the development of new logistics and training programmes. Aim: To measure the impact of a greatly expanded hemophilia humanitarian aid program. Materials and methods: In 2016, the first full year of the expanded programme, WFH, distributed products to 58 countries with factor VIII usage <1 I.U. per capita, a level incompatible with long-term survival and far below the 4 I.U. FVIII per capita minimum established in Europe. Results: The scope of the programme and utilization data for 2016 indicate primarily use for acute bleeding, orthopaedic and emergency surgeries. Compared to 2014, 2016 data showed substantial increases in patients served (5.9-fold, from 2119 to 14 579), surgeries performed (37-fold) and bleeds treated (6.9-fold). Patients on prophylaxis rose from 0 to 852, including 458 children under 10 years old. Discussion: The expanded humanitarian aid programme impacts an estimated 10% of individuals with haemophilia previously unable to access treatment. Conclusion: This programme represents an unprecedented public-private partnership to deliver medicines to individuals with no access. Further, the programme offers the prospective opportunity to engage governments to take more responsiblity for increasing training, medical management, and product supply in 58 resource constrained countries.

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