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 language | English |
|---|---|
| Pages (from-to) | 229-235 |
| Journal | Haemophilia |
| Volume | 24 |
| Issue number | 2 |
| DOIs | |
| Publication status | Published - 2018 |
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Keywords
- Clotting factor
- Donation
- Factor VIII
- Hemophilia
- Hemophilic arthropathy
- Humanitarian aid
- Public-private partnership
ASJC Scopus subject areas
- Hematology
- Genetics(clinical)
Cite this
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 journal › Article
}
TY - JOUR
T1 - First-year results of an expanded humanitarian aid programme for haemophilia in resource-constrained countries
AU - Pierce, G. F.
AU - Haffar, A.
AU - Ampartzidis, G.
AU - Peyvandi, F.
AU - Diop, S.
AU - El-Ekiaby, M.
AU - van den Berg, H. M.
PY - 2018
Y1 - 2018
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.
KW - Clotting factor
KW - Donation
KW - Factor VIII
KW - Hemophilia
KW - Hemophilic arthropathy
KW - Humanitarian aid
KW - Public-private partnership
UR - http://www.scopus.com/inward/record.url?scp=85043774957&partnerID=8YFLogxK
UR - http://www.scopus.com/inward/citedby.url?scp=85043774957&partnerID=8YFLogxK
U2 - 10.1111/hae.13409
DO - 10.1111/hae.13409
M3 - Article
AN - SCOPUS:85043774957
VL - 24
SP - 229
EP - 235
JO - Haemophilia
JF - Haemophilia
SN - 1351-8216
IS - 2
ER -