Pediatric Hematology/Oncology and ImmunopathologyPediatric Hematology/Oncology and ImmunopathologyВопросы гематологии/онкологии и иммунопатологии в педиатрии1726-17082414-9314Fund Doctors, Innovations, Science for Children77910.24287/1726-1708-2024-23-4-58-61ORIGINAL ARTICLESОРИГИНАЛЬНЫЕ СТАТЬИThe retrospective study of cost-effectiveness for prophylactic and on-demand treatment of the pediatric patients with hemophilia A in IranThe retrospective study of cost-effectiveness for prophylactic and on-demand treatment of the pediatric patients with hemophilia A in IranAlidoustTina
Iran, Islamic Republic of

Ahvaz

dr.alidoust@gmail.comhttps://orcid.org/0000-0002-7106-9799BahadoramMohammad
Iran, Islamic Republic of

Ahvaz

Mohammadbahadoram@yahoo.comhttps://orcid.org/0000-0003-3482-2195AlghasiArash
Iran, Islamic Republic of

Ahvaz

arashalqasi@yahoo.comhttps://orcid.org/0000-0002-1096-5661Mahmoudian-SaniMohammad-Reza
Iran, Islamic Republic of

Ahvaz

mohamadsani495@gmail.comhttps://orcid.org/0000-0001-7722-0165AkadeEsma’il
Iran, Islamic Republic of

Ahvaz

eakadeh@gmail.comNilsaz-DezfouliHamid
Iran, Islamic Republic of

Mahshahr

keikhaeib@yahoo.comhttps://orcid.org/0000-0002-3087-7650KeikhaeiBijan
Iran, Islamic Republic of

Dr. Bijan Keikhaei Address: Golestan Highway

Ahvaz

keikhaeib@yahoo.comThalassemia and Hemoglobinopathy Research Center, Health Research Institute, Ahvaz Jundishapur University of Medical SciencesDepartment of industrial Engineering, Mahshahr Branch, Islamic Azad University1312202423458610611202315122023Copyright ©; 2024, «D. Rogachev NMRCPHOI»Copyright ©; 2024, ФГБУ «НМИЦ ДГОИ им. Дмитрия Рогачева» Минздрава России2024«D. Rogachev NMRCPHOI»ФГБУ «НМИЦ ДГОИ им. Дмитрия Рогачева» Минздрава Россииhttps://creativecommons.org/licenses/by/4.0https://hemoncim.com/jour/article/view/779

Hemophilia is the second most common inherited bleeding disorder after von Willebrand disease. Delayed clotting is usually observed in all main types of hemophilia caused by deficiencies in blood coagulation factors. Primary prophylaxis is considered the gold standard to prevent acute hemarthrosis and chronic arthropathy in patients with severe hemophilia A. However, some low-income countries, like Iran, have to prioritize access to preventive care to balance the financial resources and per capita access to coagulation factors. In order to compare the cost-effectiveness of on-demand and preventive treatment, we conducted a retrospective study on 55 patients with hemophilia A. We collected data from two groups of patients: those who had received routine prophylactic treatment and those who had received on-demand (episodic) treatment. The results of our study revealed a significant difference in the annualized bleeding rates (ABRs) between the two groups: ABR in the prophylactic treatment group was lower than in the on-demand treatment group (2.19 vs 7.25). In addition, we found substantial differences between the prophylactic and on-demand treatment groups in the number of hospital visits per year (9.8 vs 14.41), the annual number of infusions (107.35 vs 229.58), and in the mean cost of treatment (30.96€ vs 63€). In conclusion, prophylactic therapy seems to be more effective than on-demand treatment. This study was conducted in accordance with the principles of the Declaration of Helsinki. The study was approved by the Ethics Committee of the Ahvaz Jundishapur University of Medical Sciences, Ahvaz, Iran (IR.AJUMS.REC.1398.456). Written informed consent was obtained from the patients’ parents.

Hemophilia is the second most common inherited bleeding disorder after von Willebrand disease. Delayed clotting is usually observed in all main types of hemophilia caused by deficiencies in blood coagulation factors. Primary prophylaxis is considered the gold standard to prevent acute hemarthrosis and chronic arthropathy in patients with severe hemophilia A. However, some low-income countries, like Iran, have to prioritize access to preventive care to balance the financial resources and per capita access to coagulation factors. In order to compare the cost-effectiveness of on-demand and preventive treatment, we conducted a retrospective study on 55 patients with hemophilia A. We collected data from two groups of patients: those who had received routine prophylactic treatment and those who had received on-demand (episodic) treatment. The results of our study revealed a significant difference in the annualized bleeding rates (ABRs) between the two groups: ABR in the prophylactic treatment group was lower than in the on-demand treatment group (2.19 vs 7.25). In addition, we found substantial differences between the prophylactic and on-demand treatment groups in the number of hospital visits per year (9.8 vs 14.41), the annual number of infusions (107.35 vs 229.58), and in the mean cost of treatment (30.96€ vs 63€). In conclusion, prophylactic therapy seems to be more effective than on-demand treatment. This study was conducted in accordance with the principles of the Declaration of Helsinki. The study was approved by the Ethics Committee of the Ahvaz Jundishapur University of Medical Sciences, Ahvaz, Iran (IR.AJUMS.REC.1398.456). Written informed consent was obtained from the patients’ parents.

hemophiliaprophylaxison-demandbleedingretrospective studyhemophiliaprophylaxison-demandbleedingretrospective study1. Sarangi S.N., Acharya S.S. Bleeding disorders in congenital syndromes. Pediatrics 2017; 139 (2): e20154360.Sarangi S.N., Acharya S.S. Bleeding disorders in congenital syndromes. Pediatrics 2017; 139 (2): e20154360.2. Rostami S., Namaki Khameneh S., Keikhaei Dehdezi B., Haghighizadeh M.H. The Effect of Family-centered Empowerment on Quality of Life in Children with Hemophilia. Avicenna J Nurs Midwifery Care 2020; 28 (1): 1–10.Rostami S., Namaki Khameneh S., Keikhaei Dehdezi B., Haghighizadeh M.H. The Effect of Family-centered Empowerment on Quality of Life in Children with Hemophilia. Avicenna J Nurs Midwifery Care 2020; 28 (1): 1–10.3. Ay C., Perschy L., Rejtö J., Kaider A., Pabinger I. Treatment patterns and bleeding outcomes in persons with severe hemophilia A and B in a realworld setting. Ann Hematol 2020; 99 (12): 2763–71.Ay C., Perschy L., Rejtö J., Kaider A., Pabinger I. Treatment patterns and bleeding outcomes in persons with severe hemophilia A and B in a realworld setting. Ann Hematol 2020; 99 (12): 2763–71.4. Castaman G., Matino D. Hemophilia A and B: molecular and clinical similarities and differences. Haematologica 2019; 104 (9): 1702–9.Castaman G., Matino D. Hemophilia A and B: molecular and clinical similarities and differences. Haematologica 2019; 104 (9): 1702–9.5. Thornburg C.D., Duncan N.A. Treatment adherence in hemophilia. Patient Prefer Adherence 2017; 11: 1677.Thornburg C.D., Duncan N.A. Treatment adherence in hemophilia. Patient Prefer Adherence 2017; 11: 1677.6. Mannucci P.M. Hemophilia therapy: the future has begun. Haematologica 2020; 105 (3): 545–53.Mannucci P.M. Hemophilia therapy: the future has begun. Haematologica 2020; 105 (3): 545–53.7. Nugent D., O'Mahony B., Dolan G.; International Haemophilia Access Strategy Council. Value of prophylaxis vs on‐demand treatment: Application of a value framework in hemophilia. Haemophilia 2018; 24 (5): 755–65.Nugent D., O'Mahony B., Dolan G.; International Haemophilia Access Strategy Council. Value of prophylaxis vs on‐demand treatment: Application of a value framework in hemophilia. Haemophilia 2018; 24 (5): 755–65.8. Lobet S., Hermans C., Lambert C. Optimal management of hemophilic arthropathy and hematomas. J Blood Med 2014; 5: 207–18.Lobet S., Hermans C., Lambert C. Optimal management of hemophilic arthropathy and hematomas. J Blood Med 2014; 5: 207–18.9. Mancuso M.E.E., Reding M., Negrier C., Kerlin B., Rajgarajan S., Simpson M.L. Decreased Bleeding Rates in Patients with Hemophilia A Switching from Standard-half-life FVIII to BAY 94-9207 Prophylaxis. Thromb Haemost 2021; 121 (8): 1079–86.Mancuso M.E.E., Reding M., Negrier C., Kerlin B., Rajgarajan S., Simpson M.L. Decreased Bleeding Rates in Patients with Hemophilia A Switching from Standard-half-life FVIII to BAY 94-9207 Prophylaxis. Thromb Haemost 2021; 121 (8): 1079–86.10. Krishnan S., Vietri J., Furlan R., Duncan N. Adherence to prophylaxis is associated with better outcomes in moderate and severe haemophilia: results of a patient survey. Haemophilia 2015; 21 (1): 64–70.Krishnan S., Vietri J., Furlan R., Duncan N. Adherence to prophylaxis is associated with better outcomes in moderate and severe haemophilia: results of a patient survey. Haemophilia 2015; 21 (1): 64–70.11. Manco-Johnson M.J., Soucie J.M., Gill J.C. Joint Outcomes Committee of the Universal Data Collection USHTCN. Prophylaxis usage, bleeding rates, and joint outcomes of hemophilia, 1999 to 2010: a surveillance project. Blood 2017; 129 (17): 2368–74.Manco-Johnson M.J., Soucie J.M., Gill J.C. Joint Outcomes Committee of the Universal Data Collection USHTCN. Prophylaxis usage, bleeding rates, and joint outcomes of hemophilia, 1999 to 2010: a surveillance project. Blood 2017; 129 (17): 2368–74.12. Ljung R. Aspects of prophylactic treatment of hemophilia. Thromb J 2016; 14 (1): 30.Ljung R. Aspects of prophylactic treatment of hemophilia. Thromb J 2016; 14 (1): 30.13. Castaman G., Linari S. Prophylactic versus on-demand treatments for hemophilia: advantages and drawbacks. Expert Rev Hematol 2018; 11 (7): 567–76.Castaman G., Linari S. Prophylactic versus on-demand treatments for hemophilia: advantages and drawbacks. Expert Rev Hematol 2018; 11 (7): 567–76.14. Tang L., Wu R., Sun J., Zhang X., Feng X., Zhang X., et al. Short‐term low‐dose secondary prophylaxis for severe/moderate haemophilia A children is beneficial to reduce bleed and improve daily activity, but there are obstacle in its execution: a multi‐centre pilot study in China. Haemophilia 2013; 19 (1): 27–34.Tang L., Wu R., Sun J., Zhang X., Feng X., Zhang X., et al. Short‐term low‐dose secondary prophylaxis for severe/moderate haemophilia A children is beneficial to reduce bleed and improve daily activity, but there are obstacle in its execution: a multi‐centre pilot study in China. Haemophilia 2013; 19 (1): 27–34.15. Zhao Y., Xiao J., Yang R., Wu R., Hu Y., Beckmann H., et al. Efficacy of standard prophylaxis versus on-demand treatment with bayer's sucrose-formulated recombinant FVIII (rFVIII-FS) in Chinese children with severe hemophilia A. Pediatr Hematol Oncol 2017; 34 (3): 146–56.Zhao Y., Xiao J., Yang R., Wu R., Hu Y., Beckmann H., et al. Efficacy of standard prophylaxis versus on-demand treatment with bayer's sucrose-formulated recombinant FVIII (rFVIII-FS) in Chinese children with severe hemophilia A. Pediatr Hematol Oncol 2017; 34 (3): 146–56.16. Gringeri A., Lundin B., Von Mackensen S., Mantovani L., Mannucci P., Group E.S. A randomized clinical trial of prophylaxis in children with hemophilia A (the ESPRIT Study). J Thromb Haemost 2011; 9 (4): 700–10.Gringeri A., Lundin B., Von Mackensen S., Mantovani L., Mannucci P., Group E.S. A randomized clinical trial of prophylaxis in children with hemophilia A (the ESPRIT Study). J Thromb Haemost 2011; 9 (4): 700–10.17. Morfini M., Haya S., Tagariello G., Pollmann H., Quintana M., Siegmund B., et al. European study on orthopaedic status of haemophilia patients with inhibitors. Haemophilia 2007; 13 (5): 606–12.Morfini M., Haya S., Tagariello G., Pollmann H., Quintana M., Siegmund B., et al. European study on orthopaedic status of haemophilia patients with inhibitors. Haemophilia 2007; 13 (5): 606–12.18. Rocino A., Franchini M., Coppola A. Treatment and prevention of bleeds in haemophilia patients with inhibitors to factor VIII/IX. J Clin Med 2017; 6 (4): 46.Rocino A., Franchini M., Coppola A. Treatment and prevention of bleeds in haemophilia patients with inhibitors to factor VIII/IX. J Clin Med 2017; 6 (4): 46.19. Tagliaferri A., Feola G., Molinari A.C., Santoro C., Rivolta G.F., Cultrera D.B., et al. Benefits of prophylaxis versus on-demand treatment in adolescents and adults with severe haemophilia A: the POTTER study. Thromb Haemost 2015; 114 (1): 35–45.Tagliaferri A., Feola G., Molinari A.C., Santoro C., Rivolta G.F., Cultrera D.B., et al. Benefits of prophylaxis versus on-demand treatment in adolescents and adults with severe haemophilia A: the POTTER study. Thromb Haemost 2015; 114 (1): 35–45.20. Rodríguez-Merchán E.C. The role of orthopaedic surgery in haemophilia: current rationale, indications and results. EFORT Open Rev 2019; 4 (5): 165–73.Rodríguez-Merchán E.C. The role of orthopaedic surgery in haemophilia: current rationale, indications and results. EFORT Open Rev 2019; 4 (5): 165–73.21. Singh A., Mehta S., Goyal L.K., Mehta S., Sharma B.S. Low Dose Prophylaxis vis-a-vis on-Demand Treatment Strategies for Hemophilia: A Cost Effective and Disability Attenuating Approach. J Assoc Physicians India 2019; 67: 52.Singh A., Mehta S., Goyal L.K., Mehta S., Sharma B.S. Low Dose Prophylaxis vis-a-vis on-Demand Treatment Strategies for Hemophilia: A Cost Effective and Disability Attenuating Approach. J Assoc Physicians India 2019; 67: 52.22. Daliri A.A.K., Haghparast H., Mamikhani J. Cost-effectiveness of prophylaxis against on-demand treatment in boys with severe hemophilia A in Iran. Int J Technol Assess Health Care 2009; 25 (4): 584.Daliri A.A.K., Haghparast H., Mamikhani J. Cost-effectiveness of prophylaxis against on-demand treatment in boys with severe hemophilia A in Iran. Int J Technol Assess Health Care 2009; 25 (4): 584.