Clinical and ultrasound joint outcomes in severe hemophilia A children receiving episodic treatment in Indonesian National Hemophilia Treatment Center
DOI:
https://doi.org/10.13181/mji.v26i1.1494Keywords:
arthropathy, HEAD-US, hemophilia, HJHS, inhibitor factor VIIIAbstract
Background: Recurrent joint bleeds leading to arthropathy is the main problem in severe hemophilia children. This study aimed to investigate joint status in severe hemophilia A children receiving episodic treatment in Cipto Mangunkusumo Hospital, Jakarta.
Methods: A cross-sectional study was conducted in Cipto Mangunkusumo Hospital as Indonesian National Hemophilia Treatment Center on children (4–18 years) with severe hemophilia A, who previously received episodic treatment, with no history of inhibitor factor VIII. Hemophilia Joint Health Score was evaluated according to HJHS version 2.1 2011. Joint ultrasonography was done for six index joints (bilateral elbows, knees and ankles) using Haemophilia Early Arthropathy Detection with Ultrasound (HEAD-US) methods. Data of age of first joint bleed, number of target joints and inhibitor factor VIII were obtained from the Pediatric Hemophilia Registry and medical records.
Results: There were 59 subjects aged 4 to 18 years. Twentynine out of 59 (49.2%) subjects experienced first joint bleed before of 2 years of age. The most common of joint bleeds was a right ankle. Mean total HJHS was 8.71±8.73. Subjects aged 4–10 years showed lower HJHS (4.6±3.7) as compared to subjects aged >10–18 years (12.3±10.3), p<0.001; 95% CI=4.9–13. Mean HEAD-US scores in subjects aged 4–10 years (18.7±5.6) was lower than in subjects aged >10–18 years (28±7.9), p<0.001, 95% CI= -12.9–-5.6.
Conclusion: HJHS and HEAD-US scores of severe hemophilia A children receiving episodic treatment aged 4–10 years are lower compared to subjects aged >10–18 years, indicating more severe joint destruction in older children and progressivity of joint damage over time. It is important to start prophylactic treatment to prevent progressivity of joint damage.
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van Dijk K, van der Bom JG, Fischer K, Grobbee DE, van den Berg HM. Variability in clinical phenotype of severe haemophilia: The role of the first joint bleed. Haemophilia. 2005;11:438–43 https://doi.org/10.1111/j.1365-2516.2005.01124.x
Srivastava A, Brewer AK, Mauser-bunschoten EP, Key NS, Kitchen S. Treatment Guidelines Working Group on Behalf of The World Federation of Hemophilia: Guidelines for the management of hemophilia. Haemophilia. 2013;19:1–47. https://doi.org/10.1111/hae.12074
Perhimpunan Hematologi dan Transfusi Darah Indonesia (PHTDI) & Himpunan Masyarakat Hemofilia Indonesia (HMHI). Panduan diagnosis dan tata laksana hemofilia. 1 ed: Jakarta: PHTDI & HMHI; 2013. hal. 1–31.
Acharya SS, Kaplan RN, Macdonald D, Fabiyi OT, DiMichele D, Lyden D. Neoangiogenesis contributes to the development of hemophilic synovitis. Blood. 2011;117:2484–93. https://doi.org/10.1182/blood-2010-05-284653
Handryastuti, Djajadiman G, Arwin AP Akib. Clinical characteristics of hemophilia A patients with hemarthrosis. Paediatr Indones. 2002;42:131–7.
Hilliard P, Funk S, Zourikian N, Bergstrom BM, Bradley CS, McLimont M, et al. Hemophilia Joint Health Score reliability study. Haemophilia. 2006;12:518–25. https://doi.org/10.1111/j.1365-2516.2006.01312.x
Feldman BM, Funk S, Lundin B, Doria AS, Ljung R, Blanchette V, et al. Musculoskeletal measurement tools from the International Prophylaxis Study Group (IPSG). Haemophilia. 2008;14:162–9. https://doi.org/10.1111/j.1365-2516.2008.01750.x
International Prophylaxis Study Group. Physical Therapy Expert Working Group. Hemophilia Joint Health Score. 2011:1–31.
Sierra Aisa C, Lucia Cuesta JF, Rubio Martinez A, Fernandez Mosteirin N, Iborra Munoz A, Abio Calvete M, et al. Comparison of ultrasound and magnetic resonance imaging for diagnosis and follow-up of joint lesions in patients with haemophilia. Haemophilia. 2014;20:51–7. https://doi.org/10.1111/hae.12268
Querol F, Rodriguez-Merchan EC. The role of ultrasonography in the diagnosis of the musculo-skeletal problems of haemophilia. Haemophilia. 2012;18:e215–26. https://doi.org/10.1111/j.1365-2516.2011.02680.x
Martinoli Carlo, Alberighi Ornella Della Casa, Di Minno Giovanni, Graziano Ermelinda, Molinari Angelo Claudio, Pasta Gianluigi, et al. Development and definition of a simplified scanning procedure and scoring method for Haemophilia Early Arthropathy Detection with Ultrasound (HEAD-US). J Thromb Haemost. 2013;109:1170–9. https://doi.org/10.1160/TH12-11-0874
Muca-Perja M, Riva S, Grochowska B, Mangiafico L, Mago D, Gringeri A. Ultrasonography of haemophilic arthropathy. Haemophilia. 2012;18:364–8. https://doi.org/10.1111/j.1365-2516.2011.02672.x
Pollmann H, Richter H, Ringkamp H, H J. When are children diagnosed as having severe haemophilia and when do they start to bleed? A 10-year single-centre PUP study. Eur J Pediatr. 1999;158:S166–70. https://doi.org/10.1007/PL00014347
Aledort LM, Haschmeyer RH, Petterssont H, & the Orthopaedic Outcome Study Group. A longitudinal study of orthopaedic outcomes for severe factor-VIII-deficient haemophiliacs. J lnt Med. 1994;236:391–9. https://doi.org/10.1111/j.1365-2796.1994.tb00815.x
Khanum F, Bowen DJ, Kerr BC, Collins PW. Joint health scores in haemophilia A cohort from Pakistan with minimal or no access to factor VIII concentrate : correlation with thrombin generation and underlying mutation. Haemophilia. 2014;20:426–34. https://doi.org/10.1111/hae.12326
Trakymiene SS, Ingerslev J, Rageliene L. Utility of the Haemophilia Joint Health Score in study of episodically treated boys with severe haemophilia A and B in Lithuania. Haemophilia. 2010;16:479–86. https://doi.org/10.1111/j.1365-2516.2009.02178.x
Trakymiene SS, Clausen N, Poulsen LH, Ingerslev J, Rageliene L. Progression of haemophilic arthropathy in children: a Lithuanian - Danish comparative study. Haemophilia. 2013;19:212–18. https://doi.org/10.1111/hae.12058
Gringeri A, Lundin B, von Mackensen S, Mantovani L, Mannucci PM, and The ESPRIT Study Group. A randomized clinical trial of prophylaxis in children with hemophilia A (the ESPRIT Study). J Thromb Haemost. 2011;9:700–10. https://doi.org/10.1111/j.1538-7836.2011.04214.x
Manco-Johnson MJ, Abshire TC, Shapiro AD, Riske B, Hacker MR, Kilcoyne R, et al. Prophylaxis versus episodic treatment to prevent joint disease in boys with severe hemophilia. N Engl J Med. 2007;357:535–44. https://doi.org/10.1056/NEJMoa067659
Verma SP, Dutta TK, Mahadevan S, Nalini P, Basu D, Biswal N, et al. A randomized study of very low-dose factor VIII prophylaxis in severe haemophilia - A success story from a resource limited country. Haemophilia. 2016;22:342–8. https://doi.org/10.1111/hae.12838
Wu R, Luke KH, Poon MC, Wu X, Zhang N, Zhao L, et al. Low dose secondary prophylaxis reduces joint bleeding in severe and moderate haemophilic children: a pilot study in China. Haemophilia. 2011;17:70–4. https://doi.org/10.1111/j.1365-2516.2010.02348.x
Altisent C, Martorell M, Crespo A, Casas L, Torrents C, Parra R. Early prophylaxis in children with severe haemophilia A : clinical and ultrasound imaging outcomes. Haemophilia. 2015;22:218–24. https://doi.org/10.1111/hae.12792
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