NATIONAL SURVEY OF PATIENTS WITH HEMOPHILIA AND OTHER CONGENITAL BLEEDING DISORDERS IN THAILAND

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1 NATIONAL SURVEY OF PATIENTS WITH HEMOPHILIA AND OTHER CONGENITAL BLEEDING DISORDERS IN THAILAND Ampiwn Chunsumrit 1, Chulrtn Mhsndn 2, Yingyong Chinthmmitr 2, Boonchu Pongtnkul 2, Vichi Losombt 3, Weersk Nwrwong 4, Yuchind Lektkul 5, Somporn Wngrungstid 6, Ldd Sriboriboonsin 7, Ponlpt Rojnkrin 8, Pntep Angchisuksiri 1 nd Hemophili Study Group* 1 Fculty of Medicine t Rmthibodi Hospitl, 2 Fculty of Medicine t Sirirj Hospitl, Mhidol University, Bngkok; 3 Fculty of Medicine, Prince of Songkl University, Songkhl; 4 Fculty of Medicine, Ching Mi University, Ching Mi; 5 Supprsithiprsong Hospitl, Ubon Rtchthni; 6 Buddhchinrj Hospitl, Phisnulok; 7 Lmpng Hospitl, Lmpng, 8 Fculty of Medicine, Chullongkorn University, Bngkok, Thilnd Abstrct. A ntionl survey of ptients with hemophili nd other congenitl bleeding disorders in Thilnd ws conducted in the yers 2000 to Questionnires were sent to physicins working t hospitls throughout the country. Although the overll response rte to the questionnires ws 19%, the two highest rtes of 80% nd 73.7% were found t university nd regionl hospitls, respectively, where most of the ptients received their dignosis nd tretment. A totl of 1,450 ptients comprised of hemophili 1,325 cses, von Willebrnd disese, 69 cses, congenitl fctor VII deficiency, 15 cses, hereditry pltelet dysfunction, 22 cses, nd undefined cuses of congenitl bleeding disorders, 19 cses. Most were peditric ptients <15 yers of ge. Tretment ws minly given on demnd for bleeding episode, while only 8.6% received dditionl home tretment for erly bleeding episodes. Replcement therpy primrily relied on fresh frozen plsm, cryoprecipitte nd cryo-removed plsm. Fctor concentrte ws seldom used becuse of the high price. As result, hemophili cre services in Thilnd should be strengthened by providing comprehensive eduction for medicl personnel, mking vilble simple lbortory kits to determine hemophili A nd B, ensuring n dequte supply of blood components nd ffordble fctor concentrte, nd estblishing home cre tretment. Correspondence: Prof Ampiwn Chunsumrit, Deprtment of Peditrics, Fculty of Medicine, Rmthibodi Hospitl, Mhidol University, Rm VI Rod, Bngkok 10400, Thilnd. Tel: 66 (0) ; Fx: 66 (0) , ; E-mil: rjs@mhidol.c.th *Hemophili Study Group: Theer Ruchutrkool, Fculty of Medicine t Sirirj Hospitl, Mhidol University, Bngkok; Mli Wongchnchilert, Fculty of Medicine t Songklngrind Hospitl, Prince Songkl University, Songkhl; Pttr Thnrttnkorn, Fculty of Medicine t Ching Mi Hospitl, Ching Mi University, Ching Mi; Issrng Nuchpryoon, Fculty of Medicine, Chullongkorn Hospitl, Chullongkorn University, Bngkok; Pny Seksrn, Fculty of Medicine, Chullongkorn Hospitl, Chullongkorn University, Bngkok; Arunee Jetsrisuprb, Fculty of Medicine t Khon Ken Hospitl, Khon Ken Uiversity, Khon Ken; Chittim Sirijirchi, Fculty of Medicine t Khon Ken Hospitl, Khon Ken University, Khon Ken; Triroj Krutvecho, Phrmongkutklo College of Medicine, Bngkok; Roongroj Pimchipong, Udon Thni Hospitl, Udon Thni; Ptchrt Kittiwttnwn, Buri Rm Hospitl, Buri Rm; Yuthsk Osodthnkrn, Uttrdit Hospitl, Uttrdit; Apicht Apiwttnporn, Udon Thni Hospitl, Udon Thni; Nitty Visnuyothin, Mhrt Nkhon Rtchsim Hospitl, Nkhon Rtchsim; Piypn Ruthirgo, Trng Hospitl, Trng; Kulthid Swtdee, Nkhon Nyok Hospitl, Nkhon Nyok; Songcht Siriyothinphn Prchup Khiri Khn Hospitl, Prchup Khiri Khn; Pempk Sornchi, Nkhonping Hospitl, Ching Mi; Sroj Suntykorn, Buddhchinrj Hospitl, Phisnulok; Sombt Nvrttr, Prpokklo Hospitl, Chntburi; Suebsuk Sirithorn, Bhumipol Adulydej Hospitl, Bngkok; Sutin Krongpirdee, Prnkornsiyutthy Hospitl, Ayutthy; Suwpee Burnwnich, Pholpolyuhsen Hospitl, Knchnburi; Wnpen Stworrwong, Chon Buri Hospitl, Chon Buri. Vol 35 No. 2 June

2 SOUTHEAST ASIAN J TROP MED PUBLIC HEALTH INTRODUCTION The prevlence of ptients with congenitl bleeding disorders vries from 1 to 500,000 popultion, found in congenitl fctor VII deficiency (Mrini nd Mzzucconi, 1983), to up to 1 to 1,000 popultion found in von Willebrnd disese (Werner et l, 1993). However, severe bleeding disorders re commonly found in hemophilics with prevlence of 1 to 13,000 in the Thi popultion (Isrngkur et l, 1980), similr to western countries. It is estimted tht t lest 4,500 people with hemophili live in Thilnd. Hemophili cre services hve been integrted into the existing infrstructure of the medicl helth cre system (Isrngkur nd Chunsumrit, 1991). They cn be clssified into four levels, operting with primry hemophili cre centers: 8,842 helth sttions nd 695 district hospitls, hemophili tretment centers in 73 provincil hospitls, comprehensive cre centers in 19 regionl hospitls, nd reference centers for hemosttic disorders in 10 university hospitls. The mjority of ptients often see hemtologists t university hospitls for definite dignosis nd tretment. However, hemophili cre fcilities re not exclusively for ptients with hemophili, but re utilized by ptients with other congenitl nd cquired bleeding disorders. The present study reports ntionl survey of ptients with hemophili nd other congenitl bleeding disorders in Thilnd, from 2000 to MATERIALS AND METHODS Questionnires concerning gender, ge, bleeding mnifesttions, dignosis nd tretment were collected from physicins, peditricins nd internists in the district, provincil, regionl nd university hospitls throughout Thilnd in the yers 2000 to RESULTS The response rtes for the questionnires vried from 14.3% to 80%, with n verge of 19%, s shown in Tble 1. The two highest response rtes, of 80% nd 73.7%, were from university nd regionl hospitls, respectively. A totl of 1,450 ptients, comprised of hemophili, 1,325 cses, von Willebrnd disese, 69 cses, congenitl fctor VII deficiency, 15 cses, hereditry pltelet dysfunction such s Glnzmnn thrombstheni, 22 cses, nd undefined cuses of congenitl bleeding disorders, 19 cses. Mles were predominnt mong ptients with hemophili, but both mles nd femles were found in ptients with other congenitl bleeding disorders. Most (80%) were in the peditric ge group of <15 yers old. Although hemophili ws the prominent dignosis, hemophili A nd B were determined in 70% of ptients. The mjority of ptients with hemophili were treted t university nd regionl hospitls, s shown in Tble 2. The lbortory investigtion for determining the sttus of hemophili nd other congenitl bleeding disorders t different tretment centers is shown in Tble 3. Complete fcilities re vilble t university hospitls. The fctor ssy for determining the sttus of hemophili A nd B is performed t the university hospitl nd some Tble 1 Questionnires response rtes. Type of hospitl Totl Responses % University Regionl Provincil District Others Totl Privte hospitls, government sector hospitls Tble 2 Number of ptients with hemophili being treted by physicins in different hospitls. Type of hospitl Number of ptients % University 1, Regionl Provincil District Others Totl 1, Privte hospitls, government sector hospitls 446 Vol 35 No. 2 June 2004

3 regionl hospitls. Detiled informtion on bleeding mnifesttions nd replcement therpy ws obtined from 465 ptients receiving tretment t Rmthibodi Hospitl (n=252), regionl hospitls (n=106), provincil hospitls (n=79), nd district hospitls (n=28), s shown in Tbles 4 nd 5. However, bleeding in the centrl nervous system ws minly treted t university nd regionl hospitls, while less-severe bleeding episodes were treted t provincil nd district hospitls. Replcement therpy minly relies on fresh frozen plsm nd cryoprecipitte. Very minute mounts of commercil fctor concentrte were used. Of note, desmopressin ws used in university hospitls only. In ddition, ptients with inhibitor exhibiting bleeding, were predominntly treted t university hospitls. None of the ptients t regionl, provincil or district hospitls received home cre tretment for erly bleeding, except for 8.6% of ptients from Rmthibodi, Songklngrind nd Chullongkorn hospitls. However, these ptients were lso treted by physicins nd medicl personnel t provincil nd district hospitls, nd helth sttions. DISCUSSION Although the overll response rte to the questionnires ws 19%, the two highest rtes of 80% nd 73.7% were from university nd regionl hospitls, respectively, where most of the hemophilics received dignosis nd tretment. Only 29.4% (1,325/4,500) of the estimted hemophilic Tble 3 Lbortory investigtion for ptients with hemophili nd other congenitl bleeding disorders t different tretment centers. Hospitl Venous clotting time Cogulogrm Fctor ssy Pltelet function test University Regionl + + ± + Provincil District + ± - - Government sector + + ± - Privte = vilble, - not vilble, ± my be vilble in some hospitls Tble 4 Common bleeding episodes mong 465 ptients with hemophili treted t four different tretment centers. Bleeding mnifesttion University Regionl Provincil District Totl n = 252 n = 106 n = 79 n = 28 n = 465 Hemrthrosis Hemtom Gum nd teeth bleeding Ecchymosis Epistxis CNS bleeding Others Totl Rmthibodi Hospitl Vol 35 No. 2 June

4 SOUTHEAST ASIAN J TROP MED PUBLIC HEALTH Tble 5 Replcement therpy for ptients with hemophili in Thilnd. Type Het-treted dried Frozen Cryo-removed Fresh frozen Commercil cryoprecipitte cryoprecipitte plsm plsm fctor concentrte Hemophili A Occsionl Hemophili B Occsionl Unclssified = yes, - = no ptients in Thilnd received dignosis nd tretment. Most were peditric ptients, <15 yers of ge. Unfortuntely, 70.6% of estimted hemophili ptients were still undignosed becuse of limited helth cre resources in rurl res. They lived with intense pin, disbility, nd debility. They fought lonely bttle resulting in loss of their limbs or their lives. The ptients who received little or no tretment often succumbed to bleeding episodes in their erly life nd seldom reched dulthood. In fct, in previous study, 103 out of 139 mles (74.1%) with presumptive dignosis of hemophili by history-tking, died from bleeding episodes t men ge of 8 yers, 9 months (Krsesub nd Chunsumrit, 2001). It is cler tht comprehensive eduction relted to hereditry bleeding disorders should be provided to ll helth personnel, identify more ptients with congenitl bleeding disorders. However, the determintion of hemophili A nd B ws not consistently crried out t these hospitls. In the present study, 30% of ptients received presumptive dignosis of hemophili without determining the sttus of hemophili A nd B, since the ssy of fctor VIII nd IX clotting ctivities ws not routinely performed. The min constrint in performing the ssy is tht the substrte of commercil fctor VIII or IX deficient plsm is expensive. Of note, fctor VIII or IX deficient plsm obtined from ptients with severe hemophili is recommended s the inhouse prepred substrte for the fctor ssy. The shelf life is 3 months if kept t -70ºC, nd decreses to 1 month if kept t -20ºC. In the future, n in-house lbortory kit for distinguishing hemophili A nd B should be creted nd distributed, especilly to hospitls in rurl res. The clinicl mnifesttions of bleeding episodes of hemophilics treted t the regionl, provincil nd district hospitls did not differ from the previous study of university hospitls (Chunsumrit et l, 1993). They minly received fresh frozen plsm nd cryoprecipitte on demnd for bleeding episodes. Fctor concentrte ws seldom used becuse of the high price. Indequte replcement therpy is the min constrint in hemophili cre services. Moreover, fresh frozen plsm nd cryo-removed plsm re both insufficient to rise the desirble levels of fctor VIII or IX clotting ctivity, especilly in ptients with body weight > 30 kg. Additionlly, volume overloded, pulmonry edem nd renl impirment re found in ptients receiving lrge mounts of plsm trnsfusion (Morn nd Kpsner, 1987). Therefore, n dequte supply of blood component nd ffordble fctor concentrte re essentil for mintining hemophili cre. Home-cre tretment ws only provided to ptients of Rmthibodi, Songklngrind nd Chullongkorn hospitls. In fct, home-cre tretment for erly bleeding episodes is essentil, especilly in developing countries with limited helth cre resources. Low litercy is no brrier to the estblishment of home cre tretment (Chunsumrit et l, 1999). Bleeding, if promptly treted t home, is less extensive compred with delyed tretment, nd fewer blood components re utilized thn with delyed tretment (Dietrich, 1996). In conclusion, the hemophili cre services in Thilnd should be extended to the mjority of ptients in rurl res by strengthening four spects: comprehensive eduction relted to heredi- 448 Vol 35 No. 2 June 2004

5 try bleeding disorders should be provided to medicl personnel; the vilbility of simple lbortory testing kits for determining hemophili A nd B should be distributed ntionwide; n dequte supply of blood components nd ffordble fctor concentrtes should be mintined; nd home-cre tretment for erly bleeding episodes should be estblished nd promoted. As result, medicl outrech services will grdully bring bout better outcomes for ptients with hemophili ll over Thilnd. Hopefully, they will be ble to live out their lives s helthy dults nd continue to contribute to our society. ACKNOWLEDGEMENTS The uthors would like to express their sincere thnks to the physicins nd medicl personnel who provided dt on ptients with hemophili nd other congenitl bleeding disorders in their hospitls. REFERENCES Chunsumrit A, Isrngkur P, Hthirt P, Chiewsilp P, Kittikool J. Cre of Thi hemophili ptients from 1969 to J Med Assoc Thi 1993; 76 (suppl 2): Chunsumrit A, Isrngkur P, Chntnkjornfung A, Pnthngkool W, Hthirt P. Home tretment for ptients with congenitl bleeding disorders in developing country. J Med Assoc Thi 1999; 82 (suppl 1): Dietrich SL. The tretment of hemophili bleeding; problems with limited or no use of replcement therpy. World Federtion of Hemophili, Monogrph, Mrch 1996: 1-4. Isrngkur P, Bintdit P, Hthirt P, Ssnkul W, Wtn-Ksetr S, Thnmitr S. Study of the prevlence of hereditry bleeding disorders in Thilnd. Vjir Med J 1980; 24: Isrngkur P, Chunsumrit A. Developing nd mintining hemophili progrm in Thilnd. In: Lusher JM, Kessler CM, eds. Hemophili nd von Willebrnd s disese in the 1990s. New York: Elsevier Science, 1991: Krsesub S, Chunsumrit A. Survivl nlysis of ptients with hemophili in tretment centre in Thilnd. Thromb Hemost 2001; 87 (suppl): Mrini G, Mzzucconi MG. Fctor VII congenitl deficiency. Clinicl picture nd clssifiction of the vrints. Hemostsis 1983; 13: Morn M, Kpsner C. Acute renl filure ssocited with elevted plsm oncotic pressure. N Engl J Med 1987; 317: Werner EJ, Broxson EH, Tucker EL, Giroux DS, Shults J, Abshire TC. Prevlence of von Willebrnd disese: multiethnic study. J Peditr 1993; 123: Vol 35 No. 2 June