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Benefits of prophylaxis versus on-demand treatment in adolescents and adults with severe haemophilia A: the POTTER study

, : Benefits of prophylaxis versus on-demand treatment in adolescents and adults with severe haemophilia A: the POTTER study. Thrombosis and Haemostasis 114(1): 35-45

Rigorous evidence is lacking on long-term outcomes of factor VIII (FVIII) prophylaxis initiated in adolescent or adult patients with severe haemophilia A. The prospective, open-label Prophylaxis versus On-demand Therapy Through Economic Report (POTTER) study ( NCT01159587) compared long-term late secondary prophylaxis (recombinant FVIII-FS 20-30 IU/kg thrice weekly) with on-demand treatment in patients aged 12 to 55 years with severe haemophilia A. The annual number of joint bleeding episodes (primary endpoint), total bleeding episodes, orthopaedic and radiologic (Pettersson) scores, health-related quality of life (HRQoL), pharmacoeconomic impact, and safety were evaluated over a > 5-year period (2004-2010). Fifty-eight patients were enrolled at 11 centres in Italy; 53 (27 prophylaxis, 26 on demand) were evaluated and stratified into 2 age subgroups (12-25 and 26-55 years). Patients receiving prophylaxis experienced a significantly lower number of joint bleeding episodes vs the on-demand group (annualised bleeding rate, 1.97 vs 16.80 and 2.46 vs 16.71 in younger and older patients, respectively; p=0.0043). Results were similar for total bleeding episodes. Prophylaxis was associated with significantly fewer target joints (p< 0.001), better orthopaedic (p=0.0019) and Pettersson (p=0.0177) scores, better HRQoL, and fewer days of everyday activities lost (p< 0.0001) but required significantly higher FVIII product consumption. The POTTER study is the first prospective, controlled trial documenting long-term benefits of late secondary prophylaxis in adolescents and adults with severe haemophilia A. The benefits of reduced bleeding frequency, improved joint status, and HRQoL may offset the higher FVIII consumption and costs.


PMID: 25855376

DOI: 10.1160/TH14-05-0407

Other references

Szydlowski, M.; Gengler, N., 2008: Sampling genotype configurations in a large complex pedigree. Many genetic problems can be solved by Monte Carlo method. This often requires sampling genotype configurations over pedigree. Current available samplers are inefficient for large animal pedigrees. A new sampler suitable for large complex pedigree...

A.A., 1929: A preliminary report on the mammals of kitchen middens of stone age man on Mt Verkholensk near Irkutsk. Bones of Alce alces L., Rangifer tar-andus L., Capreolus pygargus Pall., Cervus elaphus L., Megaceros sp., Bison priscus Boj., Bos primigenius Boj.?, Ovis aff. ammon L., O. aries L.?, Capra sibirica L., Equus caballus L., E. hemionus Pall., Rhinoc...

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Furnas, H.J., 1991: Silvadene toxicity. Plastic and Reconstructive Surgery 88(4): 735-735

Yamashina, I., 2011: The trail of my studies on glycoproteins from enterokinase to tumor markers. This review describes the results of the author's studies on glycoproteins which have been carried out for more than 50 years. Starting from the elucidation of basic structures of glycoproteins, i.e. the structure of the linkage between an am...

Field, J.W., 1949: Blood examination and prognosis in acute falciparum malaria. A study of 2316 cases indicates the basic value of blood examination as the most valuable and accurate diagnostic procedure yet available. The data are treated statistically and in considerable detail.

Pines, J.M.; Prabhu, A.; Hilton, J.A.; Hollander, J.E.; Datner, E.M., 2010: The effect of emergency department crowding on length of stay and medication treatment times in discharged patients with acute asthma. This study sought to determine if emergency department (ED) crowding was associated with longer ED length of stay (LOS) and time to ordering medications (nebulizers and steroids) in patients treated and discharged with acute asthma and to study ho...

Chen, C.J.; Clark, D.; Ueda, K.; Pastan, I.; Gottesman, M.M.; Roninson, I.B., 1990: Genomic organization of the human multidrug resistance (MDR1) gene and origin of P-glycoproteins. The MDR1 gene, responsible for multidrug resistance in human cells, encodes a broad specificity efflux pump (P-glycoprotein). P-glycoprotein consists of two similar halves, each half including a hydrophobic transmembrane region and a nucleotide-bi...

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Donahoe, J.M., 1975: Experimental infection of cats with Dirofilaria immitis. Cats were inoculated s/c in the right inguinal region with 25 (group I), 100 (group II) or 200-400 (group III) infective larvae of D. immitis. Microfilariae were detected in the blood of three of six cats in group II and two of three cats in group...