Hemophilia B disease Hemophilia A and B are X-linked coagulation defects that lead to deficiencies of factor VIII (FVIII) and factor IX (FIX) in approximately 1 of 5000 and 1 of 30,000 male live births, respectively, the disease phenotype is characterized by recurrent spontaneous or traumatic bleeding episodes predominantly involving the weight-bearing joints, skeletal muscle, and soft tissues. Intracranial and retroperitoneal hematomas are rare but life-threatening complications of severe hemophilia. The bleeding phenotype has been defined as “severe”, “moderate”, and “mild” based on the level of the residual endogenous factor being <1 IU/dL, 1-5 IU/dL, and 5-40 IU/dL, respectively<br />Aims of treatment: -<br />Replacement of the missing factor constitutes the mainstay of hemophilia treatment. <br />Factor replacement is given either <br /> 1-on demand to treat acute bleeding or <br /> 2-prophylactically to prevent bleeding .<br /><br />In severe hemophilia, recurrent bleeding, typically in the form of joint bleeds and skeletal muscle hematomas, results in progressive hemophilic arthropathy and muscle contractures, which eventually lead to irreversible joint damage, significant disability, and decreased quality of life unless treated with FVIII and FIX Regular prophylactic factor replacement to maintain circulating factor levels of >1 IU/dL (1%) has been recommended as the optimal therapy for people with severe hemophilia. <br />Management of hemophilia mainly depends on replacing the missing coagulation factor to stop (episodic or on-demand therapy) in mild and moderate form or prevent bleeding episodes by prophylaxis therapy in sever form. In the prophylactic setting, people with severe hemophilia B is usually treated by SHL (short or standard half-life) twice weekly, owing to the longer half-life of FIX (18-20 h ) in comparison to FVIII 3time/wk Bleeds starting from the first sign of bleed and ending 72 hours after last treatment for the bleed, within which any symptoms of bleeding at the same location or injections ≤72 hours apart, are considered to be the same bleed .<br />Bleeds starting from the first sign of bleed and ending 72 hour Prophylaxis with factor replacement therapy is the gold standard for the treatment of hemophilia.<br /><br />Prophylaxis with factor replacement therapies is the accepted strategy to: -<br />1- reduce bleeding risk <br />2- improve long-term outcomes. Leading improved quality of life However, regimens with standard half-life products (SHL): -- <br />1-require frequent infusions to achieve factor activity levels sufficient to reduce the risk of bleeding. <br /> 2- patients can still experience breakthrough bleeds even when treated with such intense regimens after last treatment for the bleed, <br />In the last decade, many long-acting recombinant FIX products have been developed that aim to address these; <br />1- unmet needs<br />2- reduce the burden on patients symptoms of bleeding at the same location or injections ≤72 hours apart, are considered to be the same bleed<br />The aims of extended half-life (EHL):-<br />1- promise optimal prophylaxis by decreasing the dose frequency and, increasing the compliance, <br /> 2- improving the quality of life without compromising safety and efficacy. <br />3- even EHL products might lead to higher trough levels without increasing infusion frequency, or could facilitate the ability to maintain trough levels while reducing Indications and Utility of EHL:<br /> Switching from SHL Factors to EHL Factors <br />1-improved Pharmacokinetics profiles with prolonged half-lives ranging from 1.2 to 1.5-fold for FVIII and 3 to 5-fold for FIX.<br /> 2- EHL products were shown to be well tolerated with no inhibitor development in the PTP population.<br />3-Extension of half-life might lead to higher trough levels without increasing infusion frequency or may be reduced infusion frequency.<br /> 4-it is associated with a better health-related quality of life (HRQOL) as compared to episodic treatment.<br /> 5-Furthermore, it leads to a decrease in bleeding-related hospitalization, shortens length of hospital stay, and thus reduces resource utilization <br />This mean that: <br />1- longer half-lives and reduced clearance of EHL factors are suggested to result in reduced factor consumption 2-maintaining higher trough levels and protection from bleeding.<br />Alprolix; (coagulation factor IX recombinant, fc fusion protein) is recombinant clotting factor therapy developed for hemophilia B It is using FC fusion technology to prolong circulation in the body.<br />It is engineered by fusing factor IX to FC portion of IgG subclass 1 or called IgG1 (it is protein commonly found in the body) enable Alprolix to use naturally occurring pathway to extend the time the therapy remains in the body (half-life reached to 82 hrs. compared to standard Benefix therapy of 18 hrs. and they show normal procoagulation activity, thereby decreasing the frequency of infusion, while fusion technology has been used for more than 15 years.<br />Alprolix is manufactured using human cell line in an environment free of animal and human additives. It is FDA and EMA approval. <br /><br />DOSAGE AND ADMINISTRATION: -<br />For intravenous use after reconstitution only. <br />• Each vial of ALPROLIX contains the labeled amount of coagulation Factor IX potency in international units (IU).<br />• On average, one unit per kilogram body weight of ALPROLIX increased the circulating Factor IX level by approximately 1% (IU/dL) in adults and children ≥6 years of age and by 0.6% (IU/dL) in children under 6 years of age. <br />ALPROLIX is available as a sterile, non-pyrogenic, preservative-free, white to off-white, lyophilized powder in single-dose vials containing nominally 250, 500, 1000, 2000<br />Al-Mustaqbal University is the first in Iraq.<br /><br /><br /><br /><br /><br /><br /><br /><br /><br />