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Immune Thrombocytopenia


Figure 1: Platelet Responses of Patients Treated with Either Romiplostim or Placebo in the Randomised Phase III Study


A. Splenectomised 200


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Number available for measurement


Placebo Romiplostim


21 21 21 21 21 21 21 21 21 21 11 12 20 20 20 20 20 20 18 19 18 18 19 17 42 42 42 42 42 42 41 42 41 41 40 39 41 39 40 40 39 40 39 39 40 38 38 39


B. Non-splenectomised 200


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Number available for measurement


Placebo Romiplostim


21 21 21 21 21 21 21 20 18 19 19 19 18 18 18 18 18 18 18 18 18 18 17 16 41 41 41 41 41 41


Romiplostim Figures 1A and B reprinted with permission from Elsevier, The Lancet, 2008;371:395–403. Placebo


Median platelet count at every weekly study visit for splenectomised (A) and non-splenectomised (B) patients. Data include all patients, even those who received rescue drugs. Error bars indicate the range from the first to third quartiles. Dashed line indicates platelet count of 50x109/l. Source: Kuter et al., 2008.38


At the end of the 52-week study period, participants receiving romiplostim achieved and maintained desirable platelet levels at 2.3 times the rate of those on standard care.40


Romiplostim


treatment was at least three times as effective as other treatments and participants receiving the drug had a 90% reduction in the need for splenectomy. Serious adverse events occurred in 23% of patients receiving romiplostim and 37% of patients receiving standard care.40


Long-term Extension Study


Patients were eligible for the open-label, multicentre extension study if they had completed a prior study using romiplostim for the treatment of chronic ITP (regardless of whether they had received romiplostim or placebo) and had a platelet count ≤50x109/l.41


who had previously received romiplostim were initially given the same dosage of the drug as they received in the prior study. They were given 1µg/kg/week if >24 weeks had elapsed since their last dose or they had previously received placebo. The romiplostim dosage was


66


adjusted throughout the study, depending on platelet response; the target platelet count range was 50–250x109/l. Patients could continue to receive stable dosages of concurrent ITP medications (corticosteroids, azathioprine or danazol). The three-year update analysis included 143 enrolees, with a median age of 53 years, 60% splenectomised and 22% on concurrent ITP therapy at baseline. Of these patients, 142 were treated with romiplostim (mean duration 69 weeks).41


40 41 41 40 40 37 40 38 40 38 39 39 38 39 38 36 38 39


17 39


2 3 4 5 6 7 8 9 101112131415161718192021222324 Study week


25


19 40


11 12 13 14 15 16 17 18 19 20 21 22 23 24 Study week 25


Those


Platelet responses (platelet count >50x109/l and double baseline) were observed in 87% of all patients and occurred on average 67% of the time in responding patients. The mean (±SD) of the average weekly dose was 5.9±3.9µg/kg. In 77% of patients, the romiplostim dose remained within 2µg/kg of their most frequent dose at least 90% of the time. Ninety patients (63%) received treatment by self-administration.


Safety


In the phase I–II ITP studies, the most frequently reported event was mild to moderate headache.42


EUROPEAN ONCOLOGY & HAEMATOLOGY In the phase III trials, serious


Median platelet count (x109


/l)


Median platelet count (x109


/l)


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