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Romiplostim – Management of Chronic Immune Thrombocytopenic Purpura in Adults
Future Perspectives need for rescue therapies such as immunoglobulins and corticosteroids.
In the past, for many adults who suffered continuous severe and As romiplostim is not an immunosuppressive agent, the problems
symptomatic chronic ITP, splenectomy was almost inevitable if they did associated with immunosuppressive treatment can be avoided.
not respond to initial treatment with corticosteroids. Today, with the
increasing number of alternative treatments, there is great interest in There are concerns about the potential increased long-term risk of
trying various therapies for at least 12 months before opting for malignancy or stimulation of solid tumour growth in patients treated
splenectomy, and this new way of thinking is reflected in falling rates of with growth factors. Some myeloid haematopoietic malignant cells
this procedure.
9
Platelet counts can be increased and sustained at have been found to express c-Mpl, which is a member of the cytokine
satisfactory levels by romiplostim in a high proportion of patients who receptor superfamily encoded by the proto-oncogene c-mpl and
were intolerant of other therapies or for whom other treatments have the receptor to which endogenous TPO and romiplostim bind.
29
failed. Indeed, the overall response rate of approximately 80% observed Follow-up of patients participating in the ITP studies of romiplostim
with romiplostim is higher than that seen with any other agent used to has shown no evidence of stimulation of tumour growth. However,
treat chronic refractory ITP. patient safety is paramount and therefore vigilant monitoring by
clinicians and regulatory bodies will help to ensure patient wellbeing
What does this all mean for the patient? Patients with chronic ITP and the rapid identification and corrective treatment of any possible
complain of fatigue, embarrassment about their appearance due to side effects that appear with time.
bruising and decreased ability to carry out their routine daily activities.
25
A recent publication highlighted the impaired health-related quality of In conclusion, romiplostim provides a novel option for the treatment of
life (HRQoL) experienced by patients with ITP.
26
Using the Short-Form 36 adults with chronic ITP and could change the way in which patients are
questionnaire, McMillan et al found that the HRQoL of 73 adults with ITP treated in the future. Looking ahead, romiplostim is currently also
was significantly worse than that of patients with hypertension, arthritis being evaluated for the treatment of other conditions in which
or cancer. Data from studies using the specific ITP Patient Assessment suboptimal platelet production contributes to thrombocytopenia,
Questionnaire (ITP-PAQ) to examine HRQoL changes in patients including myelodysplastic syndromes and certain chemotherapy-
participating in the two phase III studies
27
and the open-label romiplostim induced thrombocytopenias. ■
extension study
28
indicated that treatment with romiplostim significantly
improves HRQoL in this patient population. Acknowledgements
This article was supported by Amgen Europe GmbH, Zurich, Switzerland.
Romiplostim appears to be generally well tolerated during prolonged Professor Newland is a consultant and speaker for Amgen and a speaker
treatment periods of up to three years, with few patients discontinuing for GlaxoSmithKline. He has received research support from Amgen,
treatment for adverse events. Most patients can reduce or discontinue Baxter, Genetech and GlaxoSmithKline. The author thanks Julia Balfour,
the use of concomitant immunosuppressive treatments, including Medical Writer, Kilconquhar, Scotland and Claire Foster, Amgen Europe
corticosteroids, azathioprine and danazol, as well as decreasing their GmbH, for their assistance with the preparation of the manuscript.
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