A French study in this week’s issue of THE LANCET provides evidence of a new treatment strategy for the potentially fatal blood disorder autoimmune thrombocytopenic purpura (AITP).
AITP is an immune-system disorder that affects blood clotting; a low platelet count causes internal bleeding resulting in the appearance of purple patches under the skin. Severe bleeding can be fatal. Treatment of adults with AITP is controversial, and is often based more on clinicians’ individual experiences than on results of controlled studies. Philippe Bierling and colleagues from Hopital Henri Mondor, Paris, France, compared intravenous immunoglobulin with high-dose methylprednisolone in untreated adults with severe AITP and assessed efficacy of subsequent oral steroids compared with placebo.
122 adults with severe AITP (platelet count less than 20 x 109 per litre) were randomly assigned to receive either intravenous immunoglobulin or high-dose methylprednisolone on days 1-3 (randomisation A), and then to receive either oral prednisone or placebo (randomisation B) on days 4-21.
The number of days on which platelet counts were above 50 x 109 per litre and above 20 x 109 per litre was 18 and 19, respectively, among 56 patients receiving intravenous immunoglobulin and 14 and 17, respectively, among 60 receiving high-dose methylprednisolone, respectively. The percentage of patients who had platelet counts over 50 x 109 per litre on days 2 and 5 was 7% and 79%, respectively, in the intravenous immunoglobulin group compared with 2% and 60%, respectively, in the high-dose methylprednisolone group. Between days 1 and 21, patients who received intravenous immunoglobulin and prednisone had 18.5 and 19 days with platelet count greater than 50 x 109 per litre and 20 x 109 per litre, respectively (p=0.005 and p=0.008, respectively), and those treated with high-dose methylprednisolone and prednisone had 17.5 and 19 days, respectively.
In an accompanying Commentary (p 4), Adrian Newland from Barts and the London School of Medicine and Dentistry, UK, concludes: “To demonstrate clinical benefit what is still required are trials in which assessment is based on clinical endpoints. The difficulty is that such studies will require large numbers of patients. Meanwhile, for all other patients without severe life-threatening bleeding, there should be an initial period of judicious observation, since treatment may often be unnecessary and may increase the risk of infection. Without treatment most such patients will have a normal or near-normal quality of life. There is the prospect of specific immune-based therapies, but these new treatments are also likely to be associated with an increased incidence of adverse effects, particularly infection. Hence treatment for AITP should always be restricted to those with clinical need.”
Contact: Dr Philippe Bierling, Centre de Transfusion Sanguine, Hopital Henri Mondor, 51 Avenue du Marechal de lattre de Tassigny, 94000 Creteil Cedex, France; T) +33 1 45 12 76 76; F) +33 1 48 98 10 46; E) philippe.bierling@efs.sante.fr
Dr Adrian Newland, Department of Haematology, Barts and the London School of Medicine and Dentistry, London E1 1BB, UK; E) a.c.newland@mds.qmw.ac.uk
Journal
The Lancet