Publication Registry II
Severe hemorrhage in children with newly diagnosed immune thrombocytopenic purpura. Blood 2008;112:4003-4008
Neunert CE, Buchanan GR, Imbach P, Bolton-Maggs PHB, Bennett CM, Neufeld EJ, Vesely SK, Adix L, Blanchette VS, Kühne T.
Frequency, Location, and Timing of Severe Hemorrhage in Children with Newly-diagnosed Idiopathic Thrombocytopenic Purpura: A Study of the Intercontinental Cooperative ITP Study Group. Fortyfifth Annual Meeting of the American Society of Hematology, San Diego, California, USA December 6-9, 2003. Blood 2003;102;298a
G. R. Buchanan, T. Kühne, P. Bolton-Maggs, S. K. Vesely, L. Adix, V. S. Blanchette, P. Imbach
There is an emerging consensus that bleeding severity is an important outcome measure in classification and clinical management of childhood idiopathic thrombocytopenic purpura (ITP). It is desirable to characterize how often severe hemorrhage occurs in ITP, both at diagnosis and during the ensuing weeks, but few data are available in the literature. Therefore, ICIS, a multi-institutional consortium of pediatric centers conducting research in childhood ITP (Lancet 2001;358:2122-5), developed a prospective registry (called Registry II) aimed at characterizing hemorrhage using a novel grading classification system (Lancet 1997;350:620-3) in which severe hemorrhage was defined as “bleeding episodes’ requiring hospitalisation and/or blood transfusions - i.e., symptoms interfering seriously with quality of life.” Participating investigators were asked to submit data regarding all new ITP patients in their centers, emphasizing initial bleeding events during the subsequent 28 days. Between July 2001 and April 2003, 531 children (median age 5.0 years) with ITP were enrolled on registry II by 62 investigators in 41 countries. Of 407 patients with an initial platelet count = 20’000 per mm3, bleeding severity assessments were conducted as part of an interim analysis in 335 evaluable subjects. At presentation hemorrhage was inapparent or mild in 76% of cases, moderate in 21%, and severe in 3% (11/335, 95% CI 1.7 to 5.5%). One patient had intracranial hemorrhage (ICH) at diagnosis. Of the 255 evaluable patients with no or mild hemorrhage at diagnosis, only 3 (1.2%, 95 CI 0.3 to 3.7%) developed severe hemorrhage during the next 28 days. Two of these 3 patients were among the 64 who did not receive initial drug therapy. The other child was 1 of 73 receiving only IVIG initially. None of the 62 children receiving only steroids or the 26 receiving anti-D had a new episode of severe hemorrhage by day 28. All 3 children with severe hemorrhage after diagnosis had gastrointestinal bleeding (as well as epistaxis in 2 cases and menorrhagia in one case). Two received platelet transfusions and one RBC transfusions. No patient had ICH after diagnosis. No deaths were reported among the 531 patients. This study confirms the 3% rate of severe hemorrhage in newly-diagnosed children with ITP reported by Bolton-Maggs and Moon. It also provides, for the first time, data regarding frequency and sites of severe hemorrhage developing shortly following diagnosis and the initial management decision in children whose bleeding on presentation was mild. Such hemorrhagic events are infrequent (1.3% of cases), even when no platelet-enhancing therapy was given at diagnosis. The low frequency of severe hemorrhage during the initial month following diagnosis renders difficult the conduct of clinical trials aimed at preventing such events.