MONTREAL – Clinicians who manage the care of children with newly diagnosed idiopathic thrombocytopenic purpura (ITP) have to decide whether to treat patients early and possibly increase risk for chronic ITP down the road or to leave well enough alone and treat only as needed.
Evidence from a retrospective study suggests that, for most patients, early treatment of acute ITP does not appear to increase the risk for chronic ITP in the future, reported Chelsea L. Grama, a fourth-year medical student, and her colleagues from Penn State Health Children’s Hospital in Hershey, Pennsylvania.
“It’s controversial whether to treat or not. Some people think steroids are the best way to go, some think IVIG [intravenous immunoglobulin] is the best way to go, and some think that no treatment could be the best option. There is really no consensus in the literature to say what the ideal treatment for ITP is,” she commented in an interview at the annual meeting of the American Society of Pediatric Hematology/Oncology.
That lack of consensus is the result of the fact that serious consequences of ITP, such as intracranial hemorrhage, are rare, making it difficult for investigators to compare clinical outcomes with various forms of treatment. It’s also unclear whether early interventions could lead to later chronic disease.
In hopes of answering this question, Ms. Grama and her coinvestigators, led by Andrew S. Freiberg, MD, took a retrospective look at data on 249 patients with ITP diagnosed from birth through age 21 from 1994 to 2011.
They looked at demographic variables, treatments received, and subsequent platelet counts. Outcomes included intracranial hemorrhage and long-term improvements in platelet counts. They defined chronic ITP as a platelet count below 140,000/mcL for at least 6 months following diagnosis.
Of the 249 patients, 126 (66 boys and 60 girls) progressed to chronic ITP. The incidence of chronic ITP among treated patients was 51.1% and, in untreated patients, 49.3%.
However, in two subgroups, there was a significant association between treatment and chronic ITP. Among girls from birth through age 3 years, 58% of those treated went on to chronic ITP, compared with 0% for untreated patients (P = .008). When the age cohort was expanded to girls younger than 6 years, a similar pattern emerged, with chronic ITP rates of 45% for treated patients, compared with 0% for untreated patients. There were only 44 total patients in this age and sex subgroup, however, making it difficult to draw strong inferences about a potential relationship between treatment and chronic ITP, Ms. Grama noted.
Only three patients had cerebral hemorrhage. Two had hemorrhage as their initial ITP presentation, and the third had bleeding despite being on treatment. The sample size was too small to correlate the outcome with treatment, the authors noted.
In multivariate analysis controlling for demographic, clinical, and treatment factors, only age and platelet count at presentation independently predicted chronic thrombocytopenia (P less than .0001 for each). Steroid therapy was the only independent predictor for acute ITP (P = .0001).
Although their data suggest that there could be a benefit to treating patients who first present with acute ITP at age 12 years or older, “I do think it’s reasonable to wait and watch these patients,” Ms. Grama said.
“The incidence of brain bleeds, which is the really scary complication, was so low in these patients that I think watchful waiting would be a better way to go, just monitoring patients very closely. Hopefully, they will resolve without having treatment,” she added.
The study was internally supported. The authors reported no relevant disclosures.