Outcomes of Referrals for Mild and Moderate Thrombocytopenia

Thrombocytopenia is a common reason for referral to a Pediatric Hematologist Oncologist. However, the need for acute intervention in patients with mild or moderate thrombocytopenia is likely a rare event. Because mild and moderate thrombocytopenia is less likely to need intervention and could resolve, the potentially stressful impact of referral to an Oncology center needs consideration. But with minimal literature to support the hypothesis that mild to moderate thrombocytopenia is unlikely associated with severe or progressive hematologic diseases, our ability to provide evidence based recommendations for need for referral is limited. To better understand the course for patients with mild and moderate thrombocytopenia, we conducted a retrospective chart review to assess the prevalence, outcomes, and need for intervention among patients referred for thrombocytopenia.

Methods: We conducted a retrospective chart review of 1,140 patients referred to a large Pediatric Hematology Oncology program over a three year period (2012-2014).  The diagnosis and demographics were recorded for every patient.  Platelet counts at time of referral, lowest platelet count, and current platelet count were recorded and categorized as mild (plt: 100-149x103/mL), moderate (plt: 50-99 x103/mL), severe (plt: 20-49 x103/mL), and very severe (plt: <20 x103/mL).  Therapeutic interventions and reason for intervention were recorded.  Finally, ANA, platelet antibody and IgG levels were recorded.  Descriptive statistics and univariate analysis were conducted using JMP10.

Results: In a three year period (2012-2014), 1140 patients were referred to Pediatric Hematology Oncology, 902 of these patients for hematologic diagnoses. One hundred and three (11.4%) of 902 Hematology patients were referred for thrombocytopenia. The mean age of patients with thrombocytopenia was 9.2 years (s.d. 5.8yrs) and 58% were male. At the time of referral, 29% were categorized as mild, 33% were moderate, 17% severe and 21% very severe. The mean platelet count was 68 x103/mL (range 3-143). Younger patients had lower platelet counts (p<0.001), but no differences in mean platelet count were identified by gender (F:M 59 vs. 75 x103/mL, p=0.06). Thirty patients were categorized as mild thrombocytopenia at the time of referral. Only 2 (6%) patients needed eventual treatment (Crohn’s disease and SLE treated by subspecialist) and 7 (23%) patients had at least one episode of moderate thrombocytopenia. On their most recent platelet count, 17 (57%) patients remained categorized as mild, 1 (3%) was moderate, and 12 (40%) had a normal platelet count.  Thirty four patients were categorized as moderate thrombocytopenia at the time of referral. Only 5 (15%) patients needed eventual treatment (3 ITP patients for QOL/bruising/petechiae, one for SLE and one for NAIT) and 4 (12%) patients had at least one episode categorized as severe thrombocytopenia. On their most recent platelet count, 9 (26%) patients remained categorized as moderate, 10 (29%) were mild, and 15 (44%) had a normal platelet count. At time of diagnosis, 9 of 17 (53%) patients with severe thrombocytopenia and 19 of 22 (86%) of patients with very severe thrombocytopenia required interventions. Currently, 19 of the 39 (49%) patients have normal platelet counts.  Other known diagnoses for the 103 patients include: rheumatologic diagnoses (n=5), drug induced (n=5), NAIT (n=3), ADP deficiency (n=1), X-linked thrombocytopenia (n=1), HSP (n=1), HIV (n=1), and Crohn’s (n=1)

Conclusion: Mild and moderate thrombocytopenia does not often progress or require interventions.  Pediatricians should evaluate for Rheumatologic disorders in their initial work-up for mild to moderate thrombocytopenia as well as consider medication-induced thrombocytopenia. A safe and cost-effective approach to a patient with mild to moderate thrombocytopenia could be to observe repeat laboratory data (including ANA) in lieu of any other co-morbid condition. This approach could save families time, money, effort, and emotional stress in making appointments at large referral institutions.