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3960 Real World Practices of Thrombopoietin Receptor Agonist Use and Discontinuation in Pediatric Patients with Immune Thrombocytopenia (ITP): A Single Center Retrospective Review

Program: Oral and Poster Abstracts
Session: 311. Disorders of Platelet Number or Function: Clinical and Epidemiological: Poster III
Hematology Disease Topics & Pathways:
Research, Clinical Practice (Health Services and Quality), Clinical Research, patient-reported outcomes, real-world evidence
Monday, December 11, 2023, 6:00 PM-8:00 PM

Morgan Pines, MD1, Melanie Degliuomini, MD2 and Shipra Kaicker, MD3*

1Department of Pediatric Hematology Oncology, Saint Peter's University Hospital, New Brunswick, NJ
2Department of Pediatric Hematology Oncology, Hackensack University Medical Center, New York, NY
3Department of Pediatric Hematology Oncology, Weill Cornell Medicine, Komansky Children’s Hospital at New York Presbyterian, New York, NY


Thrombopoietin Receptor Agonists (TPO-RAs) are becoming widely used as second-line treatment in patients with immune thrombocytopenia (ITP). These can be weaned/discontinued in adults. There is a paucity of literature on weaning and discontinuation practices in pediatric ITP.


We performed a retrospective review of pediatric patients aged 0-18 years with ITP treated with TPO-RAs at our institution over an eight-year period (2015-2022). Weaning and discontinuation practices were compared between patients based on timing of TPO-RA initiation: <3 months from diagnosis (group A), ≥3 to <12 months from diagnosis (group B), <12 months from diagnosis (group AB) or ≥12 months from diagnosis (group C).


There were 11, 12 and 32 patients in group A, B, and C respectively (table 1). As of last follow up, 10 patients successfully weaned and discontinued their TPO-RA. Eighteen patients were weaning their TPO-RA monotherapy and 15 continued a TPO-RA at maximal dose or required the addition of another agent, such as an immunosuppressant, to permit TPO-RA weaning. Five patients discontinued their TPO-RA due to poor response. Seven patients had stopped the TPO-RA due to an alternate reason (family preference, side effects, etc.). There was no difference in any of these outcomes based on timing of TPO-RA initiation.

In patients whose platelet count responded to TPO-RAs, the dose was weaned at the provider’s discretion. The dose was generally not weaned for a platelet count <50k.

Among only patients in which the TPO-RA monotherapy was weaned, patients in group AB had a shorter duration of TPO-RA treatment than patients in group C (IQR 12.1-48.9 months vs 37.5-88.0 months; p=0.019). While not statistically significant, after the start of the TPO-RA dose weaning, patients in group A weaned their TPO-RA at a faster rate (IQR 4.7-20.5% of maximal dose per month) compared to patients in groups B and C (IQR 1.8-5.1% of maximal dose per month and 1.5-5.5% of maximal dose per month).

The 10 patients who fully weaned their TPO-RA had a shorter duration of TPO-RA use compared to the 18 patients who partially weaned their TPO-RA monotherapy (IQR 11.5-38.6 months vs 27.6-82.1 months; p=0.018). Once the wean of TPO-RA dose was started, patients who fully weaned their TPO-RA did so at a faster rate than patients who partially weaned (IQR 5.6-29.6% of maximal dose per month vs 1.0-3.4% of maximal dose per month; p=0.017; figure 1).

Seven patients who fully weaned their TPO-RA had follow up CBCs between one and three months after discontinuation. Of these, five patients had platelet counts >100k, while the other two had platelet counts >40k that continued to rise to >80k within a year of discontinuation without further treatment. Of the remaining three patients, two had CBCs more than three months post TPO-RA discontinuation with platelet counts >100k without additional treatment and one had no follow up CBC after TPO-RA discontinuation. Two patients with follow-up had transient ITP exacerbations with platelet counts <10k at 14 and 31 months after TPO-RA discontinuation that were treated with anti-D/steroids and IVIG respectively with subsequent sustained improvement in platelet counts that did not require any further intervention.

There was no difference in the number of rescue medications (IVIG, anti-D, steroids) required between patient groups based on timing of TPO-RA initiation or type of agent used. Patients who were treated with both romiplostim and eltrombopag required adjunctive therapies (such as immunosuppressants) more than patients treated with either agent alone (p=0.022).


TPO-RAs were weaned and successfully discontinued in 10 of 55 (18.2%) pediatric patients with ITP in our small cohort with a low rate of subsequent ITP exacerbation. There was no difference in TPO-RA discontinuation rate in patients with a good platelet response based on timing of TPO-RA initiation. Patients who started a TPO-RA within 12 months of their ITP diagnosis had a shorter duration of TPO-RA use than patients who started a TPO-RA more than 12 months after diagnosis. Patients who started a TPO-RA within three months of diagnosis were able to wean their TPO-RA dose at a faster rate than patients who started a TPO-RA more than three months after diagnosis. TPO-RA dose was weaned at a faster rate in patients who fully weaned their TPO-RA compared to patients who were partially weaned. This data should be validated in a larger study.

Disclosures: No relevant conflicts of interest to declare.

*signifies non-member of ASH