PB0238 - Tapering of trombopoietin receptor agonist in pediatric patients with chronic ITP. Is it posible?

Sunday, July 10, 2022

6:30 PM – 7:30 PM

Location: Exhibition

Presentation Format: In-Person

Presenting Author(s)

Rubén Berrueco, PhD

Head of Department. Pediatric Hematology
Hospital Sant Joan de Déu
Esplugues Llobregat, Catalonia, Spain

Co-Author(s)

MS

María Solsona

Junior Consultant. Pediatric Hematology
Hospital Sant Joan de Déu
Esplugues de Llobregat, Catalonia, Spain
ES

Elena Sebastián

Hematologist
Hospital Universitario Niño Jesús.
Madrid, Madrid, Spain
AC

Aúrea Cervera

Pediatrician
Hospital Universitario de Móstoles
Madrid, Madrid, Spain
AS

Ana Sastre

Pediatric Hemato-Oncologist
Hospital La Paz
Madrid, Madrid, Spain
IA

Itziar Astigarraga

Pediatric Hemato-oncologist
Hospital Universitario Cruces
Bilbao, Pais Vasco, Spain
BA

Bienvenida Argiles

Pediatric Hematologist
Hospital Universitario La Fe
Valencia, Comunidad Valenciana, Spain
MD

María Angeles Dasí

Pediatric Hematologist
Hospital Universitario La Fe
Valencia, Comunidad Valenciana, Spain
JD

José Luis Dapena

Pediatric Hemato-oncologist
Hospital Sant Joan de Déu
Barcelona, Catalonia, Spain
EM

Emilio Monteagudo

Pediatrician
Hospital Universitario La Fe
Valencia, Comunidad Valenciana, Spain

Background: Treatment approach to chronic immune thrombopenia (ITPc) in pediatric patients has changed during the last decade thanks to the development of thrombopoietin receptor agonist (ar-TPO). During the last decade, series in adult patients have described that up to 30% of patients with ITPc on ar-TPO treatment may have a sustained platelet response after drug withdrawal, but there is still scarce information in children.

Aims: To describe tapering of ar-TPO in ITPc pediatric patients treated in Spanish hospitals.

Methods: A multicenter retrospective observational study was performed in children with chronic immune thrombopenia (ITPc) to describe ar-TPO tapering and withdrawal in patients who had achieved a sustained complete response to ar-TPO.

Results: Ten pediatric patients with ITPc were included. Table 1 resumes patients characteristics and information regarding ar-TPO tapering. None of them required relevant dose increasing during this process. One case had an asymptomatic relevant thrombopenia ( < 30x109/L), but platelet count increased progressively without the need of dose escalation. After a median time of 24 months (SD 8.7), two patients relapsed (figure 1). One of them achieved a new complete response with ar-TPO. The other was refractory to ar-TPO and immunosuppressants but responded to rituximab. Three patients had a transient decrease of total platelet count (34-72x109/L) that improved without treatment administration.

Conclusion(s): This series shows that tapering and withdrawal of ar-TPO treatment can be considered in pediatric patients with ITPc who have achieved a sustained complete response. Our experience suggests that discontinuation should be progressive and slow.