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TREATMENT OF REFRACTORY IMMUNE THROMBOCYTOPENIA WITH THROMBOPOIETIN RECEPTOR AGONISTS: OUR EXPERIENCE IN CHILDHOOD
Author(s):
Joud Zanabili Al-Sibai
,
Joud Zanabili Al-Sibai
Affiliations:
Hematology,HUCA,Oviedo,Spain
Tamara Arias Fernández
,
Tamara Arias Fernández
Affiliations:
Hematology,HUCA,Oviedo,Spain
Maria Pilar Palomo Moraleda
,
Maria Pilar Palomo Moraleda
Affiliations:
Hematology,HUCA,Oviedo,Spain
Lucia Rita Morais Bras
,
Lucia Rita Morais Bras
Affiliations:
Hematology,HUCA,Oviedo,Spain
Christelle Castañón Fernández
,
Christelle Castañón Fernández
Affiliations:
Hematology,HUCA,Oviedo,Spain
Laura Francisca Ávila Idrovo
,
Laura Francisca Ávila Idrovo
Affiliations:
Hematology,HUCA,Oviedo,Spain
Antonio Solé Magdalena
,
Antonio Solé Magdalena
Affiliations:
Hematology,HUCA,Oviedo,Spain
Soledad González Muñiz
,
Soledad González Muñiz
Affiliations:
Hematology,HUCA,Oviedo,Spain
Maria Ángeles Fernandez Rodríguez
Maria Ángeles Fernandez Rodríguez
Affiliations:
Hematology,HUCA,Oviedo,Spain
(Abstract release date: 05/18/17) EHA Library. J. 05/18/17; 182835; PB2122
Mrs. Joud
Mrs. Joud
Contributions
PDF Abstract
Abstract

Abstract: PB2122

Type: Publication Only

Background
Immune thrombocytopenia (ITP) is an autoimmune disease in which antibodies develop against platelets (plts) and dysregulation of cellular immunity result in premature destruction of plts and impaired plt production. For most affected children, ITP is a self-limiting disease. Approximately, 10% of all ITP patients eventually develop refractory ITP (RITP). Thrombopoietin receptor agonists (TPO-RA) stimulate thrombopoiesis and are an alternative to Rituximab (Rtx) and splenectomy.

Aims
We present 3 different children with RITP treated with TPO-RA.

Methods
CASE 1

A 5-year-old girl admitted to the hospital due to ITP with mucocutaneous bleeding. She was refractory to corticoids, immune globulin (Ig) and anti-D Ig. Rtx was started. After the 3rd dose, she responded temporarily along with fever, renal insufficiency and arterial hypertension, probably related to Ig A deficiency, not previously diagnosed. Romiplostim was indicated, reaching complete remission after 2 doses and it was stopped after the 4th dose, without any adverse reaction. Nowadays, plt count remains within normal limits (pic.1A).
CASE 2
A 5-year-old boy was diagnosed of ITP with cutaneous bleeding. He received treatment with prednisone and Ig with short response. Rtx was indicated; after 4th dose, severe thrombocytopenia and cutaneous bleeding persisted. Eltrombopag was started with response after 6 weeks of treatment (pic.1B) and bleeding symptoms recovery.
CASE 3
A 4-years-old boy with RITP was referred to our hospital. We decided to initiate treatment with Eltrombopag. He developed response after 4 weeks of treatment with a dose of 75mg/24h. Six weeks later, he presented 600,000plts/µL, so the drug was stopped. We observed a quick descent in plts levels and Eltrombopag was restarted with progressive response (pic.1C).

Results
In all cases, splenectomy was avoided due to long-term risk of sepsis, as well as immunosuppressive agents like RTX in 3rd case. In 1st case, TPO-RA was able to stop with sustained response as described in some publications.

Conclusion
In our experience, TPO-RA appear to be efficacy and well tolerated in children.

Session topic: 32. Platelets disorders

Keyword(s): Thrombopoietin (TPO), Thrombocytopenia, Childhood

Abstract: PB2122

Type: Publication Only

Background
Immune thrombocytopenia (ITP) is an autoimmune disease in which antibodies develop against platelets (plts) and dysregulation of cellular immunity result in premature destruction of plts and impaired plt production. For most affected children, ITP is a self-limiting disease. Approximately, 10% of all ITP patients eventually develop refractory ITP (RITP). Thrombopoietin receptor agonists (TPO-RA) stimulate thrombopoiesis and are an alternative to Rituximab (Rtx) and splenectomy.

Aims
We present 3 different children with RITP treated with TPO-RA.

Methods
CASE 1

A 5-year-old girl admitted to the hospital due to ITP with mucocutaneous bleeding. She was refractory to corticoids, immune globulin (Ig) and anti-D Ig. Rtx was started. After the 3rd dose, she responded temporarily along with fever, renal insufficiency and arterial hypertension, probably related to Ig A deficiency, not previously diagnosed. Romiplostim was indicated, reaching complete remission after 2 doses and it was stopped after the 4th dose, without any adverse reaction. Nowadays, plt count remains within normal limits (pic.1A).
CASE 2
A 5-year-old boy was diagnosed of ITP with cutaneous bleeding. He received treatment with prednisone and Ig with short response. Rtx was indicated; after 4th dose, severe thrombocytopenia and cutaneous bleeding persisted. Eltrombopag was started with response after 6 weeks of treatment (pic.1B) and bleeding symptoms recovery.
CASE 3
A 4-years-old boy with RITP was referred to our hospital. We decided to initiate treatment with Eltrombopag. He developed response after 4 weeks of treatment with a dose of 75mg/24h. Six weeks later, he presented 600,000plts/µL, so the drug was stopped. We observed a quick descent in plts levels and Eltrombopag was restarted with progressive response (pic.1C).

Results
In all cases, splenectomy was avoided due to long-term risk of sepsis, as well as immunosuppressive agents like RTX in 3rd case. In 1st case, TPO-RA was able to stop with sustained response as described in some publications.

Conclusion
In our experience, TPO-RA appear to be efficacy and well tolerated in children.

Session topic: 32. Platelets disorders

Keyword(s): Thrombopoietin (TPO), Thrombocytopenia, Childhood

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