Use of Romiplostim for Primary Immune Thrombocytopenia in Children

Vilaplana, Vicente Escudero; Aragonés, J. Huerta; Fernández-Llamazares, Cecilia M.; Bieler, Cristina Beléndez; Manrique-Rodríguez, Silvia; Sáez, María Sanjuro

doi:10.3109/08880018.2011.629401

Cited by 23 publications

(6 citation statements)

References 32 publications

(46 reference statements)

Supporting

Mentioning

Contrasting

Order By: Relevance

“…In 2012, a general hospital in Madrid, Spain published a retrospective, longitudinal observational study of three pediatric patients refractory to treatment who were treated with romiplostim ( 15 ). Among the three patients, one was newly diagnosed and two had chronic ITP.…”

Section: Experience In Pediatricsmentioning

confidence: 99%

Use of Thrombopoietin Receptor Agonists in Childhood Immune Thrombocytopenia

Garzon

Mitchell

2015

Front. Pediatr.

View full text Add to dashboard Cite

Most children with immune thrombocytopenia (ITP) will have spontaneous remission regardless of therapy, while about 20% will go on to have chronic ITP. In those children with chronic ITP who need treatment, standard therapies for acute ITP may have adverse effects that complicate their long-term use. Thus, alternative treatment options are needed for children with chronic ITP. Thrombopoietin receptor agonists (TPO-RA) have been shown to be safe and efficacious in adults with ITP, and represent a new treatment option for children with chronic ITP. One TPO-RA, eltrombopag, is now approved for children. Clinical trials in children are ongoing and data are emerging on safety and efficacy. This review will focus on the physiology of TPO-RA, their clinical use in children, as well as the long-term safety issues that need to be considered when using these agents.

show abstract

Section: Experience In Pediatricsmentioning

confidence: 99%

Use of Thrombopoietin Receptor Agonists in Childhood Immune Thrombocytopenia

Garzon

Mitchell

2015

Front. Pediatr.

View full text Add to dashboard Cite

show abstract

“…Several real-world studies and case reports have assessed romiplostim in children with newly diagnosed ITP [ 29 – 34 ] (Table 1 ). The study with the largest cohort of children with newly diagnosed ITP who received romiplostim was a retrospective multicenter chart review, performed at 12 sites in the Pediatric Immune Thrombocytopenia Consortium of North America (ICON) [ 29 ].…”

Section: Romiplostim For Children With Newly Diagnosed Itpmentioning

confidence: 99%

“…chronic ITP), 86% of children achieved a platelet count ≥ 50 × 10 9 /l with romiplostim at least once • 73% achieved a platelet count ≥ 20 × 10 9 /l above baseline for 2 consecutive weeks without requiring new or increased concomitant ITP treatment Ramaswamy et al (2014) [ 30 ] USA Retrospective observational study (N = 33) Newly diagnosed, n = 2 Persistent, n = 5 • Both children with newly diagnosed ITP and 4/5 with persistent ITP showed a platelet response ≥ 50 × 10 9 /l • Newly diagnosed: 1/2 achieved a CR; 1/2 achieved a PR • Persistent: 3/5 achieved a CR; 1/5 achieved a PR Suntsova et al (2020) [ 31 ] Russia Retrospective observational study (N = 6) Newly diagnosed, n = 6 • CR was achieved in 5/6 patients 4–8 weeks after initiating therapy • Three children remained in remission for 1–3 years after the discontinuation of romiplostim Grace et al (2019) [ 32 ] USA Prospective observational study (N = 120) Newly diagnosed, n = 6 Persistent, n = 9 • Overall (incl. chronic ITP) at month 6, 71% of patients achieved a CR and 15% achieved a PR Suntsova et al (2017) [ 33 ] Russia Prospective observational study (N = 20) Newly diagnosed, n = 1 Persistent, n = 3 • Newly diagnosed: platelet count increased from 3 × 10 9 /l to 16 × 10 9 /l after treatment with romiplostim and eltrombopag; the patient was deemed a non-responder to both treatments • Persistent: 2/3 were considered responders to romiplostim Escudero Vilaplana et al (2012) [ 34 ] Spain Retrospective case report (N = 3) Newly diagnosed, n = 1 • The time for CR was 14 days, and response was maintained for 6 months • Median platelet count was 215 × 10 9 /l over the 6 months CR , complete response; ITP , immune thrombocytopenia; PR , partial response …”

Section: Romiplostim For Children With Newly Diagnosed Itpmentioning

confidence: 99%

Romiplostim in children with newly diagnosed or persistent primary immune thrombocytopenia

et al. 2021

View full text Add to dashboard Cite

Immune thrombocytopenia (ITP) is a disease of heterogenous origin characterized by low platelet counts and an increased bleeding tendency. Three disease phases have been described: newly diagnosed (≤ 3 months after diagnosis), persistent (> 3–12 months after diagnosis), and chronic (> 12 months after diagnosis). The majority of children with ITP have short-lived disease and will not need treatment. For children with newly diagnosed ITP, who have increased bleeding symptoms, short courses of steroids are recommended. In children who do not respond to first-line treatment or who become steroid dependent, thrombopoietin receptor agonists (TPO-RAs) are recommended because of their efficacy and safety profiles. In this narrative review, we evaluate the available evidence on the use of the TPO-RA romiplostim to treat children with newly diagnosed or persistent ITP and identify data from five clinical trials, five real-world studies, and a case report. While the data are more limited for children with newly diagnosed ITP than for persistent ITP, the collective body of evidence suggests that romiplostim is efficacious in increasing platelet counts in children with newly diagnosed or persistent ITP and may result in long-lasting treatment-free responses in some patients. Furthermore, romiplostim was found to be well tolerated in the identified studies. Collectively, the data suggest that earlier treatment with romiplostim may help children to avoid the side effects associated with corticosteroid use and reduce the need for subsequent treatment.

show abstract

“…Seven retrospective observational studies of children treated with romiplostim for chronic ITP have been published. Escudero Vilaplana et al31 reported on two children with chronic ITP who were treated with romiplostim. A 10 year old was followed for 27 weeks with maintenance of a complete response defined as a platelet count >100,000/µL for 10 weeks and a partial response defined as a platelet count of 30,000–100,000/µL and a platelet count two times the baseline count for 4 weeks.…”

Section: Introductionmentioning

confidence: 99%

Spotlight on romiplostim in the treatment of children with chronic immune thrombocytopenia: design, development, and potential place in therapy

Buchbinder¹,

Nugent²,

Hsieh³

2017

DDDT

View full text Add to dashboard Cite

Primary immune thrombocytopenia (ITP) is an autoimmune disorder characterized by isolated thrombocytopenia. In approximately one-third of cases, the duration of thrombocytopenia will extend beyond 12 months consistent with a diagnosis of chronic ITP. Minor bleeding manifestations are common in chronic ITP while severe or life-threatening bleeding complications are uncommon. Moreover, spontaneous resolution occurs in the majority of children with chronic ITP necessitating treatment in only those children with ongoing bleeding manifestations or impairment in health-related quality of life (HRQOL). The characterization of thrombopoietin (TPO) and remarkable advancements in our understanding of the pathophysiology of ITP has led to the development of a new class of agents, the TPO-receptor agonists that have documented efficacy in the amelioration of thrombocytopenia and bleeding manifestations in chronic ITP. Romiplostim is a second-generation TPO-receptor agonist that has undergone limited evaluation in the treatment of chronic ITP in children. Evolving data suggest that romiplostim may be a safe and effective agent in the treatment of chronic ITP in children. Additional data are needed to confirm its ability to increase platelet counts, decrease bleeding manifestation, and improve the HRQOL of children and caregivers impacted by chronic ITP.

show abstract

Use of Romiplostim for Primary Immune Thrombocytopenia in Children

Cited by 23 publications

References 32 publications

Use of Thrombopoietin Receptor Agonists in Childhood Immune Thrombocytopenia

Use of Thrombopoietin Receptor Agonists in Childhood Immune Thrombocytopenia

Romiplostim in children with newly diagnosed or persistent primary immune thrombocytopenia

Spotlight on romiplostim in the treatment of children with chronic immune thrombocytopenia: design, development, and potential place in therapy

Contact Info

Product

Resources

About