Romiplostim in children with chronic immune thrombocytopenia (<scp>ITP</scp>): the <scp>F</scp>rench <scp>E</scp>xperience

Pasquet, Marlène; Aladjidi, Nathalie; Guiton, Corinne; Courcoux, Mary-France; Munzer, Martine; Auvrignon, Anne; Lutz, Patrick; Ducassou, Stéphane; Leroy, G.; Munzer, Caroline; Leverger, Guy

doi:10.1111/bjh.12609

Cited by 32 publications

(28 citation statements)

References 15 publications

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“…In the last few years, romiplostim and eltrombopag have been increasingly used in some haematological disorders. Reports of sustained remission after thrombopoietin receptor agonist discontinuation in adults (Červinek et al , ) and children (Kühne, ) suffering from ITP have been reported, and encouraging results have been published in some ES children, (Pasquet et al , ; Seidel et al , ). Its use in ES remains anecdotal, however, and might be clarified by future studies.…”

Section: Second and Further‐line Treatmentmentioning

confidence: 99%

How I manage Evans Syndrome and AIHA cases in children

Miano

2015

Br J Haematol

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The management of Evans Syndrome in children is challenging due to the lack of evidence-based data on treatment. Steroids, the first-choice therapy, are successful in about 80% of cases. For children who are resistant, relapse or become steroid-dependent, rituximab is considered a valid second-line treatment, with the exception of those with an underlying diagnosis of autoimmune lymphoproliferative syndrome who may benefit from other options such as mycophenolate mofetil and sirolimus. Better knowledge of the immunological mechanisms underlying cytopenias and the availability of new immunosuppressive drugs can be helpful in the choice of more targeted therapies that would enable the reduction of the use of long-term steroid administration or other more aggressive options, such as splenectomy or stem cell transplantation. This manuscript provides an overview of the pathogenic background of the disease, and suggests a clinical approach to diagnosis and treatment with a particular focus on the management of relapsing/resistant disease.

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Section: Second and Further‐line Treatmentmentioning

confidence: 99%

How I manage Evans Syndrome and AIHA cases in children

Miano

2015

Br J Haematol

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“…In the open‐label phase of these trials, 80% of patients were able to achieve a platelet count ≥50 × 10 9 /l at least once . Additional data about the use of these agents in pediatric ITP is limited to small single center retrospective reviews and case series focused on children with persistent and chronic ITP …”

Section: Introductionmentioning

confidence: 99%

“…[6,7] Additional data about the use of these agents in pediatric ITP is limited to small single center retrospective reviews and case series focused on children with persistent and chronic ITP. [9][10][11] Despite a paucity of literature regarding TPO-RA use in children, physicians are prescribing TPO-RAs to pediatric patients off-label and outside of clinical trials. Furthermore, physicians may not be reserving these agents exclusively for chronic primary ITP, the only group in whom these agents have been investigated and are indicated.…”

Section: Introductionmentioning

confidence: 99%

Thrombopoietin Receptor Agonist Use in Children: Data From the Pediatric ITP Consortium of North America ICON2 Study

Neunert

Despotovic

Haley

et al. 2016

Pediatr Blood Cancer

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Background Data on second-line treatment options for pediatric patients with immune thrombocytopenia (ITP) are limited. Thrombopoietin receptor agonists (TPO-RA) provide a non-immunosuppressive option for children who require an increased platelet count. Procedure We performed a multi-center retrospective study of pediatric ITP patients followed at ITP Consortium of North America (ICON) sites to characterize TPO-RA use. Results Seventy-nine children had a total of 87 treatments (28 eltrombopag, 43 romiplostim, and 8 trialed on both). The majority had primary ITP (82%) and most (60.8%) had chronic ITP. However, 22% had persistent ITP and 18% had newly diagnosed ITP. During the first three months of treatment, 89% achieved a platelet count ≥ 50 × 109/l (86% romiplostim, 81% eltrombopag, p=0.26) at least once in the absence of rescue therapy. The average time to a response was 6.4 weeks for romiplostim and 7.0 weeks for eltrombopag (p=0.83). Only 40% of patients demonstrated a stable response with consistent dosing over time. An intermittent response with constant dose titration was seen in 15%, and an initial response that waned to no response was seen in 13%. Significant adverse events were minimal with the exception of two patients with thrombotic events and one who developed a neutralizing antibody. Conclusions Our results demonstrate that TPO-RA agents are being used in children with ITP of varying duration and severity. The response was similar to clinical trials, but the sustainability of response varied. Future studies need to focus on the ideal timing and rationale for these medications in pediatric patients.

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“…39 All children had a bone marrow aspiration at diagnosis, which was compatible with ITP. The children were treated for 3-36 months with a romiplostim dose between 4-10 mg/kg, starting at 1 mg/kg with weekly escalation to a maximum of 10 mg/kg.…”

Section: Experience Of Tpo-ras In Pediatricsmentioning

confidence: 99%

Treatment of Pediatric Primary Immune Thrombocytopenia With Thrombopoietin Receptor Agonists

Kühne

2015

Seminars in Hematology

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Romiplostim in children with chronic immune thrombocytopenia (ITP): the French Experience

Cited by 32 publications

References 15 publications

How I manage Evans Syndrome and AIHA cases in children

How I manage Evans Syndrome and AIHA cases in children

Thrombopoietin Receptor Agonist Use in Children: Data From the Pediatric ITP Consortium of North America ICON2 Study

Treatment of Pediatric Primary Immune Thrombocytopenia With Thrombopoietin Receptor Agonists

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