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

Abstract

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.

A Narrative Approach to Describe QoL in Children With Chronic ITP

“…A broader outlook at the therapeutic impact would result from the interpretation of these data together with narratives describing children receiving new-generation therapies (romiplostim), by enabling comparisons of new vs. traditional drugs. This approach would be in line with current research perspectives that emphasize both the inclusion of HRQoL among the indicators used to assess the effectiveness of romiplostim (43, 44) and the need to expand the pediatric sample size to understand the potential role of this new class of drugs in chronic cases (45).…”

Efficacy and safety of thrombopoietin receptor agonists in children with chronic immune thrombocytopenia: a meta-analysis

“…This result was in accordance with studies [ 6 , 13 , 35 ] on the treatment of chronic ITP in adults. TPO-RAs are drugs identical to endogenous TPOs, which can bind and activate the TPO-receptor to mimic TPO activity [ 5 , 11 ]. Activating platelet production via the TPO pathway, TPO-RAs often provide rapid and durable platelet responses [ 5 , 13 , 29 – 31 ].…”

“…Although ITP in most children is usually a benign condition that they can spontaneously recover from within half a year to one year [ 8 , 9 ], approximately 5–15% of pediatric patients with chronic ITP still require special treatment [ 1 , 8 – 10 ]. Meanwhile, chronic ITP in children can negatively affect their health-related quality of life (HRQoL) and impose a burden on their parents [ 8 , 11 , 12 ]. Thus, the primary goal of treatment for this disorder is to prevent bleeding by increasing the platelet count to a stable, safe level with the fewest possible treatment-associated adverse effects [ 8 , 13 – 15 ].…”

Újdonságok a gyermekkori immunthrombocytopenia kezelésében – 2017

“…A kezdeti dó-zis 1-2 μg/kg/hét, kéthetente 2 μg/kg/hét dózissal emelve, legfeljebb 10 μg/kg/hét maximális dózis eléré-séig. A kezdeti eredmények biztatóak, rezisztens esetekben is jó hatást érhetünk el, illetőleg a szteroidadag lényegesen csökkenthető [15][16][17]. A krónikus gyermekkori ITP jelentős hányadában elkerülhető a splenectomia.…”

“…A plazmaferézis jelenleg már szintén nem standard terápia, de számos ajánlás még megemlíti [10,17,21], nagyon különböző terápiás hatékonysággal (10-60%). Kimutatható, kifejezett ellenanyagképzés esetén megkí-sérelhető.…”