Quality of life is an important indication for second‐line treatment in children with immune thrombocytopenia

Shimano, Kristin A.; Neunert, Cindy; Bussel, James B.; Klaassen, Robert J.; Bhat, Rukhmi; Pastore, Yves D.; Lambert, Michele P.; Bennett, Carolyn M.; Despotovic, Jenny M.; Forbes, Peter; Grace, Rachael F.

doi:10.1002/pbc.29023

Cited by 8 publications

(15 citation statements)

References 22 publications

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“…Interestingly, the most frequently cited reason for initiating a second-line therapy in children with ITP was quality of life (e.g., limitations in daily tasks and reduced energy levels) with 73% of clinicians reporting this as a reason to treat. Bleeding was less commonly cited as a factor in initiating therapy (15-18%) [44]. Pharmacists who understand how patient preferences affect the decision to initiate and continue specific types of therapy in ITP may wish to include these counseling points to inform patients of their options and help bridge this disconnect [43].…”

Section: Itp Treatment Considerations For Pharmacistsmentioning

confidence: 99%

Thrombopoietin Receptor Agonists (TPO-RAs): Drug Class Considerations for Pharmacists

Gilreath

Bubalo

2021

Drugs

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The thrombopoietin receptor agonists (TPO-RAs) romiplostim, eltrombopag, avatrombopag, and lusutrombopag carry unique US Food and Drug Administration (US FDA)-and European Medicines Agency (EMA)-approved indications and may be used to increase platelet counts in a variety of conditions. Current indications for available TPO-RAs include treatment of chronic immune thrombocytopenia (ITP) in cases of insufficient response to prior treatment (avatrombopag, eltrombopag, romiplostim), management of thrombocytopenia in adult patients with chronic liver disease who are scheduled to undergo a procedure (avatrombopag, lusutrombopag), management of severe aplastic anemia (eltrombopag), and management of thrombocytopenia associated with interferon-based therapy for hepatitis C (eltrombopag). Across current indications, pharmacists can assist in stabilizing platelet counts and help to reduce large undulations commonly seen when starting, stopping, or transitioning between these agents. If therapy modifications may benefit the patient, pharmacists should discuss possible changes with the patient's treatment team or treating physician. When used for ITP, romiplostim, eltrombopag, and avatrombopag stimulate TPO receptors on hematopoietic stem cells (also known as c-Mpl, or CD110) to promote platelet production; however, romiplostim is the only TPO-RA that binds at the same site as endogenous TPO. These subtle mechanistic differences may explain why switching TPO-RA may be clinically advantageous in some situations. As pharmacists are called to counsel patients on TPO-RA use, a deep understanding of potential adverse events and management strategies, as well as appropriate monitoring, will increase the likelihood that patients meet their goals of therapy in the shortest timeframe. Other uses of TPO-RAs are also discussed in this review, including use following hematopoietic stem cell transplant, use in myelodysplastic syndrome, and use in chemotherapy-induced thrombocytopenia. Key PointsThrombopoietin receptor agonists (TPO-RAs) offer different clinical opportunities based on their pharmacokinetic, pharmacodynamic, and mechanistic characteristics.

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Section: Itp Treatment Considerations For Pharmacistsmentioning

confidence: 99%

Thrombopoietin Receptor Agonists (TPO-RAs): Drug Class Considerations for Pharmacists

Gilreath

Bubalo

2021

Drugs

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“…1 One of the goals of the ITP Consortium of North America (ICON)-1 prospective study was to describe the factors related to a physician's decision to initiate second-line treatments for ITP in children. 24 The study enrolled 120 children, median age 11.7 years (range 1.2-17.8 years) and 62% female. Patients had received a median of three prior treatments (range 0-8) and 53% had chronic ITP.…”

Section: Chi L Dr E N W Ith R Efr Ac Tory Itpmentioning

confidence: 99%

Burden of immune thrombocytopenia (ITP): Special considerations for refractory ITP

Rüfer,

Terrell

2023

Br J Haematol

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SummaryIt is known that patients with immune thrombocytopenia (ITP) have fatigue and impairment of health‐related quality of life (HRQoL). However, it is hypothesized that patients with refractory ITP have additional burdens that should be considered. Specifically, fatigue is more pronounced in patients with refractory disease, there are additional side effects from second‐ and third‐line treatments, additional anxiety about the long‐term course of the disease, impairment in HRQoL resulting from heavy menstrual bleeding and concerns related to family planning. The burden of disease, therefore, should be carefully assessed and considered in these patients. However, researchers have utilized numerous tools for evaluating HRQoL and fatigue, making comparison of data across studies challenging. There is a need to standardize assessment using either disease‐specific or generic instruments that can be easily implemented in routine clinical practice. Additionally, whether treatment of low platelet count and bleeding symptoms will have a positive influence on HRQoL remains to be seen and published evidence is conflicting. Nevertheless, improvement of HRQoL is a major treatment goal for both patients and physicians and should be especially considered when treating patients with refractory ITP.

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“…2,4 Patients with pITP have clinical manifestations of variable severity with unpredictable bleeding events and significant impairment of quality of life, particularly in the first few months after diagnosis. 5,6 Spontaneous remission of ITP occurs in about 62%-74% of children, and corticosteroids remain the commonly prescribed first-line therapy with a response in about 75% of ITP patients. For children with persistent/chronic ITP, thrombopoietinreceptor agonist (TPO-RA) is the preferred therapeutic option.…”

Section: Introductionmentioning

confidence: 99%

“…pITP is one of the most common bleeding disorders in children, with an estimated annual incidence of up to 6.4 per 100,000 children 2,4 . Patients with pITP have clinical manifestations of variable severity with unpredictable bleeding events and significant impairment of quality of life, particularly in the first few months after diagnosis 5,6 . Spontaneous remission of ITP occurs in about 62%–74% of children, and corticosteroids remain the commonly prescribed first‐line therapy with a response in about 75% of ITP patients.…”

Section: Introductionmentioning

confidence: 99%

Frequency of toll‐like receptor 4 variants and association with treatment response in children with primary immune thrombocytopenia

Ahmed,

Fahmy,

Abdelkreem

et al. 2023

Pediatric Blood & Cancer

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Objectives: To investigate the frequency of toll‐like receptor 4 (TLR4) variants c.896A>G (p.Asp299Gly) and c.1196C>T (p.Thr399Ile) among Egyptian children with primary immune thrombocytopenia (pITP), and their association with disease course and response to treatment.Methods: A case–control study that included 80 children with pITP and 50 age‐ and sex‐matched healthy controls. TLR4 c.896A>G and c.1196C>T variants were genotyped using polymerase chain reaction–restriction fragment length polymorphism. Patients were classified according to their response to treatment after 3 months as responders and nonresponders.Results: Compared with controls, children with pITP had significantly higher minor allele frequencies of TLR4 p.Asp299Gly (16.25% vs. 6%, odds ratio [OR] 3.04, 95% confidence interval [CI]: 1.16–9.36, p = .014) and p.Thr399Ile (20% vs. 4%, OR 6, 95% CI: 2.02–24.01, p < .001). The presence of p.Asp299Gly variant was significantly associated with chronic ITP (OR 7.78, 95% CI: 2.04–35.69, p < .001) and non‐response to therapy with steroid (OR 11.67, 95% CI: 1.32–104.08, p = .012), but not thrombopoietin‐receptor agonist (OR 1.67, 95% CI: 0.35–8.19, p = .464). Likewise, having p.Thr399Ile variant was significantly associated with chronic ITP (OR 5.14, 95% CI: 1.6–17.4, p = .002) and non‐response to therapy with steroid (OR 6.1, 95% CI: 1.01–49.06, p = .046) but not thrombopoietin‐receptor agonist (OR 1.57, 95% CI: 0.33–7.58, p = .515).Conclusion: The presence of TLR4 p.Asp299Gly or p.Thr399Ile variant may be associated with ITP predisposition, chronicity, and non‐response to upfront steroid therapy. These findings enhance our understanding of the complex pathophysiology of pITP with potentially important clinical implications.

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Quality of life is an important indication for second‐line treatment in children with immune thrombocytopenia

Cited by 8 publications

References 22 publications

Thrombopoietin Receptor Agonists (TPO-RAs): Drug Class Considerations for Pharmacists

Thrombopoietin Receptor Agonists (TPO-RAs): Drug Class Considerations for Pharmacists

Burden of immune thrombocytopenia (ITP): Special considerations for refractory ITP

Frequency of toll‐like receptor 4 variants and association with treatment response in children with primary immune thrombocytopenia

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