Patterns and influences in health‐related quality of life in children with immune thrombocytopenia: A study from the Dallas ITP Cohort

Abstract

HRQoL is low at diagnosis but significantly improves over time. Patients with ongoing disease and lower platelet counts continue to have significant disease burden.

Immune Thrombocytopenia - Current Diagnostics and Therapy: Recommendations of a Joint Working Group of DGHO, ÖGHO, SGH, GPOH, and DGTI

“…This is particularly true during the initial phase of the disease when bleeding symptoms are still frequent and when the patients and their relatives must first learn how to cope with thrombocytopenia [150]. In addition to the bleeding symptoms and low platelet counts, ITP patients experience numerous other limitations: -ITP-associated non-bleeding symptoms: cognitive impairment, fatigue, weakness, depression, -increased risk of infection (this is one of the most frequent causes of death in ITP besides bleeding), -long, sometimes even lifelong therapy, -side effects of ITP treatments (especially due to steroids) [151], -gender-specific restrictions (risk of bleeding during pregnancy and delivery, risk of thrombocytopenia in the newborn), -social stigmatization from visible hematomas (sports, beach, swimming pool), -increased risks when treating other diseases (increased risk of bleeding from anticoagulation for arrhythmias or coronary heart disease), -time spent on doctor and hospital visits, -reduced productivity, -co-payments and other costs of therapy ('financial toxicity'), -limited experience of other medical disciplines in dealing with this rare disease and frequent referral and travels to centers with experience in ITP even for simple routine procedures, -changes in lifestyle, -restrictions at work and at home, limitations when traveling, -restrictions or higher costs for health or other insurances.…”

<p>Romiplostim for the Treatment of Immune Thrombocytopenia: Spotlight on Patient Acceptability and Ease of Use</p>

“…69 Parental fear of bleeding significantly impacts HRQoL for caregivers with parental impact scores being lower than child-report score. 65,69 The reduction of parental burden in the setting of a high response rate to romiplostim supports the idea that parental burden and worry are likely dependent on platelet count and therapy response. Conversely, the lack of improvement in self-report and parent-proxy scores suggests other factors such as activity limitations, medical visits, and anxiety with venipuncture likely drive decreased HRQoL scores among children.…”

“…Additionally, substantial caregiver strain is well documented. 63,65 Generic HRQoL tools were initially employed; however, ITP specific HRQoL tools have been developed and validated to better explore diseasespecific symptoms and issues. HRQoL tools are specific to adult or pediatric populations.…”

“…HRQoL improves for both children and caregivers after 12 months of disease, reflective of the natural progression of pediatric disease with most spontaneously resolving by 12 months. 63,65 Interestingly, improvement in HRQoL scores also occurs in chronic patients after 12 months, compared to HRQoL scores shortly after diagnosis. 63 The Kids' ITP Tool (KIT) was developed to further elucidate the impact of disease burden on patients and caregivers and has been shown to be valid and reliable.…”

Trombocitopenia inmune. Guía de diagnóstico y tratamiento

“…Es recomendable que la conducta terapéutica por seguir sea consensuada entre el paciente, los padres y el médico tratante, luego de evaluar los distintos beneficios y riesgos de los tratamientos disponibles (Figura 2). 45 46,47 Se podrá optar por administrar en forma periódica algunos de los tratamientos para PTI de reciente diagnóstico o por indicar o t r a s t e r a p i a s d e n t r o d e l a s s i g u i e n t e s opciones: [11][12][13]15,28,31,[36][37][38][39]41,43, Esplenectomía:…”