ICON1: Treatment Decisions and Outcomes in Pediatric Refractory ITP
Ключови думи
Резюме
Описание
The purpose of this observational study is to model factors that determine physician treatment decisions in selecting specific second line agents in pediatric ITP and to determine the comparative effectiveness of second line ITP treatments by bleeding measures, platelet counts, and patient reported outcome measures. This prospective observational, longitudinal, multicenter cohort study will aim to collect routine clinical care data, quality of life information from patients, and decision making data from clinicians at enrollment and at regular clinical intervals for at least one year. The primary and secondary objectives are as follows:
Primary Objectives:
1. To model factors that determine physician treatment decisions in selecting specific second line agents in pediatric ITP.
2. To assess patient reported outcomes with relation to specific second line pediatric ITP therapies.
3. To determine the comparative effectiveness of second line ITP treatments in terms of bleeding and platelet counts.
Secondary Objectives:
1. To describe phenotypic variation among patients with refractory ITP;
2. To assess side effects and complications related to specific treatments for refractory ITP;
3. To describe monitoring and follow up practices among pediatric hematologists with each second line agent;
4. To weight factors that physicians use when deciding to treat pediatric ITP patients with second line agents;
5. To determine whether physician perception of patient quality of life correlates with patient derived quality of life measures;
6. To measure the correlation between the ITP Bleeding Scale and the Bleeding Assessment Tool in refractory pediatric ITP patients.
Дати
| Последна проверка: | 04/30/2020 |
| Първо изпратено: | 10/20/2013 |
| Очаквано записване подадено: | 10/23/2013 |
| Първо публикувано: | 10/28/2013 |
| Изпратена последна актуализация: | 05/18/2020 |
| Последна актуализация публикувана: | 05/20/2020 |
| Действителна начална дата на проучването: | 07/31/2013 |
| Приблизителна дата на първично завършване: | 03/31/2017 |
| Очаквана дата на завършване на проучването: | 03/31/2017 |
Състояние или заболяване
Интервенция / лечение
Drug: Refractory Pediatric ITP Patients
Фаза
Групи за ръце
| Arm | Интервенция / лечение |
|---|---|
| Refractory Pediatric ITP Patients Pediatric ITP patients, ages 1-18, starting a new second line ITP therapy, defined as not IVIG, steroids, anti-D, or aminocaproic acid. | Drug: Refractory Pediatric ITP Patients The treating physicians will select the second line agent and clinical data will be collected. |
Критерии за допустимост
| Възрасти, отговарящи на условията за проучване | 1 Year Да се 1 Year |
| Полове, допустими за проучване | All |
| Метод за вземане на проби | Non-Probability Sample |
| Приема здрави доброволци | Да |
| Критерии | Inclusion Criteria: - Immune Thrombocytopenia or Evans Syndrome - Ages > 12 months to <18 years - Starting a new second line therapy as defined as any therapy except IVIG, steroids, anti-D globulin, or aminocaproic acid - Starting a single agent/monotherapy Exclusion Criteria: - Evans Syndrome with a history of or current evidence of autoimmune hemolytic anemia - Unwillingness to be followed for 1 year - Physician providing care is unwilling to participate - Patient is starting multiple second line agents simultaneously |
Резултат
Първични изходни мерки
1. change from baseline in patient reported outcomes [Enrollment, 1 and 12 months]
2. change from baseline in bleeding assessment [Enrollment, 1, 6, and 12 months]
3. change from baseline in platelet count [over 1 year]
Вторични изходни мерки
1. side effects and complications of treatments [1 year]
