Patient-centric care in primary immune thrombocytopenia (ITP): shared decision-making and assessment of health-related quality of life.

ABSTRACTImmune thrombocytopenia (ITP), an autoimmune disease characterized by low platelet counts and increased bleeding risk, can impair health-related quality of life (HRQoL), impacting patients' daily lives and mental health. A number of patient-reported outcome (PRO) measures (both generic and specific to ITP) can be used to understand the impact of ITP on HRQoL and generate evidence to guide disease management. As well-developed PRO tools could help in HRQoL assessment, their optimization could help to solidify a patient-centric approach to ITP management. Shared decision-making is a collaborative process between a patient and their healthcare professional in making decisions about care. Treatment decisions based on this shared process between physician and patient are recommended by clinical guidelines. The goal of this narrative review is to discuss treatment decisions with regards to patient-centric ITP management, with a focus on the impact of PRO measures and the process of shared decision-making in practice.

The psychiatric and psychological sequelae of vaccine-induced immune thrombotic thrombocytopenia (VITT).

This review paper explores the potential psychiatric and psychological sequelae of vaccine-induced immune thrombotic thrombocytopenia, also called vaccine-induced immune thrombocytopenia, and thrombosis (VITT). In the absence of any literature to date we have extrapolated data from similar conditions, particularly data pertaining to the critical care population. We discuss both the direct and indirect effects of thrombosis, likely psychiatric and psychological challenges during recovery, and ethical issues around vaccination. We have also suggested a comprehensive guide to the psychiatric assessment and management of patients presenting with VITT with the aim of early identification of problems and maximizing rehabilitation potential and quality of life.

Survival after acute episodes of immune-mediated thrombotic thrombocytopenic purpura (iTTP) - cognitive functioning and health-related quality of life impact: a descriptive cross-sectional survey of adults living with iTTP in the United Kingdom.

OBJECTIVES: Immune-mediated thrombotic thrombocytopenic purpura (iTTP) is an ultra-rare life-threatening thrombotic microangiopathy affecting adults with unpredictable disease onset and acute presentation. This study aimed to describe the health-related quality of life (HRQoL), cognitive functioning and work productivity of survivors following acute episode(s) of iTTP in the United Kingdom (UK). METHODS: An online survey was developed in collaboration with the TTP Network. Descriptive statistics were calculated for the health questionnaire Short Form Survey-36 Version 2 (SF-36v2), the Hospital Anxiety and Depression Score (HADS), the PROMIS Cognitive Function Abilities Subset - Short Form 6a (PROMIS CFAS - SF6a), and the Work Productivity and Activity Index: Specific Health Problem (WPAI-SHP), along with several iTTP-specific bespoke questions. RESULTS: Fifty participants were recruited between July-November 2019. The mean (standard deviation [SD]) standardized SF-36v2 physical and mental component scores were 42.16 (9.59) and 33.61 (12.34), lower than population norms. The mean (SD) standardized PROMIS CFAS - SF6a score was 39.69 (7.86), lower than population norms. HADS mean (SD) scores of 12.18 (3.14) and 11.78 (2.36) indicated moderate levels of anxiety and depression, respectively. Of those employed (58%), approximately 42.73% of participants reported work productivity loss due to their iTTP. Participants also reported experiencing flashbacks, fatigue interference in family, social and intimate life, and fears of relapse. DISCUSSION AND CONCLUSION: Regardless of recency of the last acute episode, participant scores signified impairments in all domains. Remission from an acute episode of disease does not signify the conclusion of care, but rather the requirement for long-term healthcare particularly focused on psychological support.

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

BACKGROUND: The decision to initiate second-line treatment in children with immune thrombocytopenia (ITP) is complex and involves many different factors. METHODS: In this prospective, observational, longitudinal cohort study of 120 children from 21 centers, the factors contributing to the decision to start second-line treatments for ITP were captured. At study entry, clinicians were given a curated list of 12 potential reasons the patient required a second-line treatment. Clinicians selected all that applied and ranked the top three reasons. RESULTS: Quality of life (QOL) was the most frequently cited reason for starting a second-line therapy. Clinicians chose it as a reason to treat in 88/120 (73%) patients, as among the top three reasons in 68/120 (57%), and as the top reason in 32/120 (27%). Additional factors ranked as the top reason to start second-line treatment included severity of bleeding (22/120, 18%), frequency of bleeding (19/120, 16%), and severity of thrombocytopenia (18/120, 15%). Patients for whom QOL (p = .006) or sports participation (p = .02) were ranked reasons were more likely to have chronic ITP, whereas those for whom severity (p = .003) or frequency (p = .005) of bleeding were ranked reasons were more likely to have newly diagnosed or persistent ITP. Parental anxiety, though rarely the primary impetus for treatment, was frequently cited (70/120, 58%) as a contributing factor. CONCLUSION: Perceived QOL is the most frequently selected reason pediatric patients start second-line therapies for ITP. It is critical that studies of treatments for childhood ITP include assessments of their effects on QOL.

Fatigue, executive function and psychological effects in children with immune thrombocytopenia: a cross-sectional study.

Childhood ITP is often considered to be a relatively mild haematological disorder, with only a minority of patients requiring treatment for troublesome bleeding. Over recent years, wider effects of the condition have been identified in some adults, particularly relating to fatigue and cognitive impairment. In this study, we sought to investigate such effects in a group of children with ITP and further our understanding of their psychological profile. Children attending routine haematology outpatient clinics and their parents were asked to complete standardised questionnaires designed to assess a range of psychological and cognitive factors. Although the majority of children had some scores within the normal range, a significantly high proportion had difficulties with fatigue (70·6%), emotional and behavioural symptoms (25·7%) or executive functioning (19·4%). Quality of life and subjective evaluation of the illness (appraisal) correlated significantly with each of these domains, but bleeding severity and platelet count did not. Our findings provide valuable insight into the broader impact of childhood ITP, which could aid in providing holistic care, potentially contribute to decisions regarding medical treatment, and guide future research.

Immunomodulatory Second-Line Therapies for Immune Thrombocytopenia.

Management of immune thrombocytopenia (ITP) is complex requiring communication between patients and caregivers to establish a mutual understanding of the impact of the patient's disease on quality of life, the current symptoms and risk of morbidity/mortality and the goals of therapy. The currently available second-line therapies for ITP provide potential for management of thrombocytopenia and bleeding symptoms with medical therapy or surgical intervention potentially offering long-term remission. All therapies are associated with potential side effects and necessary monitoring or modifications/risks and careful discussion of these is necessary to determine the optimal therapy for each patient. This review covers second-line therapies for ITP and discusses the currently available information on immunomodulatory second-line treatments for ITP.

First-Line Therapy for Immune Thrombocytopenia.

Immune thrombocytopenia (ITP) is an autoimmune disease affecting blood platelets that causes thrombocytopenia and an increased risk of bleeding. First-line therapy is indicated for patients with bleeding complications or who are at increased risk of bleeding, and the decision to initiate therapy depends not only on the platelet count, but also on other endpoints including quality of life. The choice of first-line therapy depends primarily on how quickly a platelet count response is required, with intravenous immune globulin providing the more rapid response, followed by high-dose dexamethasone and prednisone. In this narrative review, we discuss key issues with first-line therapy in ITP including when to initiate therapy, treatment options and special considerations for children. Evidence-based guidelines are lacking for the emergency management of patients with ITP who present with significant bleeding; we provide our approach to this critical situation.

Thrombopoietin Receptor Agonist Use for Immune Thrombocytopaenia.

Management of patients with corticosteroid-refractory immune thrombocytopaenia (ITP) possesses a significant challenge to practitioners. Until recently, options included splenectomy and immunosuppression. With improved knowledge of both thrombopoiesis and the pathophysiology of ITP, novel drug development with thrombopoietin-receptor agonists (TPO-RAs) was undertaken. Two agents, romiplostim and eltrombopag, are currently approved for use in patients with chronic ITP. Both agents have been shown to increase the platelet count, improve health-related quality of life and reduce bleeding symptoms and concomitant medication use. This review will highlight the discovery of TPO-RA agents, appraise key clinical trials and explore future directions.

Management of Adult Chronic Immune Thrombocytopenia in Japan: Patient and Hematologist Perspectives from a Multi-center Cross-sectional Questionnaire Survey.

Objective The objective of this study was to explore the perspective of hematologists and their patients regarding the management of adult chronic immune thrombocytopenia (ITP). Methods This was a multi-center, questionnaire-based, cross-sectional study conducted between 2012 and 2013 throughout Japan. Patients Hematologists, members of the Japanese Society of Hematology in 171 institutions, and their patients were invited to participate in this study. The hematologists were mainly asked about their treatment strategies, while patients were asked about their opinion of the applied treatments, treatment effect, impact on their quality of life (QOL), and treatment satisfaction. Results Questionnaires from 204 hematologists and 213 patients were collected. One hundred sixty hematologists (78.4%) started treatment based on the patient's platelet count. Corticosteroids were considered to be the most effective treatment (44.1%). Forty-six percent of hematologists responded that treatment would be started after the platelet count fell below 20×10(9)/L with bleeding symptoms, compared to 62.9% for patients with no bleeding symptoms. A platelet count of 50×10(9)/L or lower was acceptable for 94.0% of hematologists and 66.8% of patients. Fatigue was most frequently experienced by patients (44.6%). Patients also experienced psychological symptoms (feeling of anxiety or depressive mood: 29.1%, labyrinthitis: 23.5%). While 70.6% of hematologists assumed that the patient QOL was impaired to a moderate to substantial degree, the QOL was impaired in 34.3% of patients. Conclusion A substantial gap which exists between hematologists and their patients highlights a need for better understanding of potential conflicts for establishing effective strategies for ITP management.

Health-related quality of life and burden of fatigue in patients with primary immune thrombocytopenia by phase of disease.

The main objective of this study was to compare health-related quality of life (HRQOL) of primary immune thrombocytopenia (pITP) patients with that of general population, overall, and by patient group (i.e., newly diagnosed, persistent, and chronic patients). Fatigue was also investigated as a secondary objective. Overall, 424 adult patients were enrolled in a multicenter observational study and the control group consisted of a representative sample from the general population. Propensity score matching plus further multivariate linear regression adjustment was used to compare HRQOL outcomes between pITP patients and general population. Mean age of patients was 54 years. Of those with HRQOL assessment, 99 patients (23.6%) were newly diagnosed, 53 (12.6%) were persistent, and 268 (63.8%) were chronic pITP patients. Comparison by patient group versus their respective peers in the general population revealed greater impairments in persistent pITP patients. Persistent pITP patients reported clinically meaningful impairments in physical functioning (-15; 95% CI -24.1 to -5.8; P = 0.002), social functioning (-15.3; 95% CI -25.5 to -5.1; P = 0.004), role physical (-28.4; 95% CI -43.1 to -13.7; P < 0.001), role emotional (-23.9; 95% CI -40.1 to -7.7; P = 0.004), and mental health scales (-11.3; 95% CI -21.2 to -1.4; P = 0.026) of the SF-36 questionnaire. Higher fatigue severity was associated with lower physical and mental HRQOL outcomes. Our findings suggest that the burden of the disease and treatment might depend on the disease phase and that persistent pITP patients are the most vulnerable subgroup. Am. J. Hematol. 91:995-1001, 2016. © 2016 Wiley Periodicals, Inc.

Health-related quality of life in children with chronic immune thrombocytopenia in China.

BACKGROUND: The concept of health-related quality of life (HRQoL) was brought up decades ago and has been well utilized in many different areas. Regarding immune thrombocytopenia (ITP) management, much work has been done to emphasize the necessity of taking HRQoL into consideration. However, data on HRQoL of children with chronic ITP remain rare. METHODS: This is a cross-sectional study. Children with chronic ITP aged from 2 to 18 and their parents were recruited. Participants completed the Pediatric Quality of Life Inventory™ (PedsQL™) and Kids' ITP Tools (KIT) questionnaires at only one time. The Pearson's correlation was examined between these measures for the pooled samples. RESULTS: A total of 42 families participated. Mean child self-report scores of KIT and PedsQL™ were 78.60 (SD = 12.40) and 85.13 (SD = 14.12), respectively, corresponding to parent proxy report scores, which were 73.40 (SD = 19.96) and 85.10 (SD = 13.56), respectively. Mean score of KIT parent impact report was only 40.39 (SD = 19.96). Significantly higher KIT scores (self-report and parent proxy) were observed in children with PLT more than 30 × 10*9/L compared to others, and this difference was even more noticeable in the PedsQL™ parent proxy report group (p < 0.001). As with intravenous immunoglobulin, the statistics difference appeared only in KIT child self-report group (p = 0.03), while for bone marrow examination, the difference appeared only in PedsQL™ parent proxy report group (p = 0.01). A negative relationship was apparent between duration of disease and scores. Gender and use of corticosteroids had no impact on the KIT and PedsQL™ scores here. Internal consistency reliability was demonstrated with Cronbach's alpha for all scales above the acceptable level of 0.89 (range from 0.88 to 0.97). There was a substantial concordance (p < 0.001) between the child and parent proxy scores (ICC for KIT is 0.59, ICC for PedsQL™ is 0.85). Meanwhile, KIT scores are correlated with PedsQL™ (r = 0.75 for child self report, r = 0.61 for parent proxy report). CONCLUSIONS: ITP affects HRQoL of children and parents. Parents are much more concerned with the disease than their children, which seriously influence their HRQoL as a result. The cross-culture translated KIT is reliable and valid with acceptable correlation to the PedsQL™. KIT provides valuable information of childhood ITP and will be a reliable outcome measure for further clinical research on HRQoL.

Health-related quality of life in children with newly diagnosed immune thrombocytopenia.

Despite its generally transient and benign course, childhood immune thrombocytopenia has a large impact on health-related quality of life. Recently published guidelines state that quality of life should be taken into account while making decisions on management in childhood immune thrombocytopenia. We, therefore, assessed health-related quality of life in children with newly diagnosed immune thrombocytopenia in a prospective multicenter study. One hundred and seven children aged 6 months-16 years (mean age 5.57 years) were included. We used Pediatric Quality of Life Inventory™ and Kids' ITP Tools questionnaires at diagnosis and during standardized follow-up. Scores on the Pediatric Quality of Life Inventory™ Core Scales were compared with those of healthy children. Relationships between health-related quality of life scores and treatment modality, bleeding tendency and course of the disease were examined. Kids' ITP Tools proxy reports and parent self-reports showed significant higher health-related quality of life scores in children who recovered than in children with persistent immune thrombocytopenia (at 3 months: Kids' ITP Tools parent self-report score 80.85 for recovered patients (n=69) versus 58.98 for patients with persistent disease (n=21), P<0.001). No significant differences in health-related quality of life were found between children with mild or moderate bleeding or between children who received intravenous immunoglobulin or children who were carefully observed. In conclusion, health-related quality of life of children with newly diagnosed immune thrombocytopenia is not influenced by treatment modality or bleeding severity, but only by clinical course of the disease. (Dutch Trial Register identifier: NTR TC1563).

ITP-QoL questionnaire for children with immune thrombocytopenia: Italian version validation's.

BACKGROUND: The ITP-QoL is a disease-specific questionnaire for the assessment of health-related quality of life (HRQoL) in children with immune thrombocytopenia (ITP) and their parents. The aim of this study was to test the psychometric characteristics of the ITP-QoL in the Italian pediatric population in terms of validity and reliability. PROCEDURE: Children aged 8-16 years with acute or chronic ITP and their parents were recruited in Italy. Participants completed the ITP-QoL together with other patient-reported outcomes (PROs). Reliability was calculated using Cronbach's alpha. Convergent validity was determined by means of the Pearson correlation coefficients. RESULTS: A total of 91 ITP patients, mean age of 12.11 ± 2.47 years, and their parents participated; 61.5% of the patients were female. Two patients had acute ITP and 30.2% had a moderate to severe status of ITP. Cutaneous symptoms were more frequent than mucosal symptoms. Due to item and scale analyses 20 items were deleted from the original ITP-QoL. Internal consistency of the ITP-QoL was found to be good with Cronbach's alpha exceeding α = 0.70 for all but one subscale. Concerning convergent validity "moderate" to "high" negative correlations were found between ITP-QoL and KINDL subscales. The ITP-QoL was able to discriminate between clinical subgroups such as number of days lost at school due to ITP and hospitalization. CONCLUSIONS: Our study was able to demonstrate that the Italian version of ITP-QoL (for children aged 8-16 years) is a valid and reliable instrument for the assessment of HRQoL in children with ITP.

Shared decision making in the management of children with newly diagnosed immune thrombocytopenia.

This study aimed to examine the treatment decision-making process for children hospitalized with newly diagnosed immune thrombocytopenia (ITP). Using focus groups, we studied children with ITP, parents of children with ITP, and health care professionals, inquiring about participants' experience with decision support and decision making in newly diagnosed ITP. Data were examined using thematic analysis. Themes that emerged from children were feelings of "anxiety, fear, and confusion"; the need to "understand information"; and "treatment choice," the experience of which was age dependent. For parents, "anxiety, fear, and confusion" was a dominant theme; "treatment choice" revealed that participants felt directed toward intravenous immune globulin (IVIG) for initial treatment. For health care professionals, "comfort level" highlighted factors contributing to professionals' comfort with offering options; "assumptions" were made about parental desire for participation in shared decision making (SDM) and parental acceptance of treatment options; "providing information" was informative regarding modes of facilitating SDM; and "treatment choice" revealed a discrepancy between current practice (directed toward IVIG) and the ideal of SDM. At our center, families of children with newly diagnosed ITP are not experiencing SDM. Our findings support the implementation of SDM to facilitate patient-centered care for the management of pediatric ITP.

Health-related quality of life of Egyptian children with immune thrombocytopenia and their parents.

Health-related quality of life has been recognized as an important pediatric outcome measurement. Kid's ITP Tool was used to measure health-related quality of life of 80 Egyptian children with immune thrombocytopenia and their parents in relation to different disease parameters. A positive correlation between scores of child/proxy reports and parent report was found. Patients with newly diagnosed immune thrombocytopenia had significantly lower scores of both child/proxy reports and parent reports than chronic patients. Longer duration of illness was correlated with higher scores of child/proxy reports. Negative correlations were found between severity of bleeding and both scores of child/proxy reports and parent reports. Platelet count was positively correlated with parent report score. Regression analysis revealed that severity of bleeding had the highest significant impact on parent report score. Improving parents' knowledge about the pathogenesis and course of the disease may improve their quality of life.

Quality of life in childhood immune thrombocytopenia: international validation of the kids' ITP tools.

BACKGROUND: The Kids ITP Tools (KIT) is a disease-specific measure of health-related quality of life for children with immune thrombocytopenia (ITP). To facilitate use in international trials it has been cross-culturally adapted for France, Germany, the United Kingdom and Uruguay. This study assessed the validity and reliability of the translated KIT in comparison to generic quality of life measures. METHODS: Children 2-18 years of age with ITP and their parents were recruited in France, Germany, the United Kingdom and Uruguay. Participants completed the KIT, PedsQL and KINDL. We examined the Pearson's correlation between these measures for our pooled sample and estimated the reliability over a 2-week time period. Findings were further explored by country. RESULTS: A total of 127 families (81 children self-reported) participated. Mean child-reported scores were: KIT 74.3 (SD = 15.3), PedsQL 81.3 (SD = 13.0), and KINDL 70.5 (SD = 14.3). Corresponding mean parent proxy-reported scores were: 70.6 (SD = 18.1), 75.7 (SD = 16.8) and 72.3 (SD = 12.7), respectively. Correlation between KIT and the generic measures was consistent with our a priori hypothesis (PedsQL r = 0.54, KINDL r = 0.48, both P < 0.0001). Child KIT scores for newly diagnosed ITP patients were significantly lower than for chronic ITP patients (67.3 vs. 77.3; P = 0.005). There was a significant correlation (P < 0.001) between the child and parent proxy KIT scores (ICC = 0.52). Child KIT test-retest reliability was acceptable at 0.71. CONCLUSIONS: The cross-culturally translated KIT is valid and reliable with acceptable correlation to the PedsQL and KINDL. There is a significant difference in child self-reported KIT scores between newly diagnosed and chronic ITP.

Single incision versus reduced port splenectomy--searching for the best alternative to conventional laparoscopic splenectomy.

BACKGROUND: Laparoscopic splenectomy (LS) is a well accepted approach for the treatment of multiple hematologic diseases. Single port access splenectomy (SPAS) emphasizes the concept of surgery through one small incision. The reduced port access splenectomy (RPAS) entails the use of fewer trocars of smaller sizes. The aim of this study was to compare the clinical outcomes after LS, SPAS, and RPAS, and to analyze the aesthetic result and patient satisfaction. METHODS: We included patients who underwent LS (group 1, n = 15), SPAS (group 2, n = 8), and RPAS (group 3, n = 10) between June 2008 and February 2012, whose final spleen weight was less of 500 g. The outcome parameters analyzed were operative time, need of additional trocars, blood loss, blood transfusion, weight of the spleen, postoperative complications, and duration of hospital stay. To evaluate the cosmetic result, patients were asked to take the Body Image Questionnaire. RESULTS: Patients in group 3 were younger than group 1. Operative time was significantly longer in group 2 compared to groups 1 and 3 (83 ± 19 vs. 131 ± 43 vs. 81 ± 22 min, p = 0.01). There was no need to convert to open surgery in any group, nor were there differences in intra- or postoperative outcome. There were no differences between the groups in relation to the analgesic requirements. Twenty-two out of the 33 patients answered the questionnaire. There was a significant advantage in group 2 and 3 in the body image index with respect to group 1. There were no differences between groups 2 and 3 (7.3 ± 2.8 vs. 5.8 ± 1.3 vs. 5.1 ± 0.4, p < 0.02). CONCLUSIONS: RPAS is a good alternative to LS and SPAS. It improves the aesthetic results as compared to LS, whereas minimizes the technical challenges faced with SPAS.

Health-related quality of life in nonsplenectomized immune thrombocytopenia patients receiving romiplostim or medical standard of care.

Immune thrombocytopenia (ITP) is an autoimmune disorder characterized by platelet destruction and insufficient platelet production. The resulting thrombocytopenia reduces patient health-related quality of life (HRQOL). In a randomized, open-label, 52-week study of non-splenectomized ITP patients treated with romiplostim or medical standard of care (SOC), patients completed the 10-scale ITP-patient assessment questionnaire (PAQ) at the start of the study and after 12, 24, 36, 48,and 52 weeks of treatment. HRQOL changes were examined for all patients in both treatment groups and by responder status, splenectomy status, and after the use of rituximab. Patients in both groups showed marked increases in all HRQOL scales over 52 weeks of treatment.These change scores exceeded the minimally important difference values (a measure of clinical relevance) for most of these scales,especially in responders to treatment. Compared with baseline,patients receiving romiplostim showed statistically significant improvements compared to SOC over 52 weeks for the ITP-PAQ scales of Symptoms, Bother, Activity, Psychological Health, Fear, Overall QOL,and Social QOL. Overall, treatment of ITP was associated with improvement in HRQOL. Patients receiving romiplostim had greater HRQOL improvements than those receiving SOC, but the magnitude ofthe difference is of uncertain clinical benefit.