The Lack of Tolerable Treatments Options That Can Induce Durable Responses without Fear of Relapse after Discontinuation Represents a Significant Unmet Need for Patients (Pts) with Immune Thrombocytopenia (ITP): Results from the ITP World Impact Survey (I-WISh) 2.0
Bibliographic record
Abstract
Background: ITP is an acquired autoimmune disease characterized by transient or persistent thrombocytopenia. ITP substantially impairs pts' quality of life (QoL), including daily activities, social interactions, work and emotional wellbeing, with fatigue being the most common symptom reported by pts. Corticosteroids are the standard first-line treatment for ITP, while thrombopoietin receptor agonists (TPO-RAs), rituximab and fostamatinib are recommended for later lines; however, these treatments have limited sustained efficacy and may be associated with short- and long-term side effects that impact QoL. Aim: To assess pt and physician (MD) perceptions of current ITP treatment and treatment goals using data from I-WISh 2.0. Methods: I-WISh 2.0 was developed using findings from I-WISh 1.0 and surveyed 1018 pts with ITP aged >18 years and 431 MDs who managed ≥3 pts with ITP. I-WISh 2.0 was conducted from February to July 2022 in 15 countries. The surveys, developed by a steering committee (supported by Novartis) comprising expert ITP MDs and pt advocates, included questionnaires about prescribed ITP treatments, their impact on QoL and treatment satisfaction and goals. Results: Of 1018 pts (median age: 46 years), 67% were female and 48% reported ≥1 comorbidity. 97% (956/987) of pts had received ≥1 treatment for ITP and 12% (126/1018) had a splenectomy; 80% (811/1018) were receiving ITP treatment at survey completion and median time on most recent treatment was 30 weeks (n=608). Corticosteroids and TPO-RAs were the most common first- and second-line treatments prescribed by MDs, respectively (Figure 1); MDs estimated that 15% of their pts have opted against receiving ITP treatment (n=431). 73% of MDs (313/431) considered their pts' perception of medications when making treatment decisions. Pts reported a significant burden due to their current ITP medication: 34% (202/597) experienced daily disruption, 37% (214/576) were bothered by needing to plan meals, and 22% (204/939) had difficulty keeping up with daily medications. 19% (172/897) of pts were dissatisfied with their treatment's platelet level control, and 18% (155/843) disagreed that their medication effectively controlled their symptoms. Pts worried about their treatment's short- (48%; 400/833) and long-term (62%; 527/844) side effects. 38% (162/431) of MDs were neutral toward current ITP treatment options and 9% (37/431) were dissatisfied. Of the 37 dissatisfied MDs, limited options (68%), inability to achieve deep/stable remission (54%), lack of efficacy (43%) and inability to reach treatment goals (43%) were the most common reasons. 32% (327/1016) and 13% (127/1016) of pts believed they were not or did not know if they were in sustained remission, respectively. A treatment offering a sustained remission or cure for ITP was an important goal for 82% (823/1007) of pts. Of treatment goals mentioned in their top 3, the most common were healthy blood counts (56%), improving QoL (44%) and increasing energy levels (43%) for pts (n=1018), and reducing spontaneous bleeds (79%), healthy blood counts (60%) and improving QoL (52%) for MDs (n=431). Limiting time on treatment would substantially affect the treatment preference of 66% (668/1009) of pts. 38% (294/775) of pts did not want to take their ITP treatment for the foreseeable future; however, 67% (680/1013) were concerned about relapse if they stopped. Immunosuppression due to ITP treatment worried 414/787 (53%) pts, with 679/1007 (67%) considering it important when discussing treatment decisions. MDs considered corticosteroids and other immunosuppressants to have the most negative impact on fatigue (Figure 2), yet only 12% (52/431) strongly agreed they aimed to limit immunosuppressive effects. Conclusions: Pts with ITP are dissatisfied with current treatment options because of a perceived lack of efficacy, the burden of short- and long-term side effects, and the need for daily, life-long administration. Additionally, some commonly prescribed treatments for ITP are associated with fatigue. Pts and MDs would like new ITP treatments to offer a sustained period of remission or cure, and pts wish to be able to discontinue them without fear of relapse. There is an unmet need for well-tolerated therapies with disease-modifying potential that can safely achieve these goals.
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How this classification was reachedexpand
Full frame machine prediction
Teacher imitationNot calibrated prevalence, not ground truth. Human validation pending. The Gemma side is a direct model label for every work in the frame, read from the title-only record. The Codex side is a classifier learned from the 10,348 direct Codex labels and calibrated to design-weighted sample rates; fields without enough sample support carry no Codex call. Candidate is the union of the two sides; consensus is their intersection. These outputs are machine_predicted_unvalidated and are not human labels.
Distilled classifier scores by category (both heads)
| Category | Codex | Gemma |
|---|---|---|
| Metaresearch | 0.003 | 0.007 |
| Meta-epidemiology (narrow) | 0.000 | 0.000 |
| Meta-epidemiology (broad) | 0.000 | 0.001 |
| Bibliometrics | 0.000 | 0.001 |
| Science and technology studies | 0.000 | 0.000 |
| Scholarly communication | 0.001 | 0.001 |
| Open science | 0.000 | 0.001 |
| Research integrity | 0.000 | 0.000 |
| Insufficient payload (model declined to judge) | 0.002 | 0.000 |
Machine scores (provisional)
The two teacher heads of the student model, read on this work. A score orders the frame for review; it never asserts a category, and the validation status ships verbatim with every row.
Baseline scores from an immature model (maturity gate not passed, 7 training rounds). Scores rank; they never assert a category.
score_only:v0-immature-baseline · verbatim from the scoring run: score_only means the number may rank works, and no category label ships from itClassification
machine, unvalidatedMachine predicted; a candidate call from one source (direct Gemma or distilled Codex), not a consensus.
How this classification was reached, model by model and score by score, is at the end of the page under "How this classification was reached".