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EARLY USE OF ELTROMBOPAG IN ADULT PATIENTS WITH ITP WHO ARE REFRACTORY OR RELAPSED AFTER FIRST-LINE CORTICOSTEROID THERAPY – AN AD HOC ANALYSIS OF THE TAPER TRIAL RESULTS
Author(s): ,
Waleed Ghanima
Affiliations:
Department of Medicine,Østfold Hospital Trust,Kalnes,Norvège;Department of Medicine,Østfold Hospital Trust,Kalnes,Norwegen;Department of Medicine,Østfold Hospital Trust,Kalnes,Norvegia;Department of Medicine,Østfold Hospital Trust,Kalnes,Norway;Department of Medicine,Østfold Hospital Trust,Kalnes,Noruega;Department of Medicine,Østfold Hospital Trust,Kalnes,Noorwegen;Department of Medicine,Østfold
,
Francesco Zaja
Affiliations:
Department of Medical, Surgical and Health Sciences,University of Trieste,Trieste,Italie;Department of Medical, Surgical and Health Sciences,University of Trieste,Trieste,Italien;Department of Medical, Surgical and Health Sciences,University of Trieste,Trieste,Italia;Department of Medical, Surgical and Health Sciences,University of Trieste,Trieste,Italy;Department of Medical, Surgical and Health S
,
Joan Maier
Affiliations:
Novartis Pharma AG,Basel,Suisse;Novartis Pharma AG,Basel,Schweiz;Novartis Pharma AG,Basel,Svizzera;Novartis Pharma AG,Basel,Switzerland;Novartis Pharma AG,Basel,Suiza;Novartis Pharma AG,Basel,Zwitserland;Novartis Pharma AG,Basel,Suíça;Novartis Pharma AG,Basel,Швейцария ;Novartis Pharma AG,Basel,Schweiz
,
Jens Haenig
Affiliations:
Novartis Pharma AG,Basel,Suisse;Novartis Pharma AG,Basel,Schweiz;Novartis Pharma AG,Basel,Svizzera;Novartis Pharma AG,Basel,Switzerland;Novartis Pharma AG,Basel,Suiza;Novartis Pharma AG,Basel,Zwitserland;Novartis Pharma AG,Basel,Suíça;Novartis Pharma AG,Basel,Швейцария ;Novartis Pharma AG,Basel,Schweiz
,
James Lee
Affiliations:
Novartis Pharmaceuticals Corporation,East Hanover, NJ,États-unis;Novartis Pharmaceuticals Corporation,East Hanover, NJ,Vereinigte Staaten;Novartis Pharmaceuticals Corporation,East Hanover, NJ,Stati Uniti;Novartis Pharmaceuticals Corporation,East Hanover, NJ,United States;Novartis Pharmaceuticals Corporation,East Hanover, NJ,Estados Unidos;Novartis Pharmaceuticals Corporation,East Hanover, NJ,Veren
Nichola Cooper
Affiliations:
Centre for Haematology, Department of Immunology and Inflammation,Imperial College London, Hammersmith Hospital,London,Royaume-uni;Centre for Haematology, Department of Immunology and Inflammation,Imperial College London, Hammersmith Hospital,London,Vereinigtes Königreich;Centre for Haematology, Department of Immunology and Inflammation,Imperial College London, Hammersmith Hospital,London,Regno Un
(Abstract release date: 05/12/22) EHA Library. Ghanima W. 06/10/22; 358495; P1638
Prof. Waleed Ghanima
Prof. Waleed Ghanima
Contributions
Abstract
Presentation during EHA2022: All (e)Poster presentations will be made available as of Friday, June 10, 2022 (09:00 CEST) and will be accessible for on-demand viewing until Monday, August 15, 2022 on the Congress platform.

Abstract: P1638

Type: Poster presentation

Session title: Platelet disorders

Background

Immune thrombocytopenia (ITP) is an autoimmune disorder characterized by reduced platelet count (<100×109/L) and increased bleeding risk in the absence of other causes. It is categorized by disease duration from diagnosis: newly diagnosed (ndITP; ≤3 months [mo]), persistent (pITP; 3-12 mo), and chronic (cITP; >12 mo). Eltrombopag (EPAG), a thrombopoietin receptor agonist, is approved in Europe for the treatment of ITP lasting ≥6 mo in patients (pts) refractory to other treatments. The TAPER trial (NCT03524612) was designed to assess sustained response off treatment following EPAG tapering in adults with ITP of any duration, who were refractory/had relapsed after first-line corticosteroids.

Aims

To conduct an ad hoc analysis of the ongoing TAPER trial, assessing initial response to EPAG therapy according to time since ITP diagnosis (ndITP, pITP, cITP).

Methods

Pts with ≥6 mo of data, or pts who discontinued the study prior to Month 6 were included. Newly enrolled pts with <6 mo of data were excluded. Pts were grouped according to time since ITP diagnosis at baseline (<3, 3 to <6, 6 to ≤12, and >12 mo). The main analysis of hematologic response (ie, percentage of pts with platelet count thresholds of ≥30, ≥50, and ≥100 ×109/L achieved at least once without rescue therapy) was restricted to data from the first 2 mo (~Day 57) to ensure pts were on treatment. Safety analyses were restricted up to and including Month 6.

Results

A total of 82/105 (78%) pts from TAPER were included. Median (range) age was 49 (18-88) years and 58.5% were female. In the first 6 months, the median (interquartile range [IQR]) duration of EPAG exposure was 5.3 (2.6-6.0) mo, and median (IQR) dose was 53.2 (37.5-69.6) mg/day. The proportions of pts with platelet counts equal to or above the specified thresholds were comparable across the time-since-ITP-diagnosis groups, ranging from 69% to 100% (Fig. 1). The median time to a platelet count of ≥50×109/L was also comparable across groups, ranging from 8 to 17 days, and the median (IQR) increase in platelet count to ~Day 57 was 84.5×109/L (36.5-165.6). The percentages of pts without World Health Organization Grade 1-4 bleeding events increased from Day 1 to ~Day 57 across all groups (<3 mo: 39.5%→84%; 3 to <6 mo: 61%→100%; 6 to ≤12 mo: 50%→88%; >12 mo: 70%→89%). The most common all-grade adverse events (AEs) were headache (pts: 18% [<3 mo], 22% [3 to <6 mo], 12.5% [6 to ≤12 mo], 10% [>12 mo]) and thrombocytopenia (pts: 21% [<3 mo], 17% [3 to <6 mo], 12.5% [6 to ≤12 mo], 0% [>12 mo]). One pt (6%; 3 to <6 mo) had grade ≥3 headache; 5 (13%; <3 mo), 2 (11%; 3 to <6 mo), and 2 (12.5%; 6 to ≤12 mo) pts had grade ≥3 thrombocytopenia. There were 3 treatment-related severe AEs (intentional product misuse [<3 mo], infectious diarrhea secondary to EPAG, and pulmonary embolism [both 6 to ≤12 mo]). Two deaths were reported by the cut-off date (central nervous system hemorrhage [n=1; <3 mo]; intracranial hemorrhage [n=1; 3 to <6 mo]) that were not considered study drug related.

Conclusion

The data show that EPAG achieved comparable rates of platelet response and reduced the frequency and severity of bleeding events in pts with ndITP, pITP, and cITP. Safety findings were comparable across the 4 groups, and aligned with the well-established safety profile of EPAG. These findings suggest that EPAG is as efficacious and well tolerated in pts who receive it early following diagnosis as in those who receive it later.

Keyword(s): Corticosteroids, ITP, Thrombocytopenia, Thrombopoietin (TPO)

Presentation during EHA2022: All (e)Poster presentations will be made available as of Friday, June 10, 2022 (09:00 CEST) and will be accessible for on-demand viewing until Monday, August 15, 2022 on the Congress platform.

Abstract: P1638

Type: Poster presentation

Session title: Platelet disorders

Background

Immune thrombocytopenia (ITP) is an autoimmune disorder characterized by reduced platelet count (<100×109/L) and increased bleeding risk in the absence of other causes. It is categorized by disease duration from diagnosis: newly diagnosed (ndITP; ≤3 months [mo]), persistent (pITP; 3-12 mo), and chronic (cITP; >12 mo). Eltrombopag (EPAG), a thrombopoietin receptor agonist, is approved in Europe for the treatment of ITP lasting ≥6 mo in patients (pts) refractory to other treatments. The TAPER trial (NCT03524612) was designed to assess sustained response off treatment following EPAG tapering in adults with ITP of any duration, who were refractory/had relapsed after first-line corticosteroids.

Aims

To conduct an ad hoc analysis of the ongoing TAPER trial, assessing initial response to EPAG therapy according to time since ITP diagnosis (ndITP, pITP, cITP).

Methods

Pts with ≥6 mo of data, or pts who discontinued the study prior to Month 6 were included. Newly enrolled pts with <6 mo of data were excluded. Pts were grouped according to time since ITP diagnosis at baseline (<3, 3 to <6, 6 to ≤12, and >12 mo). The main analysis of hematologic response (ie, percentage of pts with platelet count thresholds of ≥30, ≥50, and ≥100 ×109/L achieved at least once without rescue therapy) was restricted to data from the first 2 mo (~Day 57) to ensure pts were on treatment. Safety analyses were restricted up to and including Month 6.

Results

A total of 82/105 (78%) pts from TAPER were included. Median (range) age was 49 (18-88) years and 58.5% were female. In the first 6 months, the median (interquartile range [IQR]) duration of EPAG exposure was 5.3 (2.6-6.0) mo, and median (IQR) dose was 53.2 (37.5-69.6) mg/day. The proportions of pts with platelet counts equal to or above the specified thresholds were comparable across the time-since-ITP-diagnosis groups, ranging from 69% to 100% (Fig. 1). The median time to a platelet count of ≥50×109/L was also comparable across groups, ranging from 8 to 17 days, and the median (IQR) increase in platelet count to ~Day 57 was 84.5×109/L (36.5-165.6). The percentages of pts without World Health Organization Grade 1-4 bleeding events increased from Day 1 to ~Day 57 across all groups (<3 mo: 39.5%→84%; 3 to <6 mo: 61%→100%; 6 to ≤12 mo: 50%→88%; >12 mo: 70%→89%). The most common all-grade adverse events (AEs) were headache (pts: 18% [<3 mo], 22% [3 to <6 mo], 12.5% [6 to ≤12 mo], 10% [>12 mo]) and thrombocytopenia (pts: 21% [<3 mo], 17% [3 to <6 mo], 12.5% [6 to ≤12 mo], 0% [>12 mo]). One pt (6%; 3 to <6 mo) had grade ≥3 headache; 5 (13%; <3 mo), 2 (11%; 3 to <6 mo), and 2 (12.5%; 6 to ≤12 mo) pts had grade ≥3 thrombocytopenia. There were 3 treatment-related severe AEs (intentional product misuse [<3 mo], infectious diarrhea secondary to EPAG, and pulmonary embolism [both 6 to ≤12 mo]). Two deaths were reported by the cut-off date (central nervous system hemorrhage [n=1; <3 mo]; intracranial hemorrhage [n=1; 3 to <6 mo]) that were not considered study drug related.

Conclusion

The data show that EPAG achieved comparable rates of platelet response and reduced the frequency and severity of bleeding events in pts with ndITP, pITP, and cITP. Safety findings were comparable across the 4 groups, and aligned with the well-established safety profile of EPAG. These findings suggest that EPAG is as efficacious and well tolerated in pts who receive it early following diagnosis as in those who receive it later.

Keyword(s): Corticosteroids, ITP, Thrombocytopenia, Thrombopoietin (TPO)

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