Author Listing - Lead/ Submitting Author (we will be communicating with this individual): Name, Institution, City and State where the work was done. Co-authors will be added in a separate section.
Márcia Waddington Cruz, CEPARM, Amyloidosis Center, University Hospital, Federal University of Rio de Janeiro, Rio de Janeiro, Brazil

Main Body of the Abstract
Introduction: In the NEURO-TTRansform Phase 3 trial (NCT04136184), eplontersen significantly reduced neuropathy impairment and improved quality of life versus historical placebo in patients with hereditary transthyretin amyloidosis with polyneuropathy (ATTRv-PN). To determine the impact of sex, this exploratory analysis evaluated the treatment effect of eplontersen in male and female patients.
Methods: Changes from baseline to Week 66 in modified Neuropathy Impairment Score +7 (mNIS+7) composite score (range, –22.3 to 346.3) and Norfolk Quality of Life-Diabetic Neuropathy (Norfolk QoL-DN) total score (range, –4 to 136) were measured, with higher scores indicating worsening neuropathy and poorer quality of life.
Results: Overall, 100 male and 44 female patients were randomized to receive eplontersen in the NEURO-TTRansform trial. Sixty patients (41 male, 19 female) from NEURO-TTR (NCT01737398) formed a historical placebo group. At baseline, in eplontersen versus placebo groups, respectively, male patients’ mean age was 51.7 versus 59.2 years, mean mNIS+7 composite score was 83.2 versus 78.5, and mean Norfolk QoL-DN total score was 44.7 versus 49.3; female patients’ mean age was 56.1 versus 60.3 years, mean mNIS+7 composite score was 76.9 versus 66.6, and mean Norfolk QoL-DN total score was 42.5 versus 47.4. Mean changes from baseline to Week 66 were lower (better) with eplontersen versus placebo for mNIS+7 composite score and Norfolk QoL-DN total score in both male and female patients (Figure).
Conclusions: Regardless of sex, eplontersen reduced neuropathy impairment and improved quality of life versus historical placebo in patients with ATTRv-PN.

**Author Listing - Lead/ Submitting Author (we will be communicating with this individual): Name, Institution, City and State where the work was done. Co-authors will be added in a separate section.**
Márcia Waddington Cruz, CEPARM, Amyloidosis Center, University Hospital, Federal University of Rio de Janeiro, Rio de Janeiro, Brazil

**Main Body of the Abstract**
**Introduction:** In the NEURO-TTRansform Phase 3 trial (NCT04136184), eplontersen significantly reduced neuropathy impairment and improved quality of life versus historical placebo in patients with hereditary transthyretin amyloidosis with polyneuropathy (ATTRv-PN). To determine the impact of sex, this exploratory analysis evaluated the treatment effect of eplontersen in male and female patients.
**Methods:** Changes from baseline to Week 66 in modified Neuropathy Impairment Score +7 (mNIS+7) composite score (range, –22.3 to 346.3) and Norfolk Quality of Life-Diabetic Neuropathy (Norfolk QoL-DN) total score (range, –4 to 136) were measured, with higher scores indicating worsening neuropathy and poorer quality of life.
**Results:** Overall, 100 male and 44 female patients were randomized to receive eplontersen in the NEURO-TTRansform trial. Sixty patients (41 male, 19 female) from NEURO-TTR (NCT01737398) formed a historical placebo group. At baseline, in eplontersen versus placebo groups, respectively, male patients’ mean age was 51.7 versus 59.2 years, mean mNIS+7 composite score was 83.2 versus 78.5, and mean Norfolk QoL-DN total score was 44.7 versus 49.3; female patients’ mean age was 56.1 versus 60.3 years, mean mNIS+7 composite score was 76.9 versus 66.6, and mean Norfolk QoL-DN total score was 42.5 versus 47.4. Mean changes from baseline to Week 66 were lower (better) with eplontersen versus placebo for mNIS+7 composite score and Norfolk QoL-DN total score in both male and female patients (**Figure**).
**Conclusions:** Regardless of sex, eplontersen reduced neuropathy impairment and improved quality of life versus historical placebo in patients with ATTRv-PN.

Eplontersen for Hereditary Transthyretin Amyloidosis With Polyneuropathy: An Exploratory Analysis of Treatment Effect in Male and Female Patients PC112 (#202)

We invite you to attend the XIX International Symposium on Amyloidosis to be held in Rochester, MN.

This symposium is organized as a hybrid meeting (with the focus on in-person) held from Sunday, May 26 through Thursday, May 30, 2024.

Together with the ISA Board and members of Mayo Clinic, a very attractive scientific hybrid program is being planned. The program will cover a variety of types of systemic amyloidosis: ATTR (hereditary and wild-type), AL (light-chain), AA and less frequent forms. There will be key lectures, specific symposia including those company sponsored, oral presentations and poster sessions of accepted abstracts.

COURSE DESCRIPTION
This program will provide a comprehensive review of state-of-the-art education in systemic amyloidosis: basic science, pathophysiology, diagnosis, current and emerging treatments. Scientific presentations will include competitive oral and poster abstracts. The rapid evolution of scientific discovery and expanding treatment options has created tremendous interest in the field and this meeting will fill and important gap in educating scientists and clinicians.