**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.**
Jonas Wixner, Department of Public Health and Clinical Medicine, Umeå University, Umeå, Sweden

**Main Body of the Abstract**
Introduction: Eplontersen improved neuropathy impairment and quality of life (QoL) versus historical placebo (placebo) in patients with hereditary transthyretin amyloidosis with polyneuropathy in the NEURO-TTRansform trial. Whether these benefits were observed in patients with worsening nutritional status (modified body mass index [mBMI]) is unknown, and is the subject of this secondary analysis.
Methods: Patients from NEURO-TTRansform were categorized by treatment, by change from baseline in mBMI at Week 66 (>10% decrease; <2.5 to 10% decrease; -2.5 to +2.5% change; >2.5 to 10%; >10% increase). Change in modified Neuropathy Impairment Score+7 (mNIS+7, composite score), and Norfolk QoL-Diabetic Neuropathy (Norfolk QoL-DN) total score was evaluated.
Results: Patients (eplontersen n=144; placebo n=60) were of mean (SD) age, 54.9 (15.0) years, 69% were male. Overall distribution of mBMI trended towards improvement for eplontersen and worsening for placebo. Across mBMI categories, eplontersen demonstrated consistent benefit on neuropathy impairment and QoL versus placebo (Figure). Mean (95% CI) differences (Week 66) in mNIS+7 scores for eplontersen versus placebo were -29.5 ([-45.5, -13.5]; mBMI >10% decrease), -17.8 ([-30.1, -5.5]; <2.5 to 10% decrease), -17.1 ([-30.8, -3.4]; -2.5 to +2.5% change), and -8.8 ([-22.9, 5.3]; >2.5 to 10% increase). For Norfolk-QoL-DN; -24.3 ([-38.4, -10.2]; mBMI >10% decrease), -14.9 ([-29.1, -0.8]; <2.5 to 10% decrease), -15.6 ([-30.8, -0.5]; -2.5 to +2.5 % change), and -2.1 ([-13.5, 9.2]; >2.5 to 10% increase).
Conclusions: Benefits in neuropathy impairment and QoL were observed with eplontersen versus placebo, regardless of degree of mBMI change from baseline, including in patients with worsening nutritional status.



**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.**
Jonas Wixner, [Department of Public Health and Clinical Medicine](https://www.umu.se/institutionen-for-folkhalsa-och-klinisk-medicin/), Umeå University, Umeå, Sweden

**Main Body of the Abstract**
**Introduction:** Eplontersen improved neuropathy impairment and quality of life (QoL) versus historical placebo (placebo) in patients with hereditary transthyretin amyloidosis with polyneuropathy in the NEURO-TTRansform trial. Whether these benefits were observed in patients with worsening nutritional status (modified body mass index [mBMI]) is unknown, and is the subject of this secondary analysis.
**Methods:** Patients from NEURO-TTRansform were categorized by treatment, by change from baseline in mBMI at Week 66 (>10% decrease; <2.5 to 10% decrease; -2.5 to +2.5% change; >2.5 to 10%; >10% increase). Change in modified Neuropathy Impairment Score+7 (mNIS+7, composite score), and Norfolk QoL-Diabetic Neuropathy (Norfolk QoL-DN) total score was evaluated.
**Results:** Patients (eplontersen n=144; placebo n=60) were of mean (SD) age, 54.9 (15.0) years, 69% were male. Overall distribution of mBMI trended towards improvement for eplontersen and worsening for placebo. Across mBMI categories, eplontersen demonstrated consistent benefit on neuropathy impairment and QoL versus placebo (**Figure**). Mean (95% CI) differences (Week 66) in mNIS+7 scores for eplontersen versus placebo were -29.5 ([-45.5, -13.5]; mBMI >10% decrease), -17.8 ([-30.1, -5.5]; <2.5 to 10% decrease), -17.1 ([-30.8, -3.4]; -2.5 to +2.5% change), and -8.8 ([-22.9, 5.3]; >2.5 to 10% increase). For Norfolk-QoL-DN; -24.3 ([-38.4, -10.2]; mBMI >10% decrease), -14.9 ([-29.1, -0.8]; <2.5 to 10% decrease), -15.6 ([-30.8, -0.5]; -2.5 to +2.5 % change), and -2.1 ([-13.5, 9.2]; >2.5 to 10% increase).
**Conclusions:** Benefits in neuropathy impairment and QoL were observed with eplontersen versus placebo, regardless of degree of mBMI change from baseline, including in patients with worsening nutritional status.



Neuropathy Impairment and Nutritional Status with Eplontersen in Patients with Hereditary Transthyretin-Mediated Amyloidosis PC106 (#174)

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.