3rd European ATTR Amyloidosis meeting for Patients and Doctors

Abstracts

Poster session 1 – Doctor only

This session will take place on Tuesday, 7 September, from 1:30pm to 2:00pm on the Doctors Channel only.

  • e-P 01 - Category: Disease modifying therapies
    THE EFFECT OF TTR SILENCING IN THE CARDIAC ECM: A TRANSGENIC MICE MODEL STUDY
    Maria JOAO SARAIVA, Portugal; Cristina TEIXEIRA, Helena Sofia MARTINS
     
  • e-P 02 - Category: Diagnosis, prognosis and monitoring of disease course
    PREVALENCE OF MUTATIONS IN THE TTR GENE IN A CIAP POPULATION AND DISTINGUISHING CLINICAL FEATURES
    Janna WARENDORF, The Netherlands; Alexander F.J.E. VRANCKEN, Leonard H. VAN DEN BERG, Nicolette C. NOTERMANS
     
  • e-P 03 - Category: Diagnosis, prognosis and monitoring of disease course
    CHARACTERISTICS, NATURAL HISTORY AND OUTCOME OF EARLY STAGE TRANSTHYRETIN AMYLOID DEPOSITION AND DISEASE
    Steven LAW, United Kingdom; Melanie BEZARD, Aviva PETRIE, Liza CHACKO, Oliver C. COHEN, Sriram RAVICHANDRAN, Olabisi OGUNBIYI, Mounira KHAROUBI, Janet A. GILBERTSON, Dorota ROWCZENIO, Ashutosh WECHALEKAR, Ana MARTINEZ-NAHARRO, Helen J. LACHMANN, Carol J. WHELAN, David HUTT, Philip N. HAWKINS, Thibaud DAMY, Marianna FONTANA and Julian D. GILLMORE
     
  • e-P 04 - Category: Diagnosis, prognosis and monitoring of disease course
    TRACKING DISEASE PROGRESSION IN TRANSTHYRETIN AMYLOID CARDIOMYOPATHY WITH ECHOCARDIOGRAPHY
    Rishi PATEL, United Kingdom; Nina KARIA, Lucia VENNERI, Francesco BANDERA, Liza CHACKO, Beatrice DAL PASSO, Lodovico BUONAMICI, Jonathan LAZARI, , Yousuf RAZVI, James BROWN, Daniel KNIGHT, Ana MARTINEZ-NAHARRO, Carol WHELAN, Candida C. QUARTA, Charlotte MANISTY, James MOON, Dorota ROWCZENIO, Janet A. GILBERTSON, Helen LACHMANN, Ashutosh WECHELAKAR, Aviva PETRIE, William E. MOODY, Richard P. STEEDS, Luciano POTENA, Mattia RIEFOLO, Ornella LEONE, Claudio RAPEZZI, Philip N HAWKINS, Julian D GILLMORE, Marianna FONTANA
     
  • e-P 05 - Category: Disease modifying therapies
    EFFICACY OF INOTERSEN FOR NEUROPATHIC IMPAIRMENT SCORES IN PATIENTS WITH HEREDITARY TRANSTHYRETIN AMYLOIDOSIS WITH POLYNEUROPATHY
    Andrew LOVLEY, USA; Aaron YARLAS, Duncan BROWN, Montserrat VERA-LLONCH, Chafic KARAM, Sami KHELLA
     
  • e-P 06 - Category: Disease modifying therapies
    HELIOS-A: 9-MONTH SUBGROUP ANALYSES AND EXPLORATORY EFFICACY RESULTS FROM THE PHASE 3 STUDY OF VUTRISIRAN IN PATIENTS WITH HEREDITARY TRANSTHYRETIN-MEDIATED AMYLOIDOSIS WITH POLYNEUROPATHY
    Julian GILLMORE, United Kingdom; David ADAMS, Ivailo L. TOURNEV, Mark S. TAYLOR, Teresa COELHO, Violaine PLANTE-BORDENEUVE, John L. BERK, Alejandra GONZALEZ-DUARTE, Soon-Chai LOW, Yoshiki SEKIJIMA, Laura OBICI, Rick BLAKESLEY, Seth ARUM, Rebecca SHILLING, John VEST, Michael POLYDEFKIS
     
  • e-P 07 - Category: Disease modifying therapies
    A HUMAN ANTIBODY SELECTIVE FOR TRANSTHYRETIN AMYLOID REMOVES CARDIAC AMYLOID THROUGH PHAGOCYTIC IMMUNE CELLS
    Aubin MICHALON, Switzerland; Andreas HAGENBUCH, Christian HUY, Evita VARELA, Benoit COMBALUZIER, Thibaud DAMY, Ole B. SUHR, Maria J. SARAIVA, Christoph HOCK, Roger M. NITSCH, Jan GRIMM


Poster session 2

This session will take place on Wednesday, 8 September, from 1:30pm to 2:00pm.

  • e-P 08 - Category: Diagnosis, prognosis and monitoring of disease course
    TRANSTHYRETIN AMYLOIDOSIS QUALITY OF LIFE (ATTR-QOL) QUESTIONNAIRE: DEVELOPMENT OF A NOVEL, ATTR-SPECIFIC PATIENT-REPORTED OUTCOME MEASURE
    Kristen HSU, USA; Meaghan O’CONNOR, Lynne BRODERICK, Kristen L. McCAUSLAND, Duncan BROWN, Kaitlin LaGASSE, Sabrina REBELLO, Michelle K. WHITE, Isabelle LOUSADA
     
  • e-P 09 - Category: Diagnosis, prognosis and monitoring of disease course
    COMPARISON OF SYMPTOMS EXPERIENCED BY PATIENTS WITH ATTRWT AND HATTR: FINDINGS FROM THE DEVELOPMENT OF AN ATTR-SPECIFIC PRO
    Meaghan O’CONNOR, USA; Lynne BRODERICK, Duncan BROWN, Montserrat VERA LLONCH, Kristen HSU, Kristen McCAUSLAND, Michelle WHITE
     
  • e-P 10 - Category: Diagnosis, prognosis and monitoring of disease course
    EXPLORING THE UNMET HEALTHCARE NEEDS OF BRITISH AFRICAN AND AFRO-CARIBBEAN PATIENTS WITH HEREDITARY ATTR AMYLOIDOSIS SECONDARY TO THE V122I VARIANT, THROUGH A QUALITATIVE COMMUNITY ENGAGEMENT STUDY
    Jason DUNGU, United Kingdom; Thirusha LANE, Luis LOPEZ DE HEREDIA, Julian D. GILLMORE
     
  • e-P 11 - Category: Diagnosis, prognosis and monitoring of disease course
    REGRESSION OF BONE-TRACER UPTAKE IN PATIENTS WITH HEREDITARY ATTR AMYLOIDOSIS AFTER ONE YEAR TREATMENT WITH PATISIRAN. AN EARLY MARKER OF TREATMENT RESPONSE?
    Hendrea TINGEN, The Netherlands; J. BIJZET, P. VAN DER MEER, RHJA SLART, BPC HAZENBERG, HLA NIENHUIS
     
  • e-P 12 - Category: Disease modifying therapies
    PATISIRAN IN PATIENTS WITH HATTR AMYLOIDOSIS POST-ORTHOTOPIC LIVER TRANSPLANT: 12-MONTH RESULTS
    Hartmut SCHMIDT, Germany; Jueli Maggie LI, Francisco MUNOZ-BEAMUD, Teresa COELHO, Julian D. GILLMORE, David ADAMS, Anna MAZZEO, Jonas WIXNER, Violaine PLANTE-BORDENEUVE, Laura LLADO, Seth ARUM, Matthew T. WHITE, Patrick Y. JAY
     
  • e-P 13 - Category: Disease modifying therapies
    HELIOS-A: 9-MONTH RESULTS FROM THE PHASE 3 STUDY OF VUTRISIRAN IN PATIENTS WITH HEREDITARY TRANSTHYRETIN-MEDIATED AMYLOIDOSIS WITH POLYNEUROPATHY
    Julian GILLMORE, United Kingdom; David ADAMS, Mark S. TAYLOR, Violaine PLANTE-BORDENEUVE, John L. BERK, Alejandra GONZALEZ-DUARTE, Soon-Chai LOW, Yoshiki SEKIJIMA, Laura OBICI, Rick BLAKESLEY, Seth ARUM, Rebecca SHILLING, John VEST, Michael POLYDEFKIS
     
  • e-P 14 - Category: Diagnosis, prognosis and monitoring of disease course
    EVOLVING CONSENSUS ON THE HOLISTIC CARE FOR PATIENTS WITH HEREDITARY ATTR AMYLOIDOSIS: A MULTIDISCIPLINARY APPROACH,
    Laura OBICI, Italy; Rosaline CALLAGHAN, Joanne ABLETT, Catilena BIBILONI, Teofila BUESER, Agnès FARRUGIA, Fabian KNEBEL, Thirusha LANE, Lars-Ove LARSSON, Agnès MORIER, Vincent NICHOLAS, Teresa COELHO


E-Posters Corner

  • e-P 15 - Category: Diagnosis, prognosis and monitoring of disease course
    TRANSTHYRETIN AMYLOID CARDIOMYOPATHY ASSOCIATED WITH THE P.V142I VARIANT IS AN INHERENTLY AGGRESSIVE FORM OF TRANSTHYRETIN AMYLOID CARDIOMYOPATHY ASSOCIATED WITH SIGNIFICANT FUNCTIONAL IMPAIRMENT AND POOR PROGNOSIS.
    Yousuf RAZVI, Liza CHACKO, Sashiananthan GANESANANTHAN, Rishi K PATEL, Iona BLAKENEY, Nandita KAZA, Steven LAW, Dorota ROWCZENIO, Janet GILBERTSON, David HUTT, Shameem MAHMOOD, Helen J LACHMANN, Ashutosh D WECHALEKAR, Brendan WISNIOWSKI, Carol J WHELAN, Lucia VENNERI, Ana MARTINEZ-NAHARRO, Phillip HAWKINS, Marianna FONTANA, Julian D GILLMORE
     
  • e-P 16 - Category: Diagnosis, prognosis and monitoring of disease course
    THREE NEWLY RECOGNIZED LIKELY PATHOGENIC VARIANTS OF THE TTR GENE CAUSING HEREDITARY TRANSTHYRETIN AMYLOIDOSIS
    Jignesh PATEL, Andrew ROSEN, Adam CHAMBERLIN, Benjamin FELDMANN, Chris ANTOLIK, Heather ZIMMERMANN, Tami JOHNSTON, Arvind NARAYANA
     
  • e-P 17 - Category: Disease modifying therapies
    INOTERSEN ON NEUROPATHY SYMPTOM AND CHANGE SCORES IN HEREDITARY TRANSTHYRETIN AMYLOIDOSIS: SUBGROUPS AND RESPONDER ANALYSES
    Aaron YARLAS, Andrew LOVLEY, Duncan BROWN, Montserrat VERA-LLONCH, Sami KHELLA, Chafic KARAM, P. James B. DYCK
     
  • e-P 18 - Category: Disease modifying therapies
    RESPONDER ANALYSES EXAMINING THE IMPACT OF INOTERSEN ON NEUROPATHIC IMPAIRMENT SCORE IN PATIENTS WITH HEREDITARY TRANSTHYRETIN AMYLOIDOSIS WITH POLYNEUROPATHY
    Aaron YARLAS, Andrew LOVLEY, Duncan BROWN, Montserrat VERA-LLONCH, Sami KHELLA, Chafic KARAM
     
  • e-P 19 - Category: Diagnosis, prognosis and monitoring of disease course
    CARDIOVASCULAR SYMPTOM BURDEN PRIOR TO DIAGNOSIS OF TRANSTHYRETIN AMYLOID CARDIOMYOPATHY AMONG MEDICARE BENEFICIARIES
    Duncan BROWN; Montserrat VERA-LLONCH; Jose Tomas ORTIZ PEREZ; Sheila R. REDDY; Eunice CHANG; Marian H TARBOX; Jose NATIVI-NICOLAU; Nowell M. FINE
     
  • e-P 20 - Category: Diagnosis, prognosis and monitoring of disease course
    DYNAMICS OF NFL IN RESPONSE TO TREATMENT WITH RNAI THERAPEUTICS IN ATTRV AMYLOIDOSIS: RESULTS FROM THE HELIOS-A STUDY
    Simina TICAU, Emre ALDINC, Michael POLYDEFKIS, Hans NIENHUIS, Chafic KARAM, Senda AJROUD-DRISS, Jennifer BARNES, Paul NIOI
     
  • e-P 21 - Category: Disease modifying therapies
    EFFICACY AND SAFETY WITH >3 YEARS OF INOTERSEN TREATMENT FOR THE POLYNEUROPATHY OF HEREDITARY TRANSTHYRETIN AMYLOIDOSIS
    Teresa COELHO, Carol WHELAN, Isabel CONCEIÇÃO, Thomas H. BRANNAGAN, Annabel K. WANG, Michael J. POLYDEFKIS, Peter J. DYCK, Violaine PLANTÉ-BORDENEUVE, John L. BERK, Giampaolo MERLINI, Laura OBICI, Brian DRACHMAN, Peter GOREVIC, Hartmut SCHMIDT, Josep Maria CAMPISTOL PLANA, Josep GAMEZ, Arnt KRISTEN, Anna MAZZEO, Arvind NARAYANA, Kemi OLUGEMO, Peter AQUINO, Merrill D. BENSON, Morie GERTZ
     
  • e-P 22 - Category: Diagnosis, prognosis and monitoring of disease course
    COMPARISON OF PATIENTS WITH MUTATIONS ASSOCIATED WITH HEREDITARY TRANSTHYRETIN AMYLOIDOSIS VERSUS THOSE ASSOCIATED WITH OTHER NEUROMUSCULAR DISEASES
    Sami KHELLA, Urvi DESAI, Meghan TOWNE, Arvind NARAYANA, Kemi OLUGEMO
     
  • e-P 23 - Category: Diagnosis, prognosis and monitoring of disease course
    CLINICAL CHARACTERISTICS OF PATIENTS WITH MUTATIONS ASSOCIATED WITH HEREDITARY TRANSTHYRETIN AMYLOIDOSIS VERSUS THOSE ASSOCIATED WITH OTHER INHERITED CARDIOVASCULAR DISEASES: INSIGHTS FROM A GENETIC TESTING PROGRAMME
    Andrew KELLER, Keyur SHAH, Diego DELGADO, Ajay VALLAKATI, Olakunle AKINBOBOYE, Meghan TOWNE, Arvind NARAYANA, Kemi OLUGEMO
     
  • e-P 24 - Category: Diagnosis, prognosis and monitoring of disease courseEVIDENCE OF POLYNEUROPATHY IN PATIENTS WITH HATTR AMYLOIDOSIS WITH V122I/T60A VARIANTS IN THE PATISIRAN PHASE 4 OBSERVATIONAL STUDY
    Saurabh MALHOTRA, Francy SHU, Ronald ZOLTY, Yessar HUSSAIN, Cecilia HALE, Kelley CAPOCELLI
     
  • e-P 25 - Category: Disease modifying therapies
    REASON FOR STOPPING TRANSTHYRETIN STABILIZERS PRIOR TO HELIOS-A AND THE IMPACT OF PRIOR STABILIZER USE ON THE EFFICACY OF VUTRISIRAN IN PATIENTS WITH HEREDITARY TRANSTHYRETIN-MEDIATED AMYLOIDOSIS WITH POLYNEUROPATHY
    John BERK, Violaine PLANTÉ-BORDENEUVE, Soon Chai LOW, Jonas WIXNER, Tony CHEN, Madeline MERKEL, Seth ARUM, Rebecca SHILLING, David ADAMS
     
  • e-P 26 - Category: Diagnosis, prognosis and monitoring of disease course
    THE PATIENT JOURNEY PRIOR TO DIAGNOSIS OF HEREDITARY TRANSTHYRETIN AMYLOIDOSIS
    Montserrat VERA-LLONCH, Sheila Reiss REDDY, Eunice CHANG, Marian TARBOX, Michael POLLOCK
     
  • e-P 27 - Category: Disease modifying therapies
    TREATMENT SATISFACTION FOR GENE SILENCING PHARMACOTHERAPIES IN HEREDITARY TRANSTHYRETIN AMYLOIDOSIS WITH POLYNEUROPATHY
    Asia Sikora KESSLER, Duncan BROWN, Montserrat VERA LLONCH, Aaron YARLAS, Kristen McCAUSLAND
     
  • e-P 28 - Category: Diagnosis, prognosis and monitoring of disease course
    PATIENT-REPORTED OUTCOMES BY TIME FROM SYMPTOM ONSET TO FIRST PHARMACOTHERAPY AMONG TRANSTHYRETIN AMYLOIDOSIS PATIENTS
    Asia Sikora KESSLER, Duncan BROWN, Montserrat VERA-LLONCH, Aaron YARLAS, Kristen McCAUSLAND
     
  • e-P 29 - Category: Diagnosis, prognosis and monitoring of disease course
    CAREGIVING BURDEN AMONG CAREGIVERS OF PATIENTS WITH ATTR AMYLOIDOSIS
    Duncan BROWN, Kristen McCAUSLAND, Aaron YARLAS, Asia Sikora KESSLER, Montserrat VERA-LLONCH
     
  • e-P 30 - Category: Disease modifying therapies
    IMPACT OF ATTRv AMYLOIDOSIS ON DAILY LIFE FROM THE HELIOS-A STUDY: EVALUATING PATIENTS’ ABILITY TO WORK, DISABILITY STATUS, AND NEED FOR CAREGIVER ASSISTANCE
    Jonas WIXNER, Anna MAZZEO, Hans NIENHUIS, Kon-Ping LIN, Cecilia HALE, Madeline MERKEL, Rebecca SHILLING, Laura OBICI
     
  • e-P 31 - Category: Disease modifying therapies
    EVALUATION OF THE EFFICACY AND SAFETY OF EPLONTERSEN IN PATIENTS WITH TRANSTHYRETIN-MEDIATED AMYLOID CARDIOMYOPATHY: THE CARDIO-TTRANSFORM STUDY
    Mathew MAURER, Arnt KRISTEN, Rodney FALK, Merrill D. BENSON, Gustavo BUCHELE, Michela BRAMBATTI, Sam TSIMIKAS, Nicholas VINEY, Li TAI, Cecilia MONTEIRO, Qingqing YANG, Eugene SCHNEIDER, Richard GEARY, Brett MONIA