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Journal Articles

Sky-1214, a Small Molecule Splicing Modulator Targeting FANCL and Fanci, Provides a New Mechanism of Action Targeting Multiple Myeloma and Non-Hodgkin's Lymphoma

Simone Rauch, Stefan Reber, Montserrat Perez-Salvia, Marco Pregnolato, Botao Liu, Loren Berry, Olesia Buiakova, Hasane Ratni, Brian Gudenas, Carlo Cusulin, Lauren Shanahan, Douglas V Faller, Veronica Costa, Sergey Paushkin

Blood (2024) 144 (Supplement 1): 2784.

https://doi.org/10.1182/blood-2024-209068

Published: 2024

Journal Articles

Iadademstat and Gilteritinib for the Treatment of FLT3-Mutated Relapsed/Refractory Acute Myeloid Leukemia: The Frida Study

Amir T. Fathi, Theodore P Braun, Alexander Joseph Ambinder, Gautam Borthakur, Robert L. Redner, Mabel Arevalo, Sonia Gutierrez, Ana Limon, Douglas V. Faller

Blood (2023) 142 (Supplement 1): 5974.

https://doi.org/10.1182/blood-2023-189291

Published: 2023

Journal Articles

Iadademstat Combination with Azacitidine Is a Safe and Effective Treatment in First Line Acute Myeloid Leukemia. Final Results of the Alice Trial

Olga Salamero, Tim C.P Somervaille, Antonieta Molero, Evelyn Acuña-Cruz, Jose A. Perez-Simon, Rosa Coll, Montserrat Arnan, Brayan Merchan, Ana Perez, Isabel Cano, Rebeca Rodríguez-Veiga, Mabel Arevalo, Sonia Gutierrez, Carlos Buesa, Douglas V Faller, Francesc Bosch, Pau Montesinos

Blood (2022) 140 (Supplement 1): 1711–1713.

https://doi.org/10.1182/blood-2022-168945

Published: 2022

Journal Articles

The Frida Study: An Option for Mutated FLT3 Relapsed/Refractory Acute Myeloid Leukemia Patients with a Novel Iadademstat and Gilteritinib Combination Therapy

Amir T. Fathi, Mabel Arevalo, Sonia Gutierrez, Antonieta Molero, Natalia Sacilotto, Ana Limon, Douglas V Faller

Blood (2022) 140 (Supplement 1): 11755–11756.

https://doi.org/10.1182/blood-2022-160427

Published: 2022

Journal Articles

Health-Related Quality of Life and Symptoms Among Patients with Relapsed or Refractory AL Amyloidosis Treated with Ixazomib-Dexamethasone Versus Physician's Choice: Results from a Randomized Phase 3 Trial

Vaishali Sanchorawala, Ashutosh D. Wechalekar, Kihyun Kim, Stefan Schönland, Heather J Landau, Kwok Fiona, Kenshi Suzuki, Angela Dispenzieri, Giampaolo Merlini, Raymond Comenzo, Dasha Cherepanov, Vanessa C Hayden, Arun Kumar, Richard Labotka, Douglas V. Faller, Efstathios Kastritis

Blood (2021) 138 (Supplement 1): 4771.

https://doi.org/10.1182/blood-2021-148689

Published: 2021

Journal Articles

Pevonedistat (PEV) + Azacitidine (AZA) Versus AZA Alone As First-Line Treatment for Patients with Higher-Risk Myelodysplastic Syndromes (MDS)/Chronic Myelomonocytic Leukemia (CMML) or Acute Myeloid Leukemia (AML) with 20-30% Marrow Blasts: The Randomized Phase 3 PANTHER Trial (NCT03268954)

Mikkael A. Sekeres, Larisa Girshova, Vadim A Doronin, Maria Diez-Campelo, David Valcarcel, Suman Kambhampati, Nora-Athina Viniou, Dariusz Woszczyk, Raquel De Paz Arias, Argiris Symeonidis, Achilles Anagnostopoulos, Eduardo Ciliao Munhoz, Uwe Platzbecker, Valeria Santini, Robert J. Fram, Ying Yuan, Douglas V. Faller, Lionel Ades

Blood (2021) 138 (Supplement 1): 242.

https://doi.org/10.1182/blood-2021-146154

Published: 2021

Journal Articles

Randomized Phase 2 Trial of Pevonedistat Plus Azacitidine Versus Azacitidine in Higher-Risk Myelodysplastic Syndromes/Chronic Myelomonocytic Leukemia or Low-Blast Acute Myeloid Leukemia: Exploratory Analysis of Patient-Reported Outcomes

Joshua F. Zeidner, MD, Flora Mazerolle, MSc, Jill A. Bell, PhD MS, Lauren E. Cain, MHS, ScM, PhD, Douglas V. Faller, Mehul Dalal, PhD, Antoine Regnault, PhD, Robert J. Fram

Blood (2020) 136 (Supplement 1): 39–40.

https://doi.org/10.1182/blood-2020-136935

Published: 2020

Journal Articles

Oral Nanatinostat (Nstat) and Valganciclovir (VGCV) in Patients with Recurrent Epstein-Barr Virus (EBV)-Positive Lymphomas: Initial Phase 2 Results

Pierluigi Porcu, MD, Bradley Haverkos, MD MPH, MS, Onder Alpdogan, MD, Robert Baiocchi, MD PhD, Jonathan E Brammer, MD, Tatyana A. Feldman, MD, Marcelo Capra, MD PhD, Elizabeth A Brem, MD, Phillip Scheinberg, MD, Juliana Pereira, MD PhD, Leyla Shune, MD, Afton Katkov, MSc, Robert McRae, Lisa Rojkjaer, MD, Ivor Royston, MD, Douglas V. Faller, MD PhD

Blood (2020) 136 (Supplement 1): 7–8.

https://doi.org/10.1182/blood-2020-140843

Published: 2020

Journal Articles

A Randomized Phase 2 Study of Pevonedistat, Venetoclax, and Azacitidine Versus Venetoclax Plus Azacitidine in Adults with Newly Diagnosed Acute Myeloid Leukemia (AML) Who Are Unfit for Intensive Chemotherapy

Nicholas J. Short, MD, Farhad Sedarati, Dan Zhao, Olga Tsukurov, Sharon Friedlander, Douglas V. Faller

Blood (2020) 136 (Supplement 1): 34–35.

https://doi.org/10.1182/blood-2020-136968

Published: 2020

Journal Articles

Combination of Oral Nanatinostat (Nstat), a Novel Histone Deacetylase Inhibitor (HDACi), and the Oral Anti-Viral, Valganciclovir (VGCV), Is Active in Relapsed/Refractory (R/R) Epstein-Barr Virus (EBV)-Positive B-Cell, T-Cell, and Hodgkin Lymphoma: Interim Safety and Efficacy Results from a Phase 1b/2a Study

Blood (2019) 134 (Supplement_1): 465.

https://doi.org/10.1182/blood-2019-132004

Published: 2019

Journal Articles

Primary Results from the Phase 3 Tourmaline-AL1 Trial of Ixazomib-Dexamethasone Versus Physician's Choice of Therapy in Patients (Pts) with Relapsed/Refractory Primary Systemic AL Amyloidosis (RRAL)

Angela Dispenzieri, MD, Eftathios Kastritis, Ashutosh D Wechalekar, MB BS, MD FRCP, FRCPath, DM, Stefan O. Schönland, MD, Kihyun Kim, MD, Vaishali Sanchorawala, MD, Heather J Landau, MD, Fiona Kwok, FRACP,FRCPA,MBBS, Kenshi Suzuki, MD PhD, Raymond L Comenzo, MD, Deborah Berg, RN, MSN, Guohui Liu, PhD, Douglas V. Faller, MD PhD, Giampaolo Merlini, MD

Blood (2019) 134 (Supplement_1): 139.

https://doi.org/10.1182/blood-2019-124409

Published: 2019

Journal Articles

Patient-Centered Evaluation of Clinical Benefit in Acute Myeloid Leukemia: Importance of Early Engagement with Patients

Jill A. Bell, PhD MS, Farrah Anne Pompilus, MA, Alissa Rams, BA, Yanyan Zhu, PhD MPH, Anna Ciesluk, MPH, Rafael Bejar, MD PhD, Robert J. Fram, MD, Douglas V. Faller, MD PhD, Patrick Marquis, MD MBA

Blood (2019) 134 (Supplement_1): 5898.

https://doi.org/10.1182/blood-2019-128517

Published: 2019

Journal Articles

Assessing Patient-Reported Outcomes in People with Myelodysplastic Syndromes: Can a Customized Selection of Items from the EORTC Library Enhance the EORTC QLQ-C30?

Jill A Bell, PhD, Farrah A Pompilus, Anna Christian, MPH, Flora Mazerolle, MSc, Fatima Scipione, Rafael Bejar, MD PhD, Aaron Galaznik, PhD, Robert J. Fram, MD, Douglas V. Faller, MD PhD, Stefan Cano, PhD CPsychol, AFBPsS, Antoine Regnault, PhD

Blood (2018) 132 (Supplement 1): 4856.

https://doi.org/10.1182/blood-2018-99-116977

Published: 2018

Journal Articles

Pevonedistat, a first-in-class NEDD8-activating enzyme inhibitor, combined with azacitidine in patients with AML

Ronan T. Swords, Steven Coutre, Michael B. Maris, Joshua F. Zeidner, James M. Foran, Jose Cruz, Harry P. Erba, Jesus G. Berdeja, Wayne Tam, Saran Vardhanabhuti, Iwona Pawlikowska-Dobler, Hélène M. Faessel, Ajeeta B. Dash, Farhad Sedarati, Bruce J. Dezube, Douglas V. Faller, Michael R. Savona

Blood (2018) 131 (13): 1415–1424.

https://doi.org/10.1182/blood-2017-09-805895

Published: 2018

Includes: Supplemental data

Journal Articles

Patient Perspectives on Symptom Experience and Impact on Health-Related Quality of Life in Higher-Risk Myelodysplastic Syndromes, Chronic Myelomonocytic Leukemia and Low-Blast Acute Myeloid Leukemia

Jill A Bell, PhD, Aaron Galaznik, MD, Farrah A Pompilus, Sara Strzok, Robert J. Fram, MD, Douglas V. Faller, MD PhD, Patrick Marquis

Blood (2017) 130 (Supplement 1): 3458.

https://doi.org/10.1182/blood.V130.Suppl_1.3458.3458

Published: 2017

Journal Articles

Healthcare Costs in Elderly Patients with Acute Myeloid Leukemia (AML) in Routine Clinical Care in the United States (US)

Jill A Bell, PhD, Aaron Galaznik, MD, Sharanya Murty, PhD, Megan Pollack, PharmD, Augustina Ogbonnaya, MPH, Michael T. Eaddy, PharmD, PhD, Robert J. Fram, MD, Douglas V. Faller, MD PhD, Vamsi Kota, MBBS

Blood (2017) 130 (Supplement 1): 3459.

https://doi.org/10.1182/blood.V130.Suppl_1.3459.3459

Published: 2017

Journal Articles

Enhancing the Content of the EORTC QLQ-C30 to Maximize the Detection of Treatment Benefit in Higher-Risk Myelodysplastic Syndromes, Chronic Myelomonocytic Leukemia, and Low-Blast Acute Myeloid Leukemia Using the EORTC Item Bank

Jill A Bell, PhD, Aaron Galaznik, MD, Farrah A Pompilus, Sara Strzok, Rafael Bejar, MD PhD, Robert J. Fram, MD, Douglas V. Faller, MD PhD, Patrick Marquis

Blood (2017) 130 (Supplement 1): 3457.

https://doi.org/10.1182/blood.V130.Suppl_1.3457.3457

Published: 2017

Journal Articles

Treatment Characteristics and Outcomes for Newly Diagnosed Elderly Patients with Acute Myeloid Leukemia (AML) Treated in the United States with Either 7+3 or a Hypomethylating Agent (HMA)

Jill A Bell, PhD, Aaron Galaznik, MD, Eileen Farrelly, MPH, Sharanya Murty, PhD, Augustina Ogbonnaya, MPH, Michael T. Eaddy, PharmD, PhD, Robert J. Fram, MD, Douglas V. Faller, MD PhD, Vamsi Kota, MBBS

Blood (2017) 130 (Supplement 1): 2167.

https://doi.org/10.1182/blood.V130.Suppl_1.2167.2167

Published: 2017

Journal Articles

Multiple Oral Therapeutics Suppress Repressors (LSD-1, HDACs, and BCL11A) of Gamma Globin Gene Expression

Yan Dai, PhD, Douglas V Faller, MD PhD, Jose I Sangerman, PhD, Suthat Fucharoen, Susan P Perrine, MD

Blood (2014) 124 (21): 2687.

https://doi.org/10.1182/blood.V124.21.2687.2687

Published: 2014

Journal Articles

Next Generation Clinical-Stage HbF Inducers Exhibit Multiple Molecular Actions In Vitro and In Vivo and Offer Personalized Therapeutic Regimens

Betty S. Pace, MD, Jose I Sangerman, PhD, Michael S Boosalis, PhD, Roman Wolf, DVM, Yan Dai, PhD, David H.K. Chui, MD, Levi Makala, PhD, DVM, Biadru Li, PhD, Douglas V Faller, MD, PhD, Susan P Perrine, MD

Blood (2013) 122 (21): 560.

https://doi.org/10.1182/blood.V122.21.560.560

Published: 2013

NARROW

Sky-1214, a Small Molecule Splicing Modulator Targeting FANCL and Fanci, Provides a New Mechanism of Action Targeting Multiple Myeloma and Non-Hodgkin's Lymphoma

Iadademstat and Gilteritinib for the Treatment of FLT3-Mutated Relapsed/Refractory Acute Myeloid Leukemia: The Frida Study

Iadademstat Combination with Azacitidine Is a Safe and Effective Treatment in First Line Acute Myeloid Leukemia. Final Results of the Alice Trial

The Frida Study: An Option for Mutated FLT3 Relapsed/Refractory Acute Myeloid Leukemia Patients with a Novel Iadademstat and Gilteritinib Combination Therapy

Health-Related Quality of Life and Symptoms Among Patients with Relapsed or Refractory AL Amyloidosis Treated with Ixazomib-Dexamethasone Versus Physician's Choice: Results from a Randomized Phase 3 Trial

Pevonedistat (PEV) + Azacitidine (AZA) Versus AZA Alone As First-Line Treatment for Patients with Higher-Risk Myelodysplastic Syndromes (MDS)/Chronic Myelomonocytic Leukemia (CMML) or Acute Myeloid Leukemia (AML) with 20-30% Marrow Blasts: The Randomized Phase 3 PANTHER Trial (NCT03268954)

Randomized Phase 2 Trial of Pevonedistat Plus Azacitidine Versus Azacitidine in Higher-Risk Myelodysplastic Syndromes/Chronic Myelomonocytic Leukemia or Low-Blast Acute Myeloid Leukemia: Exploratory Analysis of Patient-Reported Outcomes

Oral Nanatinostat (Nstat) and Valganciclovir (VGCV) in Patients with Recurrent Epstein-Barr Virus (EBV)-Positive Lymphomas: Initial Phase 2 Results

A Randomized Phase 2 Study of Pevonedistat, Venetoclax, and Azacitidine Versus Venetoclax Plus Azacitidine in Adults with Newly Diagnosed Acute Myeloid Leukemia (AML) Who Are Unfit for Intensive Chemotherapy

Primary Results from the Phase 3 Tourmaline-AL1 Trial of Ixazomib-Dexamethasone Versus Physician's Choice of Therapy in Patients (Pts) with Relapsed/Refractory Primary Systemic AL Amyloidosis (RRAL)

Patient-Centered Evaluation of Clinical Benefit in Acute Myeloid Leukemia: Importance of Early Engagement with Patients

Assessing Patient-Reported Outcomes in People with Myelodysplastic Syndromes: Can a Customized Selection of Items from the EORTC Library Enhance the EORTC QLQ-C30?

Pevonedistat, a first-in-class NEDD8-activating enzyme inhibitor, combined with azacitidine in patients with AML

Patient Perspectives on Symptom Experience and Impact on Health-Related Quality of Life in Higher-Risk Myelodysplastic Syndromes, Chronic Myelomonocytic Leukemia and Low-Blast Acute Myeloid Leukemia

Healthcare Costs in Elderly Patients with Acute Myeloid Leukemia (AML) in Routine Clinical Care in the United States (US)

Enhancing the Content of the EORTC QLQ-C30 to Maximize the Detection of Treatment Benefit in Higher-Risk Myelodysplastic Syndromes, Chronic Myelomonocytic Leukemia, and Low-Blast Acute Myeloid Leukemia Using the EORTC Item Bank

Treatment Characteristics and Outcomes for Newly Diagnosed Elderly Patients with Acute Myeloid Leukemia (AML) Treated in the United States with Either 7+3 or a Hypomethylating Agent (HMA)

Multiple Oral Therapeutics Suppress Repressors (LSD-1, HDACs, and BCL11A) of Gamma Globin Gene Expression

Next Generation Clinical-Stage HbF Inducers Exhibit Multiple Molecular Actions In Vitro and In Vivo and Offer Personalized Therapeutic Regimens

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American Society of Hematology

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American Society of Hematology

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