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Journal Articles

A randomized phase 3 trial of interferon-α vs hydroxyurea in polycythemia vera and essential thrombocythemia

Clinical Trials & Observations
John Mascarenhas, Heidi E. Kosiorek, Josef T. Prchal, Alessandro Rambaldi, Dmitriy Berenzon, Abdulraheem Yacoub, Claire N. Harrison, Mary Frances McMullin, Alessandro M. Vannucchi, Joanne Ewing, Casey L. O'Connell, Jean-Jacques Kiladjian, Adam J. Mead, Elliott F. Winton, David S. Leibowitz, Valerio De Stefano, Murat O. Arcasoy, Craig M. Kessler, Rosalind Catchatourian, Damiano Rondelli, Richard T. Silver, Andrea Bacigalupo, Arnon Nagler, Marina Kremyanskaya, Max F. Levine, Juan E. Arango Ossa, Erin McGovern, Lonette Sandy, Mohamad E. Salama, Vesna Najfeld, Joseph Tripodi, Noushin Farnoud, Alexander V. Penson, Rona Singer Weinberg, Leah Price, Judith D. Goldberg, Tiziano Barbui, Roberto Marchioli, Gianni Tognoni, Raajit K. Rampal, Ruben A. Mesa, Amylou C. Dueck, Ronald Hoffman
Journal: Blood
Blood (2022) 139 (19): 2931–2941.
https://doi.org/10.1182/blood.2021012743
Published: 2022
Includes: Supplemental data
Journal Articles

Single-Cell Multi-Omics Reveals That Pegylated Interferon-Alfa Treatment Differentially Redirects Mutated and Wildtype Hematopoietic Cell Differentiation Trajectories in CALR- mutated Essential Thrombocythemia (ET) Patients

Shira Rosenberg, Andrea Kubas-Meyer, Neelang Parghi, Nathaniel D. Omans, Neville Dusaj, Paulina Chamely, Eleni Mimitou, Amylou C. Dueck, Heidi E. Kosiorek, Rona Singer Weinberg, Peter Smibert, Ronan Chaligne, Dan A. Landau, Ronald Hoffman, Anna S Nam
Journal: Blood
Blood (2021) 138 (Supplement 1): 57.
https://doi.org/10.1182/blood-2021-150745
Published: 2021
Journal Articles

Treatment of Myelofibrosis Patients with the TGF-β 1/3 Inhibitor AVID200 (MPN-RC 118) Induces a Profound Effect on Platelet Production

John Mascarenhas, Heidi E. Kosiorek, Rupali Bhave, Jeanne M. Palmer, Andrew T. Kuykendall, Ruben A. Mesa, Raajit Rampal, Aaron T. Gerds, Abdulraheem Yacoub, Kristen M. Pettit, Moshe Talpaz, Rami S. Komrokji, Marina Kremyanskaya, Agapito Gonzalez, Frank Fabris, Lonette Sandy, Kathryn Johnson, Mikaela Doughtery, Erin McGovern, Juan Arango Ossa, Dylan Domenico, Noushin Farnoud, Anna Rita Migliaccio, Mohamed E Salama, Rona Singer Weinberg, Amy Kong, Vesna Najfeld, Carolyn Mead-Harvey, Amylou C. Dueck, Lilian Varricchio, Ronald Hoffman
Journal: Blood
Blood (2021) 138 (Supplement 1): 142.
https://doi.org/10.1182/blood-2021-148995
Published: 2021
Journal Articles

Characterization of Peripheral Blood Mononuclear Cells Addback Following CD34 Enrichment, Engraftment and T and NK Cells Immune Reconstitution in Patients with High Risk Sickle Cell Disease (SCD) (IND 14359)

Yaya Chu, PhD, Julie-An Talano, MD, Lee Ann Baxter-Lowe, PhD, Carolyn A. Keever-Taylor, PhD, Erin Morris, RN, BSN, Harshini Mahanti, BS, MSCR, Janet Ayello, MS, MT(ASCP), Bryon Johnson, PhD, Rona Singer Weinberg, PhD, Qiuhu Shi, PhD, Theodore B. Moore, MD, Sandra Fabricatore, RN, PNP, MSN, Brenda J. Grossman, MD, Carmella van de Ven, MA, Shalini Shenoy, MD, Mitchell S Cairo, MD
Journal: Blood
Blood (2020) 136 (Supplement 1): 20–21.
https://doi.org/10.1182/blood-2020-140425
Published: 2020
Journal Articles

Rationale for and Results of a Phase I Study of the TGF-β 1/3 Inhibitor AVID200 in Subjects with Myelofibrosis: MPN-RC 118 Trial

John Mascarenhas, MD, Heidi E. Kosiorek, MS, Lilian Varricchio, PhD, Rupali Bhave, MD, Andrew T. Kuykendall, MD, Rami Komrokji, MD, Aaron T. Gerds, MDMS, Jeanne M. Palmer, MD, Amelia R. Gabler, Lonette Sandy, Anna Rita Migliaccio, PhD, Mohamed E Salama, MD, Rona Singer Weinberg, PhD, Maureen O'Connor-McCourt, PhD, Gilles Tremblay, PhD, Paul I. Nadler, MD, Amylou C. Dueck, PhD, Ruben A. Mesa, MD FACP, Ronald Hoffman, MD
Journal: Blood
Blood (2020) 136 (Supplement 1): 6–8.
https://doi.org/10.1182/blood-2020-140830
Published: 2020
Journal Articles

Analysis of the Global Methylation Profile of Accelerated and Blast Phase Myeloproliferative Neoplasms and Its Association with Response to Decitabine-Based Therapy

Raajit K. Rampal, MD PhD, Qin Yang, Richard P. Koche, PhD, Erin McGovern, BA, Christopher Famulare, MS, Rona Singer Weinberg, PhD, John Mascarenhas, MD, Ross L. Levine, MD, Ronald Hoffman, MD, Maria E. Figueroa, MD
Journal: Blood
Blood (2020) 136 (Supplement 1): 18–20.
https://doi.org/10.1182/blood-2020-139505
Published: 2020
Journal Articles

Landscape of TP53 Mutations in MPN

Noushin Farnoud, PhD, Christopher Famulare, MS, Elli Papaemmanuil, PhD, Erin McGovern, BA, Juan Medina, MS, Juan E. Arango Ossa, BS, Raajit K. Rampal, MD PhD, Bing Li, MD, Ross L. Levine, MD, John Mascarenhas, MD, Rona Singer Weinberg, PhD, Minal A Patel, MPH, Ronald Hoffman, MD
Journal: Blood
Blood (2019) 134 (Supplement_1): 1681.
https://doi.org/10.1182/blood-2019-132014
Published: 2019
Journal Articles

Identifying Cytokine Biomarkers of Response to Pegylated-Interferon Therapy in Polycythemia Vera and Essential Thrombocythemia: Correlative Analysis from the MPN-RC 111/112 Trials

Andrew Dunbar, MD, Heidi E. Kosiorek, MS, Aishwarya Krishnan, Erin McGovern, BA, Young Park, BS, Minal A Patel, MPH, Rona Singer Weinberg, PhD, Abdulraheem Yacoub, MD, Amylou C. Dueck, PhD, John Mascarenhas, MD, Ross L. Levine, MD, Ronald Hoffman, MD, Raajit K. Rampal, MD PhD
Journal: Blood
Blood (2019) 134 (Supplement_1): 1661.
https://doi.org/10.1182/blood-2019-129571
Published: 2019
Journal Articles

Sustained Donor Chimerism and Rapid Immune Cell Reconstitution Following Familial Haploidentical (FHI) CD34 Enriched Stem Cell Transplantation with Pbmnc Addback in Patients with High Risk Sickle Cell Disease (SCD) (IND 14359)

Yaya Chu, PhD, Julie-An Talano, MD, Lee Ann Baxter-Lowe, PhD, Carolyn A. Keever-Taylor, PhD, Erin Morris, RN, BSN, Harshini Mahanti, BS, MSCR, Janet Ayello, MS, MT(ASCP), Rona Singer Weinberg, PhD, Qiuhu Shi, PhD, Theodore B. Moore, MD, Sandra Fabricatore, RN, PNP, MSN, Brenda J. Grossman, MD, Carmella van de Ven, MA, Shalini Shenoy, MD, Mitchell S Cairo, MD
Journal: Blood
Blood (2019) 134 (Supplement_1): 1990.
https://doi.org/10.1182/blood-2019-126757
Published: 2019
Journal Articles

Final Results of Prospective Treatment with Pegylated Interferon Alfa-2a for Patients with Polycythemia Vera and Essential Thrombocythemia in First and Second-Line Settings

Abdulraheem Yacoub, MD, John Mascarenhas, MD, Ruben A. Mesa, MD, Heidi E. Kosiorek, MS, Raajit K. Rampal, MD PhD, Richard T. Silver, MD, Mohamed E. Salama, MD, Olivia Siwoski, MD, Amylou C. Dueck, PhD, Lonette Sandy, Mary Frances McMullin, MD FRCPath, FRCP, Joanne Ewing, PhDBMBS BSc FRPATH, Casey L. O'Connell, MD, Adam J. Mead, MD PhD, Valerio De Stefano, MD, Rona Singer Weinberg, PhD, Maria R. Baer, MD, Craig M. Kessler, MD, Elliott F. Winton, MD, Alessandro M. Vannucchi, MD, Marina Kremyanskaya, Joseph Vadakara, MBBS, Vittorio Rosti, MD, Elizabeth O. Hexner, MDMS, Damiano Rondelli, MD, Murat O. Arcasoy, MD, Alessandro Rambaldi, MD, Ellen K. Ritchie, MD, Tiziano Barbui, MD, Jean-Jacques Kiladjian, MD PhD, Claire N Harrison, Professor, Josef T. Prchal, MD, Ronald Hoffman, MD
Journal: Blood
Blood (2019) 134 (Supplement_1): 2943.
https://doi.org/10.1182/blood-2019-124865
Published: 2019
Journal Articles

Pegylated interferon alfa-2a for polycythemia vera or essential thrombocythemia resistant or intolerant to hydroxyurea

Clinical Trials & Observations
Abdulraheem Yacoub, John Mascarenhas, Heidi Kosiorek, Josef T. Prchal, Dmitry Berenzon, Maria R. Baer, Ellen Ritchie, Richard T. Silver, Craig Kessler, Elliott Winton, Maria Chiara Finazzi, Alessandro Rambaldi, Alessandro M. Vannucchi, David Leibowitz, Damiano Rondelli, Murat O. Arcasoy, Rosalind Catchatourian, Joseph Vadakara, Vittorio Rosti, Elizabeth Hexner, Marina Kremyanskaya, Lonette Sandy, Joseph Tripodi, Vesna Najfeld, Noushin Farnoud, Elli Papaemmanuil, Mohamed Salama, Rona Singer-Weinberg, Raajit Rampal, Judith D. Goldberg, Tiziano Barbui, Ruben Mesa, Amylou C. Dueck, Ronald Hoffman
Journal: Blood
Blood (2019) 134 (18): 1498–1509.
https://doi.org/10.1182/blood.2019000428
Published: 2019
Includes: Supplemental data
Journal Articles

Low Dose Umbilical Cord Blood Transplant Results in Skewed Immune Cell Composition Which May Impact Immune Reconstitution after Allogeneic Hematopoietic Stem Cell Transplantation

Chi Hua Sarah Lin, PhD, Beth Shaz, MD, Rona Singer Weinberg, PhD
Journal: Blood
Blood (2018) 132 (Supplement 1): 5672.
https://doi.org/10.1182/blood-2018-99-111541
Published: 2018
Journal Articles

Results of the Myeloproliferative Neoplasms - Research Consortium (MPN-RC) 112 Randomized Trial of Pegylated Interferon Alfa-2a (PEG) Versus Hydroxyurea (HU) Therapy for the Treatment of High Risk Polycythemia Vera (PV) and High Risk Essential Thrombocythemia (ET)

John Mascarenhas, MD, Heidi E. Kosiorek, MS, Josef T. Prchal, MD, Alessandro Rambaldi, MD, Dmitriy Berenzon, MD, Abdulraheem Yacoub, MD, Claire N. Harrison, MD DM, FRCP, PRCPath, Mary Frances McMullin, MD, Alessandro M. Vannucchi, MD, Joanne Ewing, PhD BMBS, BSc, FRPATH,, Casey L. O'Connell, MD, Jean-Jacques Kiladjian, MD PhD, Adam J. Mead, MD PhD, Elliott F. Winton, MD, David S. Leibowitz, MD, Valerio De Stefano, MD, Murat O. Arcasoy, MD, Craig M. Kessler, Rosalind Catchatourian, MD, Damiano Rondelli, MD, Richard T. Silver, MD, Andrea Bacigalupo, MD, Arnon Nagler, MD, Marina Kremyanskaya, MD PhD, Lonette Sandy, Mohamed E. Salama, MD, Vesna Najfeld, PhD, Joseph Tripodi, Rona Singer Weinberg, PhD, Leah Price, Judith D Goldberg, ScD, Raajit K. Rampal, MD PhD, Ruben A. Mesa, MD FACP, Amylou C. Dueck, PhD, Ronald Hoffman, MD
Journal: Blood
Blood (2018) 132 (Supplement 1): 577.
https://doi.org/10.1182/blood-2018-99-111946
Published: 2018
Journal Articles

Efficacy of Combined Ruxolitinib and Decitabine in Patients with Accelerated and Blast-Phase Myeloproliferative Neoplasms: Results of a Phase II Study (MPN-RC 109 trial)

Raajit K. Rampal, MD PhD, John Mascarenhas, MD, Heidi E. Kosiorek, MS, Rupali Bhave, MD, Elizabeth O. Hexner, MD MS, Eunice S. Wang, MD, Aaron T. Gerds, MD MS, Mark L Heaney, MD PhD, Camille N Abboud, MD, Marina Kremyanskaya, MD PhD, Dmitriy Berenzon, MD, Olatoyosi Odenike, MD, Elizabeth Virtgaym, Lonette Sandy, Rona Singer Weinberg, PhD, Vesna Najfeld, PhD, Mohamed E. Salama, MD, Ross L. Levine, MD, Ruben A. Mesa, MD FACP, Amylou C. Dueck, PhD, Ronald Hoffman, MD
Journal: Blood
Blood (2018) 132 (Supplement 1): 3027.
https://doi.org/10.1182/blood-2018-99-111935
Published: 2018
Journal Articles

Significantly Improved Long Term Health Related Quality of Life (HRQL) and Neurocognition Following Familial Haploidentical Stem Cell Transplantation (HISCT) Utilizing CD34 Enrichment and Mononuclear (CD3) Addback in High Risk Patients with Sickle Cell Disease (SCD)

Mitchell Cairo, MD, Susan K Parsons, MD MRP, Suzanne Braniecki, PhD, Julie-An Talano, MD, Theodore B Moore, MD, Qiuhu Shi, PhD, Carolyn A. Keever-Taylor, DPhil, Rona Singer Weinberg, PhD, Brenda J. Grossman, MD, Allen J Dozor, MD, Deborah Friedman, MD, Robert C McKinstry, MD PhD, James Verbsky, Chitti R Moorthy, MD, Ruth Ann Weidner, MBA, MRP, Angie Mae Rodday, PhD, Janet Ayello, MS, MT(ASCP), Mildred Semidei-Pomales, MS, Allyson M. Flower, MD, Yaya Chu, PhD, Erin Morris, RN, BSN, Harshini Mahanti, BS, MSCR, Sandra Fabricatore, RN, PNP, MSN, Olga Militano, PharmD, Liana Klejmont, Pharm D, Carmella van de Ven, MA, Lee Ann Baxter-Lowe, PhD, Elliott Vichinsky, MD, Mark C. Walters, MD, Shalini Shenoy, MD
Journal: Blood
Blood (2018) 132 (Supplement 1): 162.
https://doi.org/10.1182/blood-2018-162
Published: 2018
Journal Articles

Impact on MPN Symptoms and Quality of Life of Front Line Pegylated Interferon Alpha-2a Vs. Hydroxyurea in High Risk Polycythemia Vera and Essential Thrombocythemia: Results of Myeloproliferative Disorders Research Consortium (MPD-RC) 112 Global Phase III Trial

Ruben A. Mesa, MD FACP, Heidi E. Kosiorek, MS, John Mascarenhas, MD, Josef T. Prchal, MD, Alessandro Rambaldi, MD, Dmitriy Berenzon, MD, Abdulraheem Yacoub, MD, Claire N. Harrison, MD DM, FRCP, PRCPath, Mary Frances McMullin, MD, Alessandro M. Vannucchi, MD, Joanne Ewing, PhD BMBS, BSc, FRPATH,, Casey L. O'Connell, MD, Jean-Jacques Kiladjian, MD PhD, Adam J. Mead, MD PhD, Elliott F. Winton, MD, David S. Leibowitz, MD, Valerio De Stefano, MD, Murat O. Arcasoy, MD, Craig M. Kessler, Rosalind Catchatourian, MD, Damiano Rondelli, MD, Richard T. Silver, MD, Andrea Bacigalupo, MD, Arnon Nagler, MD, Marina Kremyanskaya, MD PhD, Lonette Sandy, Mohamed E. Salama, MD, Vesna Najfeld, PhD, Joseph Tripodi, Rona Singer Weinberg, PhD, Raajit K. Rampal, MD PhD, Ronald Hoffman, MD, Amylou C. Dueck, PhD
Journal: Blood
Blood (2018) 132 (Supplement 1): 3032.
https://doi.org/10.1182/blood-2018-99-111929
Published: 2018
Journal Articles

Single-Arm Phase II Trial of Pegylated Interferon Alpha 2a in Patients with High-Risk Essential Thrombocythemia or Polycythemia Vera Resistant or Intolerant to Hydroxyurea: MPN Symptom Impact & Quality of Life Results of the Myeloproliferative Disorders-Research Consortium (MPD-RC) Protocol 111 Global Phase II Trial

Amylou C. Dueck, PhD, John Mascarenhas, MD, Heidi Elizabeth Kosiorek, MS, Abdulraheem Yacoub, MD, Josef T. Prchal, MD, Dmitriy Berenzon, MD, Maria R. Baer, MD, Ellen K Ritchie, MD, Richard T Silver, MD, Craig M. Kessler, MD, Elliott F. Winton, MD, Maria Chiara Finazzi, MD, Alessandro Rambaldi, MD Prof, Alessandro M. Vannucchi, David S. Leibowitz, MD, Damiano Rondelli, MD, Murat O. Arcasoy, MD, Rosalind Catchatourian, MD, Joseph Vadakara, MBBS, Vittorio Rosti, MD, Elizabeth Hexner, MDMS, Marina Kremyanskaya, MD PhD, Alicia Orellana, MD MPH, Lonette Sandy, Joseph Tripodi, Vesna Najfeld, PhD, Mohamed E. Salama, MD, Rona Singer Weinberg, PhD, Raajit K. Rampal, MD PhD, Ronald Hoffman, MD, Ruben A Mesa, MD
Journal: Blood
Blood (2017) 130 (Supplement 1): 4206.
https://doi.org/10.1182/blood.V130.Suppl_1.4206.4206
Published: 2017
Journal Articles

Promising Results at 1 Year Follow-up Following Familial Haploidentical (FHI) T-Cell Depleted (TCD) with CD34 Enrichment and T-Cell (CD3) Addback Allogeneic Stem Cell Transplantation in Patients with High-Risk Sickle Cell Disease (SCD) (IND 14359)

Julie-An Talano, MD, Theodore B Moore, MD, Carolyn A. Keever-Taylor, DPhil, Shalini Shenoy, MD, Mark C. Walters, MD, Susan K Parsons, MD MRP, Allen J Dozor, MD, Deborah Friedman, MD, Qiuhu Shi, PhD, Suzanne Braniecki, PhD, Brenda J. Grossman, MD, Rona Singer Weinberg, PhD, Elliott Vichinsky, MD, Yaya Chu, PhD, Erin Morris, RN, BSN, Sandra Fabricatore, RN, PNP, MSN, Janet Ayello, MS, MT(ASCP), Lee Ann Baxter-Lowe, PhD, Mitchell S. Cairo, MD
Journal: Blood
Blood (2017) 130 (Supplement 1): 4602.
https://doi.org/10.1182/blood.V130.Suppl_1.4602.4602
Published: 2017
Journal Articles

SETBP1 and NRAS Mutations Are Frequent Events in Post-Myeloproliferative Neoplasm Acute Myeloid Leukemia (post-MPN AML) Lacking JAK-STAT Activating Mutations

Kamal Menghrajani, MD, Franck Rapaport, PhD, Caroline Jane McNamara, MBBS, Sean M. Devlin, PhD, Christopher Famulare, MS, Minal Patel, BA, Juan Medina, Lonette Sandy, Heidi Kosiorek, MS, Rona Singer Weinberg, PhD, Andrea Arruda, Jessica Schulman, Taghi Manshouri, Marina Kremyanskaya, MD PhD, John Mascarenhas, MD, Ronald Hoffman, MD, Vikas Gupta, MD PhD, Srdan Verstovsek, MD PhD, Ross L. Levine, MD, Raajit K. Rampal, MD PhD
Journal: Blood
Blood (2017) 130 (Supplement 1): 203.
https://doi.org/10.1182/blood.V130.Suppl_1.203.203
Published: 2017
Journal Articles

Single-Arm Salvage Therapy with Pegylated Interferon Alfa-2a for Patients with High-Risk Polycythemia Vera or High-Risk Essential Thrombocythemia Who Are Either Hydroxyurea-Resistant or Intolerant: Final Results of the Myeloproliferative Disorders-Research Consortium (MPD-RC) Protocol 111 Global Phase II Trial

Abdulraheem Yacoub, MD, John Mascarenhas, MD, Heidi Elizabeth Kosiorek, MS, Josef T. Prchal, MD, Dmitriy Berenzon, MD, Maria R. Baer, MD, Ellen K Ritchie, MD, Richard T Silver, MD, Craig M. Kessler, MD, Elliott F. Winton, MD, Maria Chiara Finazzi, MD, Alessandro Rambaldi, MD Prof, Alessandro M. Vannucchi, David S. Leibowitz, MD, Damiano Rondelli, MD, Murat O. Arcasoy, MD, Rosalind Catchatourian, MD, Joseph Vadakara, MBBS, Vittorio Rosti, MD, Elizabeth Hexner, MDMS, Marina Kremyanskaya, MD PhD, Alicia Orellana, MD MPH, Lonette Sandy, Joseph Tripodi, Vesna Najfeld, PhD, Mohamed E. Salama, MD, Rona Singer Weinberg, PhD, Raajit K. Rampal, MD PhD, Ruben A Mesa, MD, Judith D Goldberg, ScD, Amylou C. Dueck, PhD, Ronald Hoffman, MD
Journal: Blood
Blood (2017) 130 (Supplement 1): 321.
https://doi.org/10.1182/blood.V130.Suppl_1.321.321
Published: 2017