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Journal Articles

Blinatumomab in Pediatric Patients with Relapsed/Refractory B-Cell Precursor and Molecularly Resistant Acute Lymphoblastic Leukemia (R/R ALL): Updated Analysis of 110 Patients Treated in an Expanded Access Study (RIALTO)

Franco Locatelli, MD, Gerhard Zugmaier, Peter Bader, MD, Sima Jeha, MD, Paul-Gerhardt Schlegel, Jean-Pierre Bourquin, MD PhD, Rupert Handgretinger, MD, Benoit Brethon, MD, Claudia Rossig, Christiane Chen-Santel

Blood (2019) 134 (Supplement_1): 1294.

https://doi.org/10.1182/blood-2019-121615

Published: 2019

Journal Articles

Long-Term Outcomes of Subjects with Epstein-Barr Virus-Driven Post-Transplant Lymphoproliferative Disorder (EBV + PTLD) Following Solid Organ (SOT) or Allogeneic Hematopoietic Cell Transplants (HCT) Treated with Tabelecleucel on an Expanded Access Program

Susan Prockop, MD, Ran Reshef, MDMSc, Donald E. Tsai, MD PhD, Nancy Bunin, MD, Rolla Abu-Arja, MD, Kris Michael Mahadeo, MD, Wen-Kai Weng, MD PhD, Koen Van Besien, MD PhD, David Loeb, MD PhD, Sunita Dwivedy Nasta, MD, Eneida R. Nemecek, MDMBA,MSc, Minoti Hiremath, MBBS, PhD, Susan Yue, MD, Yan Sun, PhD, Willis Navarro, MD, Sarah Nikiforow, MD PhD

Blood (2019) 134 (Supplement_1): 4071.

https://doi.org/10.1182/blood-2019-124904

Published: 2019

Journal Articles

Results from an Expanded Access Program of Anti-CD3/CD7 Immunotoxin Combination (T-Guard®) for the Treatment of Steroid-Refractory Acute Gvhd

Lenneke F J Groningen, Christoph Groth, MD, Manita E J Bremmers, Eric G Hooren, Ypke V J M van Oosterhout, Nicole M A Blijlevens, Matthias Stelljes, Walter J F M van der Velden

Blood (2019) 134 (Supplement_1): 4553.

https://doi.org/10.1182/blood-2019-122923

Published: 2019

Journal Articles

Targeted FGFR Inhibition Results in Hematologic and Cytogenetic Remission in a Myeloid Neoplasm Driven By a Novel PCM1-FGFR1 Fusion: Data from an Expanded Access Program

Monica Kasbekar, Valentina Nardi, MD, Paola Dal Cin, Andrew M. Brunner, MD, Yi-Bin Chen, Christine Connolly, Amir T. Fathi, MD, Julia Foster, ANP, Molly Macrae, Steven L McAfee, MD, Kristin McGregor, Rupa Narayan, Aura Y. Ramos, Tina T. Som, RN, Megan Vartanian, RN, Robb S Friedman, MD, Karim Benhadji, MD MSC, Gabriela S. Hobbs, MD

Blood (2019) 134 (Supplement_1): 5371.

https://doi.org/10.1182/blood-2019-124100

Published: 2019

Journal Articles

The Real Life Accessibility to CAR T-Cell Therapy: Current Experience in the Only Active Center in Italy

Anna Guidetti, MD, Giulia Perrone, MD, Paola Coluccia, MD, Luca Fumagalli, MD, Anna Dodero, MD, Lucia Farina, MD, Flavio Arienti, MD, Filippo Bagnoli, MD, Luca Pappalettera, MD, Debora Degli Innocenti, Cristiana Carniti, PhD, Daniela Codazzi, MD, Paolo Corradini

Blood (2019) 134 (Supplement_1): 5619.

https://doi.org/10.1182/blood-2019-125286

Published: 2019

Journal Articles

Open-Label, Expanded Access Study of Taliglucerase Alfa in Patients with Gaucher Disease Requiring Enzyme Replacement Therapy

David J. Kuter, MD DPhil, Michael Wajnrajch, MD MPA, Betina Hernandez, MD, Rong Wang, MPH, Raul Chertkoff, MD MBA, Ari Zimran, MD

Blood (2018) 132 (Supplement 1): 3755.

https://doi.org/10.1182/blood-2018-99-112660

Published: 2018

Journal Articles

Safety of Gemtuzumab Ozogamicin As Monotherapy or Combination Therapy in an Expanded-Access Protocol for Patients with Relapsed or Refractory Acute Myeloid Leukemia

Eunice S. Wang, MD, Richard Aplenc, MD PhD, Deborah Chirnomas, Michael John Dugan, MD, Salman Fazal, MD, Swaminathan Iyer, Tara L Lin, MD, Sucha Nand, MD, Kristen J Pierce, Paul J Shami, MD, Jennifer J Vermette, Camille N Abboud, MD

Blood (2018) 132 (Supplement 1): 3979.

https://doi.org/10.1182/blood-2018-99-110804

Published: 2018

Journal Articles

Use of Gemtuzumab Ozogamicin for the Treatment of Relapsed or Refractory Acute Myeloid Leukemia (AML) or Acute Promyelocytic Leukemia (APL) in an Expanded Access Setting at Our Cancer Consortium

Blood (2018) 132 (Supplement 1): 2710.

https://doi.org/10.1182/blood-2018-99-116908

Published: 2018

Journal Articles

Midostaurin in Adults with Newly Diagnosed FLT3 -Mutation-Positive Acute Myeloid Leukemia Eligible for Standard Chemotherapy: Update from the Radius-X Midostaurin Expanded Access Program

Alexander E. Perl, MD, Kendra L. Sweet, MD, Gail J. Roboz, MD, Stephen A. Strickland, MD, Mark R. Litzow, MD, Gaetano J Bonifacio, MD, Kelly Haines, Alysha Barbera, Das Purkayastha, Andrew Dalovisio, MD

Blood (2018) 132 (Supplement 1): 4038.

https://doi.org/10.1182/blood-2018-99-113608

Published: 2018

Journal Articles

High Molecular Remission Rate in Pediatric Patients (pts) with Relapsed/Refractory B-Cell Precursor Acute Lymphoblastic Leukemia (r/r ALL) Treated with Blinatumomab: Rialto an Open-Label, Multicenter, Expanded Access Study

Franco Locatelli, Gerhard Zugmaier, Peter Bader, MD PhD, Sima Jeha, MD, Paul-Gerhardt Schlegel, MD, Jean-Pierre Bourquin, MD PhD, Rupert Handgretinger, MD PhD, Benoit Brethon, Claudia Rossig, MD, Christiane Chen-Santel, MD

Blood (2018) 132 (Supplement 1): 1375.

https://doi.org/10.1182/blood-2018-99-109855

Published: 2018

Journal Articles

Incidence of Post-Hematopoietic Stem Cell Transplantation (HSCT) Veno-Occlusive Disease/Sinusoidal Obstruction Syndrome (VOD/SOS) without Hyperbilirubinemia at Diagnosis and Efficacy of Defibrotide in an Expanded-Access Program

Selim Corbacioglu, MD, Nancy A. Kernan, Antonio Pagliuca, Robert Ryan, William Tappe, Paul Richardson

Blood (2018) 132 (Supplement 1): 2080.

https://doi.org/10.1182/blood-2018-99-117173

Published: 2018

Journal Articles

Primary Analysis of JUMP, a Phase 3b, Expanded-Access Study Evaluating the Safety and Efficacy of Ruxolitinib in Patients with Myelofibrosis (N = 2233)

Haifa Kathrin Al-Ali, Lynda Foltz, Giuseppe A. Palumbo, Bruno Martino, Francesca Palandri, PhD MD, Anna Marina Liberati, Philipp Le Coutre, Carmen Garcia-Hernandez, Andrey Zaritskey, Renato Tavares, Martin Griesshammer, Vikas Gupta, MD PhD, Pia Raanani, Pilar Giraldo, PhD, Catherine Bouard, Julian Perez Ronco, Ranjan Tiwari, Francesco Mannelli, Alessandro M. Vannucchi

Blood (2017) 130 (Supplement 1): 4204.

https://doi.org/10.1182/blood.V130.Suppl_1.4204.4204

Published: 2017

Journal Articles

Real-World Data of Nivolumab in Classical Hodgkin Lymphoma: Results from the Italian Expanded Access Programme

Blood (2017) 130 (Supplement 1): 5171.

https://doi.org/10.1182/blood.V130.Suppl_1.5171.5171

Published: 2017

Journal Articles

Treatment of Hepatic Veno-Occlusive Disease/Sinusoidal Obstruction Syndrome (VOD/SOS) Post-Hematopoietic Stem Cell Transplantation (HSCT) in Patients with Neuroblastoma: Final Data from the Defibrotide Expanded-Access Program

Stephan A Grupp, MD PhD, Brandon Triplett, MD, Araz Marachelian, MD, Sonata Jodele, MD, Robert J. Ryan, Robin L. Hume, MS, William Martin Tappe, MD, Paul G Richardson, MD

Blood (2017) 130 (Supplement 1): 4549.

https://doi.org/10.1182/blood.V130.Suppl_1.4549.4549

Published: 2017

Journal Articles

Efficacy and Safety of Defibrotide in Pediatric Patients with Veno-Occlusive Disease/Sinusoidal Obstruction Syndrome (VOD/SOS) after Hematopoietic Stem Cell Transplantation (HSCT): Final Results from the Expanded-Access Program

Nancy A. Kernan, MD, Angela R. Smith, MD MS, Brandon Triplett, MD, Leslie Lehmann, MD, Robert J. Ryan, Wei Liang, Robin L. Hume, MS, William Martin Tappe, MD, Stephan A Grupp, MD PhD

Blood (2017) 130 (Supplement 1): 1948.

https://doi.org/10.1182/blood.V130.Suppl_1.1948.1948

Published: 2017

Journal Articles

Expanded Access to Obinutuzumab from Cost-Savings Generated By Biosimilar Filgrastim-Sndz in the Prophylaxis of Chemotherapy-Induced (Febrile) Neutropenia: US Simulation Study

Ali McBride, PharmD,MS, Kim Campbell, Mohan Bikkina, Karen MacDonald, Ivo Abraham, PhD RN, Sanjeev Balu

Blood (2017) 130 (Supplement 1): 3380.

https://doi.org/10.1182/blood.V130.Suppl_1.3380.3380

Published: 2017

Journal Articles

Final Analysis of Day +100 Survival By Prior Hematopoietic Stem Cell Transplant Type from an Expanded Access Study of Defibrotide for Hepatic Veno-Occlusive Disease/Sinusoidal Obstruction Syndrome

Paul G Richardson, MD, Angela R. Smith, MD MS, Brandon Triplett, MD, Nancy Kernan, MD, Leslie Lehmann, MD, Robert J. Ryan, Robin L. Hume, MS, William Martin Tappe, MD, Stephan A Grupp, MD PhD

Blood (2017) 130 (Supplement 1): 3224.

https://doi.org/10.1182/blood.V130.Suppl_1.3224.3224

Published: 2017

Journal Articles

Adults Receiving Defibrotide for the Treatment of Hepatic Veno-Occlusive Disease/Sinusoidal Obstruction Syndrome (VOD/SOS) after Hematopoietic Stem Cell Transplantation (HSCT): Final Results from the Expanded-Access Program

Paul G Richardson, MD, Joseph H. Antin, MD, Sergio A. Giralt, MD, Robert J. Ryan, Wei Liang, Robin L. Hume, MS, William Martin Tappe, MD, Nancy A. Kernan, MD

Blood (2017) 130 (Supplement 1): 3225.

https://doi.org/10.1182/blood.V130.Suppl_1.3225.3225

Published: 2017

Journal Articles

Safety and Efficacy of Ruxolitinib for the Final Enrollment of JUMP: An Open-Label, Multicenter, Single-Arm, Expanded-Access Study in Patients with Myelofibrosis (N = 2233)

Lynda Foltz, MD FRCP, Giuseppe A. Palumbo, MD, Bruno Martino, MD, Francesca Palandri, MD PhD, Haifa Kathrin Al-Ali, MD, Anna Marina Liberati, MD, Philipp D. Le Coutre, MD, Alessandro M. Vannucchi, MD, Carmen Garcia-Hernandez, MD, Renato Tavares, MD, Martin Griesshammer, MD, Vikas Gupta, MD, Pia Raanani, MD, Pilar Giraldo, MD PhD, Catherine Bouard, Julian Perez Ronco, MD MBA, Sadhvi Khanna, MS, Andrey Zaritskey, MD

Blood (2016) 128 (22): 3107.

https://doi.org/10.1182/blood.V128.22.3107.3107

Published: 2016

Journal Articles

Treatment of Hepatic Veno-Occlusive Disease/Sinusoidal Obstruction Syndrome (VOD/SOS) Post-Hematopoietic Stem Cell Transplantation (HSCT) in Patients with Acute Leukemias: A Subgroup Analysis from the Defibrotide Expanded-Access Program

Paul G. Richardson, MD, Angela R. Smith, MD, Brandon M. Triplett, MD, Nancy A. Kernan, MD, Stephan A. Grupp, MD PhD, Joseph H. Antin, MD, Leslie Lehmann, MD, Wei Liang, PhD, Robin Hume, MS, William Tappe, MD, Robert J. Soiffer, MD

Blood (2016) 128 (22): 3412.

https://doi.org/10.1182/blood.V128.22.3412.3412

Published: 2016

NARROW

Blinatumomab in Pediatric Patients with Relapsed/Refractory B-Cell Precursor and Molecularly Resistant Acute Lymphoblastic Leukemia (R/R ALL): Updated Analysis of 110 Patients Treated in an Expanded Access Study (RIALTO)

Long-Term Outcomes of Subjects with Epstein-Barr Virus-Driven Post-Transplant Lymphoproliferative Disorder (EBV + PTLD) Following Solid Organ (SOT) or Allogeneic Hematopoietic Cell Transplants (HCT) Treated with Tabelecleucel on an Expanded Access Program

Results from an Expanded Access Program of Anti-CD3/CD7 Immunotoxin Combination (T-Guard®) for the Treatment of Steroid-Refractory Acute Gvhd

Targeted FGFR Inhibition Results in Hematologic and Cytogenetic Remission in a Myeloid Neoplasm Driven By a Novel PCM1-FGFR1 Fusion: Data from an Expanded Access Program

The Real Life Accessibility to CAR T-Cell Therapy: Current Experience in the Only Active Center in Italy

Open-Label, Expanded Access Study of Taliglucerase Alfa in Patients with Gaucher Disease Requiring Enzyme Replacement Therapy

Safety of Gemtuzumab Ozogamicin As Monotherapy or Combination Therapy in an Expanded-Access Protocol for Patients with Relapsed or Refractory Acute Myeloid Leukemia

Use of Gemtuzumab Ozogamicin for the Treatment of Relapsed or Refractory Acute Myeloid Leukemia (AML) or Acute Promyelocytic Leukemia (APL) in an Expanded Access Setting at Our Cancer Consortium

Midostaurin in Adults with Newly Diagnosed FLT3 -Mutation-Positive Acute Myeloid Leukemia Eligible for Standard Chemotherapy: Update from the Radius-X Midostaurin Expanded Access Program

High Molecular Remission Rate in Pediatric Patients (pts) with Relapsed/Refractory B-Cell Precursor Acute Lymphoblastic Leukemia (r/r ALL) Treated with Blinatumomab: Rialto an Open-Label, Multicenter, Expanded Access Study

Incidence of Post-Hematopoietic Stem Cell Transplantation (HSCT) Veno-Occlusive Disease/Sinusoidal Obstruction Syndrome (VOD/SOS) without Hyperbilirubinemia at Diagnosis and Efficacy of Defibrotide in an Expanded-Access Program

Primary Analysis of JUMP, a Phase 3b, Expanded-Access Study Evaluating the Safety and Efficacy of Ruxolitinib in Patients with Myelofibrosis (N = 2233)

Real-World Data of Nivolumab in Classical Hodgkin Lymphoma: Results from the Italian Expanded Access Programme

Treatment of Hepatic Veno-Occlusive Disease/Sinusoidal Obstruction Syndrome (VOD/SOS) Post-Hematopoietic Stem Cell Transplantation (HSCT) in Patients with Neuroblastoma: Final Data from the Defibrotide Expanded-Access Program

Efficacy and Safety of Defibrotide in Pediatric Patients with Veno-Occlusive Disease/Sinusoidal Obstruction Syndrome (VOD/SOS) after Hematopoietic Stem Cell Transplantation (HSCT): Final Results from the Expanded-Access Program

Expanded Access to Obinutuzumab from Cost-Savings Generated By Biosimilar Filgrastim-Sndz in the Prophylaxis of Chemotherapy-Induced (Febrile) Neutropenia: US Simulation Study

Final Analysis of Day +100 Survival By Prior Hematopoietic Stem Cell Transplant Type from an Expanded Access Study of Defibrotide for Hepatic Veno-Occlusive Disease/Sinusoidal Obstruction Syndrome

Adults Receiving Defibrotide for the Treatment of Hepatic Veno-Occlusive Disease/Sinusoidal Obstruction Syndrome (VOD/SOS) after Hematopoietic Stem Cell Transplantation (HSCT): Final Results from the Expanded-Access Program

Safety and Efficacy of Ruxolitinib for the Final Enrollment of JUMP: An Open-Label, Multicenter, Single-Arm, Expanded-Access Study in Patients with Myelofibrosis (N = 2233)

Treatment of Hepatic Veno-Occlusive Disease/Sinusoidal Obstruction Syndrome (VOD/SOS) Post-Hematopoietic Stem Cell Transplantation (HSCT) in Patients with Acute Leukemias: A Subgroup Analysis from the Defibrotide Expanded-Access Program

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American Society of Hematology

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American Society of Hematology

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