UMBILICAL CORD BLOOD STEM CELLS EXPANDED IN THE PRESENCE OF NICOTINAMIDE (NICORD) PROVIDE LONG TERM MULITI-LINEAGE ENGRAFTMENT
|
|
- Mary Manning
- 5 years ago
- Views:
Transcription
1 UMBILICAL CORD BLOOD STEM CELLS EXPANDED IN THE PRESENCE OF NICOTINAMIDE (NICORD) PROVIDE LONG TERM MULITI-LINEAGE ENGRAFTMENT Mitchell E. Horwitz, MD Duke University Medical Center Duke Cancer Institute Durham, NC Gamida Cell Ltd. Jerusalem, Israel International Society of Cellular Therapy Philadelphia Pa. September 9, 213
2 Limitations of Umbilical Cord Blood Transplantation Low stem cell dose Graft failure 1-2% Highest in adult population Delayed immunologic recovery Delayed hematopoietic recovery Modulation or expansion of UCB stem cells may address these limitations
3 Source of Problem Donor Graft Comparison (adult recipients) Bone Marrow Umbilical Cord Peripheral Blood Nucleated Cells 1-5 x 1 8 /kg.15-1 x 1 8 /kg 1-4 x 1 8 /kg CD34+ cells.5-2 x 1 6 /kg.5-.2 x 1 6 /kg 2-2 x 1 6 /kg T-Lymphocytes 1 x x x 1 8
4 Cumulative Incidence, % 5-DCB; Phase II Multicenter Myeloablative Dual UCB Transplantation 1 89% (8-96) at day % (76-93) at day Days Barker J et al. 213 In Preparation
5 Goals of Ex Vivo Graft Manipulation 1. Augment Migration and Homing 2. Supplement Committed Progenitor Cell Content 3. Preserve or Increase Pleuripotent Stem Cell Numbers* Blood Endothelium Migration 1. Rolling 2. Integrin Activation 3. Arrest 4. Transendothelial Migration Homing & Retention Self renewal Differentiation Hematopoietic Stem Cell (HSC)
6 NiCord Cord Blood Expansion Technology NiCord active molecule; Nicotinamide (NAM) Vitamin B3 derivative Precursor of NAD Potent inhibitor of enzymes that utilize NAD as a cofactor Combined with stem cell early active cytokines TPO, IL-6, FLT-3 ligand, SCF Cultured in media containing FBS
7 Migration (%) No. of CD34 + cells NAM NAM Stimulates Augments the Migration Migration and Bone and BM Marrow Homing Homing Efficacy of Ex Vivo of Ex Vivo Expanded Expanded CD34 + Cells CD34 + Cells A Migration of CD34+ cells in response to SDF-1 5 *P <.2 **P =.5 B BM homing of CD34+ cells *P <.1 * 4 ** * Noncultured Cultured Cultured + NAM Noncultured Cultured Cultured + NAM B Mice transplanted with 1x1 6 cells/mouse Human progenitor cells (CFSE+CD34+) evaluated 24 hrs. post transplantation Peled T et al. Exp. Heme 212; 4: 342
8 Increased Engraftment Efficacy of HPC Expanded with Cytokines (Cyt) + NAM vs. Cyt only in SCID/NSG Mice % from human CD45 + cells About 1 fold increase in the number of SRC Limiting dilution experiments PB Myeloid and lymphoid reconstitution by NAM expanded HPC in NSG mice (12 weeks) 3 x x CD3 CD3/4 CD3/8 CD56 CD15 CD11 CD41 CD33 CD19
9 NiCord Cord Blood Expansion Technology Heat Map construct: clusters of genes differentially expressed in CD133 + cells expanded ±NAM for 3-weeks vs. non-cultured CD133 + cells
10 % from total cells CD34 NAM Mechanism of Action Sir2 enzymes are suggested as the primary target of NAM NAM (mm) EX527 (µm) NAM: Potent inhibitor of enzymes that utilize NAD as a co-factor. Sir2 (SIRT1-7): class-iii NAD-dependent histone-deacetylases. *EX527: Specific inhibitor of SIRT1 6 5 SDF-1 induced migration Inhibition of differentiation.2% 62% 53% Cyt % 22% 16% NAM 1 Cytokines only + NAM 2.5 mm +EX µm 3.4% 24% 15% EX527 CD38 CD14 CD11
11 NiCord Phase I/II Trial; Hematologic Malignancies Study Design Duke University Medical Center/Loyola University Adult patients with high risk hematological malignancies Myeloablative conditioning regimen Total body irradiation (135cGy) Fludarabine 16mg/m 2 Cyclophosphamide 12mg/kg (optional; n=2) Double cord configuration: NiCord + Unmanipulated unit GvHD prophylaxis: tacrolimus/mycophenolate mofetil Minimum pre-freeze TNC dose requirement NiCord unit; 1.5 x 1 7 /kg Unmanipulated unit of 2.5 x 1 7 /kg Outcomes compared to Duke Historical Controls
12 Patient and Graft Characteristics Characteristics Subjects n=11 Historical Controls n = 17 Age (years) Median Range Weight (kg) Median Range Diagnosis (%) AML or MDS 8 (73) 1 (58) ALL 1 (9) 4 (24) CML 1 (6) Lymphoma 2 (18) 2 (12) Disease status at time of transplantation (%) Complete remission First Remission 4(36) 2(12) Second or subsequent remission 3(28) 12(71) Active disease 4(36) 3(17) Preparative Regimen TBI 135cGy/Fludarabine 9 17 TBI 135cGy/Fludarabine/Cyclophosphamide 2 Donor-recipient HLA compatibility NiCord + Unmanipulated Unmanip. Unmanip. 6/6 + 6/6 1 5/6 + 6/ /6 + 5/ /6 + 5/6 2 5/6 + 4/ /6 + 4/6 6 7
13 NiCord Graft Processing and Transplantation Schema CD133+ Fraction CD133- Fraction II. NiCord non-cultured Fraction (NF) Day -21: cryopreserved I. NiCord cultured fraction (CF) Day -21: Cultured with cytokines (FLT3, SCF, TPO, IL-6) + Nicotinamide (2.5mM) in cultured bags for 21± 2 days Day : Cells harvested, safety and quality tested Hand delivery to clinical site (18hr stability) CONDITIONING: Day -9 to TRANSPLANTATION I. NiCord CF II. NiCord NF III. Unmanipulated CBU FOLLOW UP MMF 6 18 Tacrolimus ARRIVAL OF NiCord NF TO CLINICAL SITE ARRIVAL OF NiCord CF TO CLINICAL SITE
14 CD34/kg (x1 6 ) TNC/kg (x1 7 ) TNC/kg (x1 7 ) Cell Dose Comparison Cryopreserved Cell Dose Infused Cell Dose A. B ( ) Unmanipulated ( 3-23) p=ns p=ns Unmanipulated ( ) NiCord (Pre-expansion) ( 5-34) NiCord (Preexpansion) C. CD3/kg (x1 6 ) CD34/kg (x1 6 ) E P=.3 ( ) Unmanipulated ( ) ( ) NiCord Cultured + Negative Fractions D. 6 ( ) 5 4 P< ( 3 48) Unmanipulated NiCord Cultured Fraction P=.9 ( ) (1-6 4) NiCord Cultured Fraction ( ) NiCord Negative Fraction 1 Unmanipulated NiCord Negative Fraction
15 WBC NiCord Engraftment Shortens Hospitalization Avg. hospitalization days Patients engrafted with NiCord : 23.5 (Day 14 discharge) Patients engrafted with the unmanipulated CBU: 4 (Day 31 discharge) Duke control cohort (n=17) average 42.2 (Day 29 discharge) Rapid PB WBC Reconstitution in Patients Engrafted with NiCord ANC>5 (median) ANC>5 (average) ANC>5 (median) NiCord engrafted (n=8) Unmanipulated engrafted (n=2) Duke controls (n=17) Days post transplantation
16 Whole Blood Chimerism
17 Myeloid Chimerism
18 T-cell Chimerism
19 White Blood Cell Count Durable Myeloid Hematopoiesis Neutrophil Engraftment (median day) NiCord; 11 Duke Controls; 25 P= Days Following Transplantation NiCord engrafted (n=8) Unmanipulated engrafted (n=2) Duke controls (n=17)
20 Platelet Count (x 1 3 ) Durable Platelet Hematopoiesis NiCord engrafted (n=7) Unmanipulated engrafted (n=2) Duke controls (n=17) Platelet Engraftment (median day) NiCord; 3 Duke Controls; Days Following Transplantation
21 Lymphoid Immune Reconstitution
22 Summary of Clinical Data 11 patients have received NiCord - containing dual UCB transplantation Median follow-up 1 months NiCord expanded HSC s outcompeted unmanipulated HSC s in 8 of 11 recipients NiCord engraftment is stable, hematopoiesis robust NiCord engraftment shortens hematopoietic recovery (Historical Controls) Neutrophils >5 (mean days); Platelets > 2K (mean days); No adverse events attributable to NiCord infusion Immunologic recovery comparable to dual UCBT controls Grade I/II agvhd; 4 patients No patients experienced grade III/IV agvhd Limited chronic GvHD; n=2 2yr overall survival; 68% 2yr progression-free survival; 65%
23 Potential Impact of ex vivo UCB Stem Cell Expansion 54y/o 8kg male, high risk AML in 1 st complete remission. No matched siblings. Umbilical cord blood transplantation proposed NMDP Cord Blood Search #1 4/6 (HLA-A,B, DRB1) matched; TNC-2.4 x1 7 /kg #2 4/6 (HLA-A,B, DRB1) matched; TNC-2.1 x1 7 /kg #3 5/6 (HLA-A,B, DRB1) matched; TNC-1.6 x1 7 /kg Option 1 (Current Standard) Unit selection: #1+#2; #1+#3 Myeloablative Conditioning UCB unit costs GvHD Hospital stay Hematopoietic Recovery Blood Product Utilization - - Option 2 Unit selection; #3 (Expanded) Myeloablative Conditioning UCB unit costs GvHD Hospital stay Hematopoietic Recovery Blood Product Utilization
24 Conclusions 1. Results provided justification for use of NiCord as a single umbilical cord blood graft for transplantation of adults with hematologic malignancies 1. Results provide justification for use of NiCord for umbilical cord blood transplantation in patients with hemoglobinopathies
25 Acknowledgements Duke Adult BMT Program Nelson Chao Joanne Kurtzberg Gwynn Long David Rizzieri Cristina Gasparetto Keith Sullivan John Chute Carolyn McDonald Janet Adcock Jackie McPherson Barbara Waters-Pick Loyola BMT Program Patrick Stiff Gamida-Cell Ltd. Tony Peled David Snyder Einat Galamidi Mirit Dekel Orna Srur-Kidron Yael Karasik Emmes Corp. Steven Wease Laura Devillier
NiCord Single Unit Expanded Umbilical Cord Blood Transplantation: Results of Phase I/II Trials
NiCord Single Unit Expanded Umbilical Cord Blood Transplantation: Results of Phase I/II Trials Mitchell E. Horwitz, MD Duke University Medical Center Duke Cancer Institute Adult Umbilical Cord Blood Transplantation
More informationValencia, 05/04/ #EBMT16
Transplantation of Ex vivo Expanded Umbilical Cord Blood (NiCord ) Results in Decreased Infection Burden and Hospital Length of Stay in the First 100 Days Sarah Anand, Samantha Thomas, Terry Hyslop, Kelly
More informationResults of Nicord Phase I II Trials and Plans for Phase III Trial
Results of Nicord Phase I II Trials and Plans for Phase III Trial Mitchell E. Horwitz, MD Duke Cancer Institute Duke University Medical Center June 10, 2016 Learning Objectives To provide an overview of
More informationIntroduction 6/7/2013. Umbilical Cord Blood Transplantation: Research and Clinical Applications
Umbilical Cord Blood Transplantation: Research and Clinical Applications Omar Aljitawi, MD Hematology/Oncology Pathology and Laboratory Medicine University of Kansas Medical Center April 27, 13 Introduction
More informationUmbilical Cord Blood Transplantation
Umbilical Cord Blood Transplantation Current Results John E. Wagner, M.D. Blood and Marrow Transplant Program and Stem Cell Institute University of Minnesota Donor Choices Unrelated Marrow/PBSC Results
More informationFebruary Company Overview. Curative Treatments for Cancer and Orphan Genetic Diseases
February 2017 Company Overview Curative Treatments for Cancer and Orphan Genetic Diseases Curative Treatments for Orphan Indications NiCord - a bone marrow transplantation treatment for patients with high
More informationRapid and Robust CD4+ and CD8+ T-, NK-, BTitel and Monocyte Cell Reconstitution after Nicotinamide-Expanded Cord Blood (NiCord) Transplantation
Rapid and Robust CD4+ and CD8+ T-, NK-, BTitel and Monocyte Cell Reconstitution after Nicotinamide-Expanded Cord Blood (NiCord) Subtitel Transplantation Boelens/Nierkens lab Jaap Jan Boelens, Central Immune
More informationThe National Marrow Donor Program. Graft Sources for Hematopoietic Cell Transplantation. Simon Bostic, URD Transplant Recipient
1988 199 1992 1994 1996 1998 2 22 24 26 28 21 212 214 216 218 Adult Donors Cord Blood Units The National Donor Program Graft Sources for Hematopoietic Cell Transplantation Dennis L. Confer, MD Chief Medical
More informationCompany Overview. January 2019
Company Overview January 2019 1 Disclaimer This Presentation includes certain projections and forward-looking statements as of the date of this Presentation provided by Gamida Cell Ltd (the Company ).
More informationHaploidentical Transplantation: The Answer to our Donor Problems? Mary M. Horowitz, MD, MS CIBMTR, Medical College of Wisconsin January 2017
Haploidentical Transplantation: The Answer to our Donor Problems? Mary M. Horowitz, MD, MS CIBMTR, Medical College of Wisconsin January 2017 Allogeneic Transplant Recipients in the US, by Donor Type 9000
More informationSECOND ANNUAL INTERNATIONAL UMBILICAL CORD BLOOD SYMPOSIUM
Biology of Blood and Marrow Transplantation 10:728-739 (2004) 2004 American Society for Blood and Marrow Transplantation 1083-8791/04/1010-0009$30.00/0 doi:10.1016/j.bbmt.2004.06.010 SECOND ANNUAL INTERNATIONAL
More informationHaplo vs Cord vs URD Debate
3rd Annual ASBMT Regional Conference for NPs, PAs and Fellows Haplo vs Cord vs URD Debate Claudio G. Brunstein Associate Professor University of Minnesota Medical School Take home message Finding a donor
More informationCompany Overview. October 2018
Company Overview October 2018 1 Disclaimer This presentation, the information contained herein and the materials accompanying it (collectively, this Presentation ) does not purport to contain all of the
More informationOne Day BMT Course by Thai Society of Hematology. Management of Graft Failure and Relapsed Diseases
One Day BMT Course by Thai Society of Hematology Management of Graft Failure and Relapsed Diseases Piya Rujkijyanont, MD Division of Hematology-Oncology Department of Pediatrics Phramongkutklao Hospital
More informationCord Blood Transplant. E. Gluckman Eurocord ESH-EBMT training course Vienna 2014
Cord Blood Transplant E. Gluckman Eurocord ESH-EBMT training course Vienna 2014 Background Since 1988, umbilical cord blood (CB) has been successfully used to treat children and adults needing stem cell
More informationReduced-intensity Conditioning Transplantation
Reduced-intensity Conditioning Transplantation Current Role and Future Prospect He Huang M.D., Ph.D. Bone Marrow Transplantation Center The First Affiliated Hospital Zhejiang University School of Medicine,
More informationHaploidentical Transplantation today: and the alternatives
Haploidentical Transplantation today: and the alternatives Daniel Weisdorf MD University of Minnesota February, 2013 No matched sib: where to look? URD donor requires close HLA matching and 3-12 weeks
More informationUNRELATED DONOR TRANSPLANTATION FOR SICKLE CELL DISEASE AN UPDATE
UNRELATED DONOR TRANSPLANTATION FOR SICKLE CELL DISEASE AN UPDATE Naynesh Kamani, M.D. Children s National Medical Center GW University School of Medicine Washington, DC SCD scope of problem in USA Commonest
More informationEBMT Complications and Quality of Life Working Party Educational Course
EBMT Complications and Quality of Life Working Party Educational Course Organisers: R. Duarte, G. Basak 23-24 October 2014, Warsaw, Poland #EBMT2014 www.ebmt.org EBMT Complications and Quality of Life
More informationWhat s a Transplant? What s not?
What s a Transplant? What s not? How to report the difference? Daniel Weisdorf MD University of Minnesota Anti-cancer effects of BMT or PBSCT [HSCT] Kill the cancer Save the patient Restore immunocompetence
More informationTHIRD ANNUAL INTERNATIONAL UMBILICAL CORD BLOOD TRANSPLANTATION SYMPOSIUM
Biology of Blood and Marrow Transplantation 11:921-927 (2005) 2005 American Society for Blood and Marrow Transplantation 1083-8791/05/1111-0009$30.00/0 doi:10.1016/j.bbmt.2005.08.028 THIRD ANNUAL INTERNATIONAL
More informationClinical Use of Umbilical Cord Blood Hematopoietic Stem Cells
Biology of Blood and Marrow Transplantation 12:34-41 (2006) 2006 American Society for Blood and Marrow Transplantation 1083-8791/06/1201-0107$32.00/0 doi:10.1016/j.bbmt.2005.09.006 Clinical Use of Umbilical
More informationDuchez P, Chevaleyre J, Dazey B, Vlaski M, Ivanovic Z.
EX-VIVO EXPANSION OF CORD BLOOD HEMATOPOIETIC STEM AND PROGENITOR CELLS FOR TRANSPLANTATION USING AN ANTIOXYDANT-SUPPLIED MEDIUM AND A CYTOKINE COCKTAIL INDUCING HYPOXIC-LIKE CELLULAR RESPONSE Duchez P,
More informationTransplantation - Challenges for the future. Dr Gordon Cook S t James s Institute of Oncology, Leeds Teaching Hospitals Trust
Transplantation - Challenges for the future Dr Gordon Cook S t James s Institute of Oncology, Leeds Teaching Hospitals Trust Bone Marrow Transplantation Timeline, 1957-2006 Appelbaum F. N Engl J Med 2007;357:1472-1475
More informationProtocol. This trial protocol has been provided by the authors to give readers additional information about their work.
Protocol This trial protocol has been provided by the authors to give readers additional information about their work. Protocol for: Wagner JE Jr, Eapen M, Carter S, et al. One-unit versus two-unit cord-blood
More informationThe question is not whether or not to deplete T-cells, but how to deplete which T-cells
The question is not whether or not to deplete T-cells, but how to deplete which T-cells CD34+ positive selection Negative Depletion of: CD3/CD19 TcRαβ/CD19 T-cell depletion: positive selection versus negative
More informationBone Marrow Transplantation and the Potential Role of Iomab-B
Bone Marrow Transplantation and the Potential Role of Iomab-B Hillard M. Lazarus, MD, FACP Professor of Medicine, Director of Novel Cell Therapy Case Western Reserve University 1 Hematopoietic Cell Transplantation
More informationWhat s new in Blood and Marrow Transplant? Saar Gill, MD PhD Jan 22, 2016
What s new in Blood and Marrow Transplant? Saar Gill, MD PhD Jan 22, 2016 Division of Hematology-Oncology University of Pennsylvania Perelman School of Medicine 1 Who should be transplanted and how? Updates
More informationThe future of HSCT. John Barrett, MD, NHBLI, NIH Bethesda MD
The future of HSCT John Barrett, MD, NHBLI, NIH Bethesda MD Transplants today Current approaches to improve SCT outcome Optimize stem cell dose and source BMT? PBSCT? Adjusting post transplant I/S to minimize
More informationPotency & Release Testing for unrelated donor cord blood units
Potency & Release Testing for unrelated donor cord blood units Joanne Kurtzberg, MD Robertson Cell and Translational Therapy Program Pediatric Blood and Marrow Transplant Program Carolinas Cord Blood Bank
More informationIntroduction to Clinical Hematopoietic Cell Transplantation (HCT) George Chen, MD Thursday, May 03, 2018
Introduction to Clinical Hematopoietic Cell Transplantation (HCT) George Chen, MD Thursday, May 03, 2018 The transfer of hematopoietic progenitor and stem cells for therapeutic purposes Hematopoietic Cell
More informationMUD HSCT as first line Treatment in Idiopathic SAA. Dr Sujith Samarasinghe Great Ormond Street Hospital for Children, London, UK
MUD HSCT as first line Treatment in Idiopathic SAA Dr Sujith Samarasinghe Great Ormond Street Hospital for Children, London, UK No Financial Disclosures Guidelines for management of aplastic anaemia British
More informationSymposium Summary Fourth Annual International Umbilical Cord Blood Transplantation Symposium, Los Angeles, California, May 19-20, 2006
Biology of Blood and Marrow Transplantation 12:1206-1217 (2006) 2006 American Society for Blood and Marrow Transplantation 1083-8791/06/1211-0001$32.00/0 doi:10.1016/j.bbmt.2006.08.030 Symposium Summary
More informationHaploidentical Donor Transplants: Outcomes and Comparison to Other. Paul V. O Donnell BSBMT Education Day London 12 October 2011
Haploidentical Donor Transplants: Outcomes and Comparison to Other Donor Types Paul V. O Donnell BSBMT Education Day London 12 October 2011 Clinical Problem: Identification of a Donor for Allogeneic Transplantation
More informationStem Cell Transplantation (Cord Blood Transplants)
Stem Cell Transplantation (Cord Blood Transplants) Nelson J. Chao, Stephen G. Emerson, and Kenneth I. Weinberg Allogeneic stem cell transplantation is an accepted treatment modality for selected malignant
More informationPost Transplant Management for Sickle Cell. Title
Post Transplant Management for Sickle Cell Title Kimberly Kasow, DO October 14, 2016 Thank you for this opportunity to present this information I have no financial interests to disclose. Goal of Transplant
More informationNeutrophil Recovery: The. Posttransplant Recovery. Bus11_1.ppt
Neutrophil Recovery: The First Step in Posttransplant Recovery No conflicts of interest to disclose Bus11_1.ppt Blood is Made in the Bone Marrow Blood Stem Cell Pre-B White cells B Lymphocyte T Lymphocyte
More informationMyeloablative and Reduced Intensity Conditioning for HSCT Annalisa Ruggeri, MD, Hôpital Saint Antoine Eurocord- Hôpital Saint Louis, Paris
Myeloablative and Reduced Intensity Conditioning for HSCT Annalisa Ruggeri, MD, Hôpital Saint Antoine Eurocord- Hôpital Saint Louis, Paris 18th ESH - EBMT Training Course on HSCT 8-10 May 2014, Vienna,
More informationBiol Blood Marrow Transplant 18: (2012) Ó 2012 American Society for Blood and Marrow Transplantation
Immune Recovery in Adult Patients after Myeloablative Dual Umbilical Cord Blood, Matched Sibling, and Matched Unrelated Donor Hematopoietic Cell Transplantation Junya Kanda, 1,6 Lun-Wei Chiou, 1 Paul Szabolcs,
More informationHigh dose cyclophosphamide in HLAhaploidentical
High dose cyclophosphamide in HLAhaploidentical stem cell transplantation Ephraim J. Fuchs, M.D., M.B.A. Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins fuchsep@jhmi.edu Alternative Donor Transplantation:
More informationHematopoietic Stem Cell Therapy
Hematopoietic Stem Cell Therapy Grace Totoe, MBChB, SBB CME August 2012 Accra-Ghana Hematopoietic Stem Cells Cells capable of self renewal and differentiation into all blood cell lineages Objectives Historical
More informationStem Cell Transplantation for Severe Aplastic Anemia
Number of Transplants 10/24/2011 Stem Cell Transplantation for Severe Aplastic Anemia Claudio Anasetti, MD Professor of Oncology and Medicine Chair, Blood and Marrow Transplant Dpt Moffitt Cancer Center
More informationCHAPTER 2 PROTOCOL DESIGN
CHAPTER 2 PROTOCOL DESIGN CHAPTER 2 PROTOCOL DESIGN 2.1 ELIGIBILITY CRITERIA Participants fulfilling the following criteria will be eligible for enrollment in the protocol: 1. Participant is diagnosed
More informationStem Cell Transplantation
Stem Cell Transplantation Evelyne Willems Centre Hospitalier Universitaire, ULg, Liège Post-ASH meeting, January 11, 2012, Brussels Plan 1. Select the patient: validation of HCT-CI 2. Select the donor
More informationCompany Overview. Investor Presentation. August 2018
Company Overview Investor Presentation August 2018 1 Notice to Investors The sole purpose of this Presentation (this Presentation ) is to assist recipients in deciding whether to proceed with a further
More informationIntroduction to Hematopoietic Stem Cell Transplantation
Faculty Disclosures Introduction to Hematopoietic Stem Cell Transplantation Nothing to disclose Jeanne McCarthy-Kaiser, PharmD, BCOP Clinical Pharmacist, Autologous Stem Cell Transplant/Long- Term Follow-Up
More informationISCT Workshop #7 Perspectives in Cell Selection Immunomagnetic Selection
ISCT Workshop #7 Perspectives in Cell Selection Immunomagnetic Selection Carolyn A. Keever-Taylor, PhD Medical College of Wisconsin June 7, 2012 History of Available Devices CellPro CEPRATE Avidin/Biotin
More informationUKALL14. Non-Myeloablative Conditioning Regimen (1/1) Date started (dd/mm/yyyy) (Day 7) Weight (kg) BSA (m 2 )
Non-Myeloablative Conditioning Regimen (1/1) started (dd/mm/yyyy) (Day 7) BSA (m 2 ) Weight (kg) Please enter the daily dose given in the table below: Day Fludarabine (mg) Melphalan (mg) Alemtuzumab (mg)
More informationDr.PSRK.Sastry MD, ECMO
Peripheral blood stem cell transplantation (Haematopoietic stem cell transplantation) Dr.PSRK.Sastry MD, ECMO Consultant, Medical Oncology Kokilaben Dhirubhai Ambani Hospital Normal hematopoiesis Historical
More informationSummary of Changes BMT CTN 1101 Version 7.0 to 8.0 Dated: January 18, Original text: Changed to: Rationale
BMT CTN 1101 RIC ducb vs. Haplo Page 1 of 10 Date: January 20, 2017 Summary of Changes BMT CTN 1101 Version 7.0 to 8.0 Dated: January 18, 2017 A Multi-Center, Phase III, Randomized Trial of Reduced Intensity(RIC)
More informationObjectives. What is Aplastic Anemia. SAA 101: An Introductory Course to Severe Aplastic Anemia
SAA 101: An Introductory Course to Severe Aplastic Anemia David A. Margolis, MD Professor of Pediatrics/Medical College of Wisconsin Program Director/ Children s Hospital of Wisconsin BMT Program Objectives
More informationCD34+ Cells: A Comparison of Stem and Progenitor Cells in Cord Blood, Peripheral Blood, and the Bone Marrow
White Paper September 2016 CD34+ Cells: A Comparison of Stem and Progenitor Cells in Cord Blood, Peripheral Blood, and the Bone Marrow Lily C. Trajman, PhD Introduction: Hematopoietic Stem Cells (HSCs)
More informationTolerance Induction in Transplantation
Tolerance Induction in Transplantation Reza F. Saidi, MD, FACS, FICS Assistant Professor of Surgery Division of Organ Transplantation Department of Surgery University of Massachusetts Medical School Percent
More informationReduced-Intensity Conditioning Stem Cell Transplantation: Comparison of Double Umbilical Cord Blood and Unrelated Donor Grafts
Reduced-Intensity Conditioning Stem Cell Transplantation: Comparison of Double Umbilical Cord Blood and Unrelated Donor Grafts Yi-Bin Chen, 1 Julie Aldridge, 2 Haesook T. Kim, 2 Karen K. Ballen, 1 Corey
More informationRob Wynn RMCH & University of Manchester, UK. HCT in Children
Rob Wynn RMCH & University of Manchester, UK HCT in Children Summary Indications for HCT in children Donor selection for Paediatric HCT Using cords Achieving engraftment in HCT Conditioning Immune action
More information5/9/2018. Bone marrow failure diseases (aplastic anemia) can be cured by providing a source of new marrow
5/9/2018 or Stem Cell Harvest Where we are now, and What s Coming AA MDS International Foundation Indianapolis IN Luke Akard MD May 19, 2018 Infusion Transplant Conditioning Treatment 2-7 days STEM CELL
More informationBack to the Future: The Resurgence of Bone Marrow??
Back to the Future: The Resurgence of Bone Marrow?? Thomas Spitzer, MD Director. Bone Marrow Transplant Program Massachusetts General Hospital Professor of Medicine, Harvard Medical School Bone Marrow
More informationMATCHMAKER, MATCHMAKER, MAKE ME A MATCH, FIND ME A MISMATCHED TRANSPLANT TO CATCH
MATCHMAKER, MATCHMAKER, MAKE ME A MATCH, FIND ME A MISMATCHED TRANSPLANT TO CATCH TEJASWINI M. DHAWALE, M.D. HEME FELLOWS CONFERENCE NOVEMBER 08, 2013 CASE PRESENTATION 51 yo M with history of MDS (unilinear
More informationSamples Available for Recipient Only. Samples Available for Recipient and Donor
Unrelated HCT Research Sample Inventory - Summary for First Allogeneic Transplants in CRF and TED with biospecimens available through the CIBMTR Repository stratified by availability of paired samples,
More informationRole of NMDP Repository in the Evolution of HLA Matching and Typing for Unrelated Donor HCT
Role of NMDP Repository in the Evolution of HLA Matching and Typing for Unrelated Donor HCT Stephen Spellman, MBS Director, Immunobiology and Observational Research Assistant Scientific Director CIBMTR,
More informationMUD SCT. Pimjai Niparuck Division of Hematology, Department of Medicine Ramathibodi Hospital, Mahidol University
MUD SCT Pimjai Niparuck Division of Hematology, Department of Medicine Ramathibodi Hospital, Mahidol University Outlines Optimal match criteria for unrelated adult donors Role of ATG in MUD-SCT Post-transplant
More informationCurrent Status of Haploidentical Hematopoietic Stem Cell Transplantation
Current Status of Haploidentical Hematopoietic Stem Cell Transplantation Annalisa Ruggeri, MD, PhD Hematology and BMT Unit Hôpital Saint Antoine, Paris, France #EBMTITC16 www.ebmt.org Hematopoietic SCT
More informationSamples Available for Recipient and Donor
Unrelated HCT Research Sample Inventory - Summary for First Allogeneic Transplants in CRF and TED with biospecimens available through the CIBMTR Repository stratified by availability of paired samples,
More informationSamples Available for Recipient Only. Samples Available for Recipient and Donor
Unrelated HCT Research Sample Inventory - Summary for First Allogeneic Transplants in CRF and TED with biospecimens available through the CIBMTR Repository stratified by availability of paired samples,
More informationStem cell transplantation for haemoglobinopathies. Dr P J Darbyshire Birmingham Childrens Hospital
Stem cell transplantation for haemoglobinopathies Dr P J Darbyshire Birmingham Childrens Hospital Survival by Cohort of Birth (N=977) 1.00 85-97 80-84 75-79 70-74 0.75 Survival Probability 0.50 0.25 P
More informationRelated haploidentical donors versus matched unrelated donors
Related haploidentical donors versus matched unrelated donors Bronwen Shaw, MD PhD Professor of Medicine, MCW Senior Scientific Director, CIBMTR Definition Matched Unrelated donor Refers to HLA matching
More informationAcknowledgements. Department of Hematological Malignancy and Cellular Therapy, University of Kansas Medical Center
The Addition of Extracorporeal Photopheresis (ECP) to Tacrolimus and Methotrexate to Prevent Acute and Chronic Graft- Versus Host Disease in Myeloablative Hematopoietic Cell Transplant (HCT) Anthony Accurso,
More informationMyeloproliferative Disorders - D Savage - 9 Jan 2002
Disease Usual phenotype acute leukemia precursor chronic leukemia low grade lymphoma myeloma differentiated Total WBC > 60 leukemoid reaction acute leukemia Blast Pro Myel Meta Band Seg Lymph 0 0 0 2
More informationOriginal article. Department of Pediatrics, Samsung Medical Center, Sungkyunkwan University School of Medicine, Seoul, 2
Original article http://dx.doi.org/1.3345/kjp.212.55.3.93 Korean J Pediatr 212;55(3):93-99 Hematopoietic stem cell transplantation in children with acute leukemia: similar outcomes in recipients of umbilical
More informationUmbilical cord blood transplantation from unrelated donors in patients with Philadelphia chromosome-positive acute lymphoblastic leukemia
Stem Cell Transplantation SUPPLEMENTARY APPENDIX Umbilical cord blood transplantation from unrelated donors in patients with Philadelphia chromosome-positive acute lymphoblastic leukemia José Luis Piñana,
More informationExperience of patients transplanted with naïve T cell depleted stem cell graft in CMUH
Experience of patients transplanted with naïve T cell depleted stem cell graft in CMUH Tzu-Ting Chen, Wen-Jyi Lo, Chiao-Lin Lin, Ching-Chan Lin, Li-Yuan Bai, Supeng Yeh, Chang-Fang Chiu Hematology and
More informationUnderstanding the role of ex vivo T cell depletion
Prevention of graftversus-host disease (GVHD) Understanding the role of ex vivo T cell depletion Information for patients undergoing allogeneic stem cell transplantation in AML and their families 2 This
More informationWhat the Transfusion Scientist should know about Stem Cells. Dr Claire Wiggins BBTS September 2017
What the Transfusion Scientist should know about Stem Cells Dr Claire Wiggins BBTS September 2017 What is a stem cell? Undifferentiated cell Can divide and self renew for long periods Differentiate into
More informationHaploidentical Transplants for Lymphoma. Andrea Bacigalupo Universita Cattolica Policlinico Gemelli Roma - Italy
Haploidentical Transplants for Lymphoma Andrea Bacigalupo Universita Cattolica Policlinico Gemelli Roma - Italy HODGKIN NON HODGKIN Non Myelo Ablative Regimen Luznik L et al BBMT 2008 Comparison of Outcomes
More informationHematopoietic Stem Cells, Stem Cell Processing, and Transplantation
Hematopoietic Stem Cells, Stem Cell Processing, and Joseph (Yossi) Schwartz, M irector, Hemotherapy and Stem Cell Processing Facility Bone Marrow Can Cure: Leukemia Lymphoma Multiple Myeloma Genetic iseases:
More informationHome based hematopoietic stem cell transplantation
Home based hematopoietic stem cell transplantation Anthony Sung, MD Chao Lab Division of Hematologic Malignancies and Cellular Therapy 2 nd Annual Scientific Retreat November 14, 2014 Background Hematopoietic
More informationReview of Aplastic Anemia Guidelines. Seiji Kojima MD. PhD.
Review of Aplastic Anemia Guidelines Seiji Kojima MD. PhD. Department of Pediatrics Nagoya University Graduate School of Medicine Chairman of the Severe Aplastic Anemia Working Party Asia-Pacific Blood
More informationCord Blood Stem Cell Banking and Transplantation
Cord Blood Stem Cell Banking and Transplantation JOHN W. ADAMSON New York Blood Center, New York, New York, USA Key Words. Cord blood Stem cells Cord blood banking Cord blood transplantation. Cord blood.stern
More informationHematopoietic Stem Cell Transplantation for Fanconi Anemia
Hematopoietic Stem Cell Transplantation for Fanconi Anemia John E. Wagner, M.D. Blood and Marrow Transplant Program University of Minnesota Cell Therapy for Pediatric Diseases NHLBI PACT Workshop 14 15
More information3.1 Clinical safety of chimeric or humanized anti-cd25 (ch/anti-cd25)
3 Results 3.1 Clinical safety of chimeric or humanized anti-cd25 (ch/anti-cd25) Five infusions of monoclonal IL-2 receptor antibody (anti-cd25) were planned according to protocol between day 0 and day
More informationUmbilical Cord Blood Hematopoietic Stem Cell Transplantation, an Alternative to Bone Marrow
Umbilical Cord Blood Hematopoietic Stem Cell Transplantation, an Alternative to Bone Marrow Mortada Salman Najem (Corresponding author) Department of Biology and Biotechnology, Worcester Polytechnic Institute
More informationClinical Policy: Donor Lymphocyte Infusion
Clinical Policy: Reference Number: PA.CP.MP.101 Effective Date: 01/18 Last Review Date: 11/16 Coding Implications Revision Log This policy describes the medical necessity requirements for a donor lymphocyte
More informationKEY WORDS Fludarabine Sickle cell disease Allogeneic Pharmacokinetic Transplantation Renal failure
Biology of Blood and Marrow Transplantation 13:1422-1426 (2007) 2007 American Society for Blood and Marrow Transplantation 1083-8791/07/1312-0001$32.00/0 doi:10.1016/j.bbmt.2007.08.050 Fludarabine-Based
More informationHaematopoietic stem cells
Haematopoietic stem cells Neil P. Rodrigues, DPhil NIH Centre for Biomedical Research Excellence in Stem Cell Biology Boston University School of Medicine neil.rodrigues@imm.ox.ac.uk Haematopoiesis: An
More informationEmerging Considerations for Cord Blood Transplantation. Cord Blood Unit Panel Discussion
Emerging Considerations for Cord Blood Transplantation Juliet Barker, MBBS Joanne Kurtzberg, MD Koen van Besien, MD, PhD Moderators: Janelle Olson, PhD, CHTC Elizabeth Beduhn, CHTC Cord Blood Unit Panel
More informationOne-Unit versus Two-Unit Cord-Blood Transplantation for Hematologic Cancers
The new england journal of medicine Original Article One-Unit versus Two-Unit Cord-Blood Transplantation for Hematologic Cancers John E. Wagner, Jr., M.D., Mary Eapen, M.B., B.S., Shelly Carter, D.Sc.,
More informationNIH Public Access Author Manuscript Lancet Oncol. Author manuscript; available in PMC 2011 August 29.
NIH Public Access Author Manuscript Published in final edited form as: Lancet Oncol. 2010 July ; 11(7): 653 660. doi:10.1016/s1470-2045(10)70127-3. Effect of Graft Source on Unrelated Donor Haemopoietic
More informationCord blood transplantation and stem cell regenerative potential
Experimental Hematology 2011;39:393 412 Cord blood transplantation and stem cell regenerative potential Yanling Liao a, Mark B. Geyer a, Albert J. Yang a, and Mitchell S. Cairo a,b,c a Department of Pediatrics,
More informationHematopoietic Stem Cell Transplant in Sickle Cell Disease- An update
Hematopoietic Stem Cell Transplant in Sickle Cell Disease- An update Dr Chirag A Shah Diplomate American Board of Hematology and Medical Oncology Director, Dept of Hemato-Oncology and Stem Cell Transplant
More informationDr. Joseph McGuirk Professor of Medicine, BMT Medical Director, Interim Director, Division of Hematology/Oncology
Advances in Autologous and Allogeneic Stem Cell Transplantation Dr. Joseph McGuirk Professor of Medicine, BMT Medical Director, Interim Director, Division of Hematology/Oncology April 12, 2014 Disclosures
More informationRIC in Allogeneic Stem Cell Transplantation
RIC in Allogeneic Stem Cell Transplantation Rainer Storb, MD Fred Hutchinson Cancer Research Center and the University of Washington School of Medicine Seattle, WA Disclosure Grant Support: NIH grants
More informationKEY WORDS: Nonmyeloablative, Umbilical cord blood, Lymphoid malignancies
Promising Progression-Free Survival for Patients Low and Intermediate Grade Lymphoid Malignancies after Nonmyeloablative Umbilical Cord Blood Transplantation Claudio G. Brunstein, 1,2 Susana Cantero, 1
More informationHematopoietic Cell Transplantation in Bone Marrow Failure Syndromes
Hematopoietic Cell Transplantation in Bone Marrow Failure Syndromes The The 44 th th WBMT WBMT SYMPOSIUM 2017 2017 Riyadh, Saudi Saudi Arabia Arabia Mouhab Mouhab Ayas, Ayas, MD MD Department of Pediatric
More informationExpanding Uses of Umbilical Cord Blood: Duke Experience. Vinod K. Prasad, MD, MRCP (London) PBMT Division, Duke University Medical Center
Expanding Uses of Umbilical ord Blood: Duke Experience Vinod K. Prasad, MD, MRP (London) PBMT Division, Duke University Medical enter UBT - Record Umbilical cord blood contains sufficient numbers of stem
More informationShall young patients with severe aplastic anemia without donors receive BMT from alternative source of HCT? Elias Hallack Atta, MD, PhD
Shall young patients with severe aplastic anemia without donors receive BMT from alternative source of HCT? Elias Hallack Atta, MD, PhD Declaração de Conflito de Interesse Declaro que possuo conflito de
More informationSupplemental Table 1 Multivariate analysis of neutrophil and platelet
Transplant using vs. HLA 1-AG mismatched RD Supplemental Table 1 Multivariate analysis of neutrophil and platelet engraftment Variable Neutrophil engraftment* Platelet engraftment HR (95% CI) P value HR
More informationCONSIDERATIONS IN DESIGNING ACUTE GVHD PREVENTION TRIALS: Patient Selection, Concomitant Treatments, Selecting and Assessing Endpoints
CONSIDERATIONS IN DESIGNING ACUTE GVHD PREVENTION TRIALS: Patient Selection, Concomitant Treatments, Selecting and Assessing Endpoints CENTER FOR INTERNATIONAL BLOOD AND MARROW TRANSPLANT RESEARCH Potential
More informationHOVON 106 DOUBLE UCBT Version3 mei 2010
A phase II study to assess engraftment and engraftment kinetics after double cord blood transplantation with a reduced-intensity conditioning regimen in patients eligible for allogeneic stem cell transplantation
More informationDedicated to Gordon. Stem Cell Transplantation: The Journey
Dedicated to Gordon Stem Cell Transplantation: The Journey 1949 Jacobson et al: Radioprotection by lead shielding of the spleen of a lethally irradiated animal 1951 Lorenz et al: Radiation protection
More informationAllogeneic Stem Cell Transplantation with Peripheral Blood Stem Cells Mobilized by Pegylated G-CSF
Biology of Blood and Marrow Transplantation 12:63-67 (26) 26 American Society for Blood and Marrow Transplantation 183-8791/6/126-2$32./ doi:1.116/j.bbmt.26.3.1 Allogeneic Stem Cell Transplantation with
More information