UMBILICAL CORD BLOOD STEM CELLS EXPANDED IN THE PRESENCE OF NICOTINAMIDE (NICORD) PROVIDE LONG TERM MULITI-LINEAGE ENGRAFTMENT

Size: px
Start display at page:

Download "UMBILICAL CORD BLOOD STEM CELLS EXPANDED IN THE PRESENCE OF NICOTINAMIDE (NICORD) PROVIDE LONG TERM MULITI-LINEAGE ENGRAFTMENT"

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 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 information

Valencia, 05/04/ #EBMT16

Valencia, 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 information

Results 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 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 information

Introduction 6/7/2013. Umbilical Cord Blood Transplantation: Research and Clinical Applications

Introduction 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 information

Umbilical Cord Blood Transplantation

Umbilical 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 information

February Company Overview. Curative Treatments for Cancer and Orphan Genetic Diseases

February 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 information

Rapid 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) 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 information

The National Marrow Donor Program. Graft Sources for Hematopoietic Cell Transplantation. Simon Bostic, URD Transplant Recipient

The 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 information

Company Overview. January 2019

Company 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 information

Haploidentical 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 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 information

SECOND ANNUAL INTERNATIONAL UMBILICAL CORD BLOOD SYMPOSIUM

SECOND 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 information

Haplo vs Cord vs URD Debate

Haplo 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 information

Company Overview. October 2018

Company 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 information

One 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 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 information

Cord Blood Transplant. E. Gluckman Eurocord ESH-EBMT training course Vienna 2014

Cord 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 information

Reduced-intensity Conditioning Transplantation

Reduced-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 information

Haploidentical Transplantation today: and the alternatives

Haploidentical 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 information

UNRELATED DONOR TRANSPLANTATION FOR SICKLE CELL DISEASE AN UPDATE

UNRELATED 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 information

EBMT Complications and Quality of Life Working Party Educational Course

EBMT 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 information

What s a Transplant? What s not?

What 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 information

THIRD ANNUAL INTERNATIONAL UMBILICAL CORD BLOOD TRANSPLANTATION SYMPOSIUM

THIRD 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 information

Clinical Use of Umbilical Cord Blood Hematopoietic Stem Cells

Clinical 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 information

Duchez P, Chevaleyre J, Dazey B, Vlaski M, Ivanovic Z.

Duchez 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 information

Transplantation - 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 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 information

Protocol. 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 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 information

The 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 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 information

Bone Marrow Transplantation and the Potential Role of Iomab-B

Bone 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 information

What 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 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 information

The future of HSCT. John Barrett, MD, NHBLI, NIH Bethesda MD

The 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 information

Potency & Release Testing for unrelated donor cord blood units

Potency & 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 information

Introduction 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 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 information

MUD 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 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 information

Symposium Summary Fourth Annual International Umbilical Cord Blood Transplantation Symposium, Los Angeles, California, May 19-20, 2006

Symposium 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 information

Haploidentical 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. 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 information

Stem Cell Transplantation (Cord Blood Transplants)

Stem 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 information

Post Transplant Management for Sickle Cell. Title

Post 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 information

Neutrophil Recovery: The. Posttransplant Recovery. Bus11_1.ppt

Neutrophil 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 information

Myeloablative 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 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 information

Biol Blood Marrow Transplant 18: (2012) Ó 2012 American Society for Blood and Marrow Transplantation

Biol 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 information

High dose cyclophosphamide in HLAhaploidentical

High 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 information

Hematopoietic Stem Cell Therapy

Hematopoietic 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 information

Stem Cell Transplantation for Severe Aplastic Anemia

Stem 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 information

CHAPTER 2 PROTOCOL DESIGN

CHAPTER 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 information

Stem Cell Transplantation

Stem 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 information

Company Overview. Investor Presentation. August 2018

Company 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 information

Introduction to Hematopoietic Stem Cell Transplantation

Introduction 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 information

ISCT Workshop #7 Perspectives in Cell Selection Immunomagnetic Selection

ISCT 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 information

UKALL14. Non-Myeloablative Conditioning Regimen (1/1) Date started (dd/mm/yyyy) (Day 7) Weight (kg) BSA (m 2 )

UKALL14. 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 information

Dr.PSRK.Sastry MD, ECMO

Dr.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 information

Summary of Changes BMT CTN 1101 Version 7.0 to 8.0 Dated: January 18, Original text: Changed to: Rationale

Summary 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 information

Objectives. What is Aplastic Anemia. SAA 101: An Introductory Course to Severe Aplastic Anemia

Objectives. 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 information

CD34+ Cells: A Comparison of Stem and Progenitor Cells in Cord Blood, Peripheral Blood, and the Bone Marrow

CD34+ 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 information

Tolerance Induction in Transplantation

Tolerance 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 information

Reduced-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 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 information

Rob Wynn RMCH & University of Manchester, UK. HCT in Children

Rob 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 information

5/9/2018. Bone marrow failure diseases (aplastic anemia) can be cured by providing a source of new marrow

5/9/2018. 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 information

Back to the Future: The Resurgence of Bone Marrow??

Back 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 information

MATCHMAKER, 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 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 information

Samples Available for Recipient Only. Samples Available for Recipient and Donor

Samples 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 information

Role 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 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 information

MUD 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 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 information

Current Status of Haploidentical Hematopoietic Stem Cell Transplantation

Current 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 information

Samples Available for Recipient and Donor

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 information

Samples Available for Recipient Only. Samples Available for Recipient and Donor

Samples 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 information

Stem cell transplantation for haemoglobinopathies. Dr P J Darbyshire Birmingham Childrens Hospital

Stem 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 information

Related haploidentical donors versus matched unrelated donors

Related 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 information

Acknowledgements. Department of Hematological Malignancy and Cellular Therapy, University of Kansas Medical Center

Acknowledgements. 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 information

Myeloproliferative Disorders - D Savage - 9 Jan 2002

Myeloproliferative 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 information

Original article. Department of Pediatrics, Samsung Medical Center, Sungkyunkwan University School of Medicine, Seoul, 2

Original 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 information

Umbilical cord blood transplantation from unrelated donors in patients with Philadelphia chromosome-positive acute lymphoblastic leukemia

Umbilical 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 information

Experience 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 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 information

Understanding the role of ex vivo T cell depletion

Understanding 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 information

What 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 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 information

Haploidentical Transplants for Lymphoma. Andrea Bacigalupo Universita Cattolica Policlinico Gemelli Roma - Italy

Haploidentical 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 information

Hematopoietic Stem Cells, Stem Cell Processing, and Transplantation

Hematopoietic 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 information

Home based hematopoietic stem cell transplantation

Home 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 information

Review of Aplastic Anemia Guidelines. Seiji Kojima MD. PhD.

Review 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 information

Cord Blood Stem Cell Banking and Transplantation

Cord 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 information

Hematopoietic Stem Cell Transplantation for Fanconi Anemia

Hematopoietic 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 information

3.1 Clinical safety of chimeric or humanized anti-cd25 (ch/anti-cd25)

3.1 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 information

Umbilical Cord Blood Hematopoietic Stem Cell Transplantation, an Alternative to Bone Marrow

Umbilical 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 information

Clinical Policy: Donor Lymphocyte Infusion

Clinical 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 information

KEY WORDS Fludarabine Sickle cell disease Allogeneic Pharmacokinetic Transplantation Renal failure

KEY 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 information

Haematopoietic stem cells

Haematopoietic 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 information

Emerging Considerations for Cord Blood Transplantation. Cord Blood Unit Panel Discussion

Emerging 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 information

One-Unit versus Two-Unit Cord-Blood Transplantation for Hematologic Cancers

One-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 information

NIH Public Access Author Manuscript Lancet Oncol. Author manuscript; available in PMC 2011 August 29.

NIH 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 information

Cord blood transplantation and stem cell regenerative potential

Cord 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 information

Hematopoietic Stem Cell Transplant in Sickle Cell Disease- An update

Hematopoietic 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 information

Dr. Joseph McGuirk Professor of Medicine, BMT Medical Director, Interim Director, Division of Hematology/Oncology

Dr. 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 information

RIC in Allogeneic Stem Cell Transplantation

RIC 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 information

KEY WORDS: Nonmyeloablative, Umbilical cord blood, Lymphoid malignancies

KEY 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 information

Hematopoietic Cell Transplantation in Bone Marrow Failure Syndromes

Hematopoietic 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 information

Expanding Uses of Umbilical Cord Blood: Duke Experience. Vinod K. Prasad, MD, MRCP (London) PBMT Division, Duke University Medical Center

Expanding 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 information

Shall 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 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 information

Supplemental Table 1 Multivariate analysis of neutrophil and platelet

Supplemental 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 information

CONSIDERATIONS 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 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 information

HOVON 106 DOUBLE UCBT Version3 mei 2010

HOVON 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 information

Dedicated to Gordon. Stem Cell Transplantation: The Journey

Dedicated 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 information

Allogeneic Stem Cell Transplantation with Peripheral Blood Stem Cells Mobilized by Pegylated G-CSF

Allogeneic 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