Transplant and Cellular Therapy Options for Non-Malignant Hematologic Disorders

Therapies available for the treatment of non-malignant hematologic disorders have significantly improved survival rates, but many are unaware of new and innovative options available to their patients. The goal of this interactive live symposium is to review and discuss cutting edge treatments for pediatric and adult patients. Relevant clinical cases and treatment modalities to be covered include:

• Stem cell transplant and/or gene therapy options for non-malignant disorders in pediatric and adult patients including:
• Hemoglobinopathies (Sickle cell disease)
• Congenital and acquired immunodeficiencies (Primary immunodeficiencies, SCID,  HIV)
• Inherited and acquired bone marrow failure disorders (Fanconi anemia, aplastic anemia)
• Disease specific expert panels addressing integration of care, innovative therapies and recent changes in transplant and cellular therapy options
• Optimized transplant preparative regimens including individualized conditioning, chemotherapy and radiation sparing conditioning regimens
• Integration of care with referring physicians/centers for complex care of non-malignant disorders

This live virtual program will include both didactic and interactive sessions.

Who Should Attend

1. Recall new and cutting edge treatment options for patients
2. Identify institutions and opportunities for collaboration that will improve patient care and access
3. Determine differences in potential treatment options
4. Recommend various interprofessional teams and interdisciplinary services for patient care support.

Registration for this course is complimentary for all attendees. However, you must complete registration through the 'Register' tab above in order to attend. Access details will be emailed to all registered attendees before the start of the course. If you need to cancel your registration, please notify [email protected] at least seven (7) days prior to the start of the course. 

Online registration is available on the MSK CME Portal until 7:00 AM on October 15, 2021. If you wish to register after this date, please email [email protected] and we will ensure your registration is processed as well as access instructions are emailed to you prior to the start of the course.

Please note that MSK CME reserves the right to cancel or postpone any course due to unforeseen circumstances. In the unlikely event we must cancel or postpone this course, you will be notified via email from MSK CME [email protected].

Memorial Sloan Kettering Cancer Center is accredited by the Accreditation Council for Continuing Medical Education to provide continuing medical education for physicians.

AMA Credit Designation Statement

Memorial Sloan Kettering Cancer Center designates this live activity for a maximum of 9.25 AMA PRA Category 1 Credits™. Physicians should claim only the credit commensurate with the extent of their participation in the activity.

ABIM & ABP MOC Recognition Statement

Successful completion of this CME activity, which includes participation in the evaluation component, enables the participant to earn up to:

• 9.25 Medical Knowledge MOC points in the American Board of Internal Medicine's (ABIM) Maintenance of Certification (MOC) program; and
• 9.25 MOC points in the American Board of Pediatrics’ (ABP) Maintenance of Certification (MOC) program.

It is the CME activity provider's responsibility to submit participant completion information to ACCME for the purpose of granting ABIM or ABP MOC credit.

MSK Course Directors

Maria Cancio, MD
Pediatric Hematologic Oncologist

Rekha Parameswaran, MD
Hematologic Oncologist

MSK Course Planning Committee 

Jaap Jan Boelens, MD
Chief, Pediatric Stem Cell Transplantation and Cellular Therapies Service
Richard J. O’Reilly Chair in Honor of Kevin Zoltan Merszei

Christina Cho, MD
Medical Oncologist

 
Sergio Giralt, MD
Deputy Division Head, Division of Hematologic Malignancies
Melvin Berlin Family Chair in Multiple Myeloma

Jodi V. Mones, MD 
Hematologist

 
Miguel-Angel Perales, MD
Chief, Adult Bone Marrow Transplant Service

 
Roni Tamari, MD
Hematologic Oncologist

Invited Course Faculty 

Alice Bertaina, MD, PhD
Associate Professor of Pediatrics, Stem Cell Transplantation
Stanford University
Stanford, CA

Monica Bhatia, MD
Director, Pediatric Stem Cell Transplant Program
Columbia University Irving Medical Center
New York, NY

Marc Bierings, MD, PhD
Pediatric Oncologist, Head of SCT Department
Prinses Maxima Centrum
Utrecht, Netherlands

Catherine Bollard, MD, MBChB
Director, Center for Cancer and Immunology
Director, Program for Cell Enhancement and Technologies for Immunotherapy
Children’s National
Washington, DC

Carmem Bonfim, MD, PhD 
Director, Pediatric Blood and Marrow Transplantation Program
Hospital Pequeno Principe
Professor, Federal University of Parana
Curitiba, Brazil

Selim Corbacioglu, MD
Associate Professor
Chair of Pediatric Hematology, Oncology, and Stem Cell Transplantation
Children’s Hospital Regensburg
Regensburg, Germany

Agnieszka Czechowicz, MD, PhD
Assistant Professor, Pediatrics - Stem Cell Transplantation
Stanford University
Stanford, CA

Amy DeZern, MD, MHS
Director, Bone Marrow Failure and MDS Program
Associate Professor of Oncology
Johns Hopkins School of Medicine
Baltimore, MD

Florian Eichler, MD
Director of the Leukodystrophy Service
Mass General Hospital for Children
Boston, MA

Francesca Fumagalli, MD, PhD
Neurologist
San Raffaele Telethon Institute For Gene Therapy, San Raffaele Hospital
Milan, Italy

R. Brad Jones, PhD
Viral Immunologist; Associate Professor
Division of Infectious Disease 
Weill Cornell Medicine

Neena Kapoor, MD
Director, Blood and Marrow Transplant Laboratory
Attending Physician; Hematology, Oncology and Blood and Marrow Transplantation
Investigator, Hematology-Oncology 
Professor of Clinical Pediatrics, 
Keck School of Medicine of USC
Children’s Hospital Los Angeles
Los Angeles, CA

Jeffrey Lebensburger, DO
Associate Professor
Director, Hematology Section
Division of Pediatric Hematology-Oncology
University of Alabama at Birmingham
Birmingham, AL

Caroline Lindemans, MD, PhD
Pediatric Immunologist
Prinses Maxima Centrum
Utrecht, Netherlands

Giovanna Lucchini, MD
Locum Consultant Immunologist in Immunology and Stem Cell Transplantation
Great Ormond Street Hospital for Children
London, England

Margaret MacMillan, MD
Professor, Department of Pediatrics
Division Director, Division of Blood and Marrow Transplantation (BMT)
University of Minnesota
Minneapolis, MN

Eric Mallack, MD, MBE
Assistant Professor of Pediatrics and Neurology 
New York-Presbyterian
New York, NY

Ewelina Mamcarz, MD
Assistant Member, St. Jude Faculty
St. Jude Children’s Research Hospital
Memphis, TN

Deepa Manwani, MD
Professor, Department of Pediatrics, Albert Einstein College of Medicine
Director of Hematology, Department of Pediatrics
The Children’s Hospital at Montefiore
Bronx, NY

Parinda Mehta, MD
Professor, UC Department of Pediatrics
Cincinnati Children’s
Cincinnati, OH 

Sung-Yun Pai, MD
Chief, Immune Deficiency Cellular Therapy Program
National Institutes of Health
Bethesda, MD 

Matthew Porteus, MD, PhD
Sutardja Chuk Professor of Definitive and Curative Medicine
Stanford University
Stanford, CA

Charles Quinn, MD, MS
Medical Director, Pediatric Sickle Cell Program
Director, Erythrocyte Diagnostic Laboratory
Professor, UC Department of Pediatrics
Cincinnati Children’s
Cincinnati, OH 

Gerald Raymond, MD
Professor of Genetic Medicine
Professor of Neurology
Johns Hopkins Hospital
Baltimore, MD

Sharon Savage, MD
DCEG Clinical Director
Branch Chief
Senior Investigator
National Institutes of Health
Rockville, MD

Elisa Seeger 
Founder 
Aidan Jack Seeger Foundation/ALD Alliance
Brooklyn, NY

Akiko Shimamura, MD, PhD
Director, Bone Marrow Failure and Myelodysplastic Syndrome Programs
Professor of Pediatrics, Harvard Medical School
Boston Children’s Hospital
Boston, MA

Agata Smogorzewska, MD, PhD
Associate Professor 
The Rockefeller University
New York, NY

Melissa Wasserstein, MD
Chief, Division of Pediatric Genetic Medicine
The Children’s Hospital at Montefiore
Bronx, NY

Annemarie Wensing, MD, PhD
Clinical Virologist
University Medical Center Utrecht
Utrecht, Netherlands

MSK Course Faculty 

Deborah Goldfrank, MD, FACOG
Head, General Gynecology 

Julia Kearney, MD
Child Psychiatrist

Jennifer Kennedy, MS, CGC
Genetic Counselor

Ross Levine, MD
Laurence Joseph Dineen Chair in Leukemia Research
Chief, Molecular Cancer Medicine Service, HOPP

Alina Markova, MD
Assistant Attending of Dermatology

Ariela Noy, MD
Medical Oncologist

Richard O’Reilly, MD
Claire L. Tow Chair in Pediatric Oncology Research 

Susan Prockop, MD
Pediatric Oncologist

Bhuvanesh Singh, MD, PhD, FACS
Director, Laboratory of Epithelial Cancer Biology 
Co-Director, Multidisciplinary Skin Cancer 
Management Program

Roni Tamari, MD
Hematologic Oncologist

Mike Walsh, MD
Geneticist & Pediatric Oncologist 

Faculty Disclosures (All Faculty and Planners)

Member Information

Role in activity

Nature of Relationship(s) / Name of Ineligible Company(s)

Rekha Parameswaran, MD
MSK

Activity Co-Director, Conflict of Interest Peer Reviewer, Faculty

Nothing to disclose

Maria I Cancio, MD
MSK

Activity Director, Conflict of Interest Peer Reviewer, Faculty

Nothing to disclose

Alice Bertaina, MD, PhD
Stanford University

Faculty

Nothing to disclose

Monica Bhatia, MD
Columbia University Irving Medical Center

Faculty

Nothing to disclose

Marc Bierings, MD, PhD
Prinses Maxima Centrum

Faculty

Nothing to disclose

Catherine Bollard, MD
Children's National Research Institute

Faculty

Membership on Advisory Committees or Review Panels, Board Membership, etc.-Cabaletta Bio|Advisor-Pfizer, Inc. (Relationship has ended)|Membership on Advisory Committees or Review Panels, Board Membership, etc.-Mana Therapeutics|Membership on Advisory Committees or Review Panels, Board Membership, etc.-Catamaran Bio|Advisor-Roche|Advisor-Bristol-Myers Squibb (Relationship has ended)|Honoraria-Sobi

Carmem Bonfim, MD, PhD
Hospital Pequeno Principe/Federal University of Parana
Brazil

Faculty

Consulting Fee-Novartis AG (Relationship has ended)|Membership on Advisory Committees or Review Panels, Board Membership, etc.-Zodiac (Relationship has ended)|Consulting Fee-Zodiac (Relationship has ended)|Consulting Fee-AMGEN (Relationship has ended)

Selim Corbacioglu, MD
University of Regensburg 
Germany

Faculty

Nothing to disclose

Agnieszka Czechowicz , MD, PhD
Stanford University

Faculty

Grant or research support-ROCKET PHARMACEUTICALS, INC|Stocks or stock options, excluding diversified mutual funds-GLOBAL BLOOD THERAPEUTICS|Advisor-BEAM THERAPEUTICS|Advisor-SPOTLIGHT THERAPEUTICS|Advisor-STEMODONTICS|Stocks or stock options, excluding diversified mutual funds-EDITAS MEDICINES|Stocks or stock options, excluding diversified mutual funds-MAGENTA THERAPEUTICS|Stocks or stock options, excluding diversified mutual funds-DECIBEL THERAPEUTICS|Consulting Fee-GV|Grant or research support-JASPER THERAPEUTICS|Other: Licensing revenue-GILEAD SCIENCES|Advisor-LYRIK THERAPEUTICS

Amy DeZern, MD, MHS
Johns Hopkins School of Medicine

Faculty

Consulting Fee-taiho|Membership on Advisory Committees or Review Panels, Board Membership, etc.-Geron|Advisor-Takeda Pharmaceuticals, Inc. (Relationship has ended)|Advisor-Novartis AG (Relationship has ended)

Florian Eichler, MD
Mass General Hospital for Children

Faculty

Honoraria-SwanBio Therapeutics|Consulting Fee-bluebird bio|Independent Contractor (included contracted research)-Minoryx Therapeutics|Grant or research support-Takeda Pharmaceuticals, Inc.|Grant or research support-Novartis AG|Consulting Fee-Autobahn Therapeutics|Independent Contractor (included contracted research)-Ionis Pharmaceuticals|Consulting Fee-Taysha Gene Therapies (Relationship has ended)|Consulting Fee-Poxel SA

Francesca Fumagalli, MD, PhD
San Raffaele Telethon Institute For Gene Therapy, San Raffaele Hospital

Faculty

Membership on Advisory Committees or Review Panels, Board Membership, etc.-Takeda Pharmaceuticals, Inc. (Relationship has ended)|Consulting Fee-Orchard Therapeutics

Deborah Goldfrank, MD
MSK

Faculty

Nothing to disclose

Brad Jones, PhD

Faculty

Nothing to disclose

Neena Kapoor, MD
Children's Hospital Los Angeles

Faculty

Nothing to disclose

Julia A. Kearney, MD
MSK

Faculty

Nothing to disclose

Jennifer A. Kennedy, MS
MSK

Faculty

Nothing to disclose

Jeffrey Lebensburger, DO
University of Alabama at Birmingham

Faculty

Consulting Fee-Novartis AG|Consulting Fee-Bioproducts Laboratory|Grant or research support-Pfizer, Inc. (Relationship has ended)

Ross Levine, MD
MSK

Faculty

Stocks or stock options, excluding diversified mutual funds-Loxo (Relationship has ended)|Grant or research support-Roche (Relationship has ended)|Honoraria-Astellas (Relationship has ended)|Other: Honorarium and Travel Expenses-AstraZeneca (Relationship has ended)|Honoraria-Morphosys (Relationship has ended)|Other: Honorarium, Consultant fee , Travel Expenses-Novartis AG (Relationship has ended)|Honoraria-Janssen (Relationship has ended)|Honoraria-Incyte (Relationship has ended)|Other: Honorarium and Travel Expenses-Gilead (Relationship has ended)|Other: consultant fee, Honorarium -Lilly (Relationship has ended)|Other: Honorarium, research support-Constellation (Relationship has ended)|Consulting Fee-Bridge Therapeutics (Relationship has ended)|Consulting Fee-BMS (Relationship has ended)|Consulting Fee-Jubilant (Relationship has ended)|Consulting Fee-Celgene|Other: Stock Shareholder, cash compensation, travel expenses-Qiagen|Stocks or stock options, excluding diversified mutual funds-Imago|Other: Stock Shareholder, consultant fee, Travel expenses-C4 Therapeutics|Stocks or stock options, excluding diversified mutual funds-Isoplexis|Other: Stock shareholder, Research support-Prelude Therapeutics|Stocks or stock options, excluding diversified mutual funds-Zentalis|Other: Stock Shareholder, consultant fee-Mana|Stocks or stock options, excluding diversified mutual funds-Auron|Stocks or stock options, excluding diversified mutual funds-Ajax|Other: Stock Shareholder, consultant fee-Kurome|Stocks or stock options, excluding diversified mutual funds-Mission Bio|Stocks or stock options, excluding diversified mutual funds-Scorpion|Grant or research support-Cure Breast Cancer Foundation|Grant or research support-ECOG-ACRIN |Other: honoarium, grant support-Mark Foundation|Honoraria-Genome Canada|Consulting Fee-Syndax|Consulting Fee-Stelexis|Consulting Fee-Vida Ventures|Consulting Fee-Anovia|Stocks or stock options, excluding diversified mutual funds-Bakx Tx |Consulting Fee-Bridge Bio|Consulting Fee-Bridge Medicines|Consulting Fee-Daiichi|Stocks or stock options, excluding diversified mutual funds-Epiphanes |Consulting Fee-MorphoSys AG

Caroline Lindemans, MD
Prinses Maxima Centrum
Netherlands

Faculty

Advisor-Orchard therapeutics, Bluebird Bio, Sobi (Relationship has ended)

Giovanna Lucchini, MD
Great Ormond Street Hospital for Children

Faculty

Nothing to disclose

Margaret MacMillan, MD
University of Minnesota

Faculty

Nothing to disclose

Eric Mallack, MD, MBE
New York-Presbyterian Memorial Sloan Kettering Cancer Center

Faculty

Nothing to disclose

Ewelina Mamcarz, MD
St. Jude Children's Research Hospital

Faculty

Nothing to disclose

Deepa Manwani, MD
Montefiore Medical Center; Albert Einstein School of Medicine

Faculty

Consulting Fee-Pfizer, Inc.|Consulting Fee-Novartis AG|Consulting Fee-Forma, GBT|Grant or research support-Grifol's

Alina Markova , MD
MSK

Faculty

Grant or research support-Incyte Corporation|Advisor-Alira Health Ventures|Advisor-Blueprint Medicines|Royalties or Patent Beneficiary-UpToDate|Grant or research support-Amryt Pharma

Parinda A. Mehta, MD, Professor of Pediatrics
Cincinnati Children's Hospital Medical Center

Faculty

Nothing to disclose

Ariela Noy, MD
MSK

Faculty

Consulting Fee-Epizyme|Consulting Fee-Janssen|Consulting Fee-medscape (Relationship has ended)|Other: Research funding-Pharmacyclics, Inc.|Other: Research funding-Rafael Pharma|Membership on Advisory Committees or Review Panels, Board Membership, etc.-Morphosys (Relationship has ended)|Honoraria-Physician Education Resource (Relationship has ended)

Richard J O'Reilly, MD
MSK

Faculty

Consulting Fee-Atara Biotherapeutics|Grant or research support-Atara Biotherapeutics|Royalties or Patent Beneficiary-Atara Biotherapeutics

Sung-Yun Pai, MD
National Institutes of Health

Faculty

Nothing to disclose

Matthew Porteus, MD, PhD
Stanford University

Faculty

Nothing to disclose

Susan Prockop, MD
MSK

Faculty

Membership on Advisory Committees or Review Panels, Board Membership, etc.-Neovii and ADMA (Relationship has ended)

Charles Quinn, MD, MS
Cincinnati Children's

Faculty

Grant or research support-Emmaus Medical|Advisor-Novo Nordisk (Relationship has ended)|Advisor-Forma Therapeutics (Relationship has ended)

Gerald Raymond, MD
Johns Hopkins Hospital

Faculty

Nothing to disclose

Sharon Savage, MD
National Institutes of Health

Faculty

Nothing to disclose

Elisa Seeger
ALD Alliance

Faculty

Nothing to disclose

Akiko Shimamura, MD, PhD
Harvard Medical School, Dana Farber/Boston Children’s Cancer and Blood Disorders Center

Faculty

Membership on Advisory Committees or Review Panels, Board Membership, etc.-Novartis AG

Bhuvanesh Singh, MD, PhD
MSK

Faculty

Advisor-Cinsano

Agata Smogorzewska, MD, PhD
Rockefeller University

Faculty

Grant or research support-Rocket Pharmaceuticals

Michael Walsh, MD
MSK

Faculty

Nothing to disclose

Melissa Wasserstein, MD
The Children’s Hospital at Montefiore

Faculty

Grant or research support-Alexion|Grant or research support-BioMarin|Grant or research support-Orchard|Grant or research support-PassageBio|Consulting Fee-Sanofi Genzyme|Grant or research support-Sanofi Genzyme|Grant or research support-Sio|Grant or research support-Takeda Pharmaceuticals, Inc.|Consulting Fee-Takeda Pharmaceuticals, Inc. (Relationship has ended)|Grant or research support-travere|Grant or research support-ultragenyx

Annemarie Wensing, MD, PhD
University Medical Center Utrecht

Faculty

Consulting Fee-Gilead|Consulting Fee-Johnson and Johnson (Relationship has ended)

Jaap-Jan Boelens, MD
MSK

Faculty, Planner

Consulting Fee-Sanofi-Aventis|Consulting Fee-Avrobio|Consulting Fee-Race Oncology|Consulting Fee-Omeros|Consulting Fee-Bluerock|Consulting Fee-advanced clinical

Christina Cho, MD
MSK

Faculty, Planner

Nothing to disclose

Sergio Giralt, MD
MSK

Faculty, Planner

Consulting Fee-Celgene|Consulting Fee-AMGEN|Consulting Fee-Johnson and Johnson|Consulting Fee-jazz|Consulting Fee-sanofi|Consulting Fee-KITE|Consulting Fee-Novartis AG

Jodi Mones, MD
MSK

Faculty, Planner

Nothing to disclose

Miguel Perales, MD
MSK

Faculty, Planner

Membership on Advisory Committees or Review Panels, Board Membership, etc.-NexImmune|Membership on Advisory Committees or Review Panels, Board Membership, etc.-Astellas|Membership on Advisory Committees or Review Panels, Board Membership, etc.-Celgene|Membership on Advisory Committees or Review Panels, Board Membership, etc.-Bristol-Myers Squibb|Membership on Advisory Committees or Review Panels, Board Membership, etc.-Incyte Corporation|Membership on Advisory Committees or Review Panels, Board Membership, etc.-Karyopharm|Membership on Advisory Committees or Review Panels, Board Membership, etc.-Gilead|Membership on Advisory Committees or Review Panels, Board Membership, etc.-Miltenyi Biotec|Membership on Advisory Committees or Review Panels, Board Membership, etc.-MorphoSys|Membership on Advisory Committees or Review Panels, Board Membership, etc.-Nektar Therapeutics|Membership on Advisory Committees or Review Panels, Board Membership, etc.-Novartis AG|Membership on Advisory Committees or Review Panels, Board Membership, etc.-OrcaBio|Membership on Advisory Committees or Review Panels, Board Membership, etc.-Takeda Pharmaceuticals, Inc.|Membership on Advisory Committees or Review Panels, Board Membership, etc.-VectivBio AG|Membership on Advisory Committees or Review Panels, Board Membership, etc.-Vor Biopharma|Consulting Fee-Merck and Company, Inc.|Consulting Fee-Omeros|Membership on Advisory Committees or Review Panels, Board Membership, etc.-Cidara Therapeutics,|Membership on Advisory Committees or Review Panels, Board Membership, etc.-Medigene|Membership on Advisory Committees or Review Panels, Board Membership, etc.-Sellas Life Sciences|Membership on Advisory Committees or Review Panels, Board Membership, etc.-Servier|Ownership-NexImmune|Ownership-Omeros|Grant or research support-Incyte Corporation|Grant or research support-Gilead|Grant or research support-Miltenyi|Grant or research support-Novartis AG|Honoraria-Astellas|Honoraria-Bristol-Myers Squibb|Honoraria-Celgene|Honoraria-Equilium|Honoraria-Incyte Corporation|Honoraria-Karyopharm|Honoraria-Gilead|Honoraria-Merck and Company, Inc.|Honoraria-Miltenyi Biotec|Honoraria-MorphoSys|Honoraria-Novartis AG|Honoraria-Nektar Therapeutics|Honoraria-Omeros|Honoraria-OrcaBio|Honoraria-Takeda Pharmaceuticals, Inc.|Honoraria-VectivBio AG|Honoraria-Vor Biopharma|Membership on Advisory Committees or Review Panels, Board Membership, etc.-Cidara Therapeutics|Membership on Advisory Committees or Review Panels, Board Membership, etc.-Medigene|Membership on Advisory Committees or Review Panels, Board Membership, etc.-Sellas Life Sciences|Membership on Advisory Committees or Review Panels, Board Membership, etc.-Servier|Membership on Advisory Committees or Review Panels, Board Membership, etc.-NexImmune

Roni Tamari, MD
MSK

Faculty, Planner

Nothing to disclose

Rachel Corke, MPA
MSK

Planner

Nothing to disclose

Alexis Folz
MSKCC

Planner

Nothing to disclose

Andrew L Kung, MD, PhD
MSK

Planner

Membership on Advisory Committees or Review Panels, Board Membership, etc.-Emendo Biotherapeutics|Membership on Advisory Committees or Review Panels, Board Membership, etc.-Karyopharm Therapeutics|Membership on Advisory Committees or Review Panels, Board Membership, etc.-Imago BioSciences|Membership on Advisory Committees or Review Panels, Board Membership, etc.-DarwinHealth|Ownership-Isabl

Jillian Guskin, MPH
MSKCC

RSS Coordinator

Nothing to disclose

Memorial Sloan Kettering gratefully acknowledges the following organizations for providing educational grants for this live activity:

Gamida Cell
Novartis
Orchard Therapeutics
Vertex Pharmaceuticals, Inc.