BONE MARROW FAILURE DISEASE SYMPOSIUM SATURDAY SEPTEMBER 27, 2014 AN UPDATE ON THE LATEST IN DIAGNOSIS AND TREATMENT

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1 SATURDAY SEPTEMBER 27, 2014 THE HOTEL HERSHEY 100 Hotel Road, Hershey, PA BONE MARROW FAILURE DISEASE SYMPOSIUM AN UPDATE ON THE LATEST IN DIAGNOSIS AND TREATMENT A CONTINUING EDUCATION SERVICE OF PENN STATE COLLEGE OF MEDICINE AND THE APLASTIC ANEMIA & MDS INTERNATIONAL FOUNDATION

2 PURPOSE This symposium will provide participants with the most up-to-date review of scientific and clinical research related to the biology and management of bone marrow failure diseases, including aplastic anemia, large granular lymphocyte leukemia, myelodysplastic syndromes, and paroxysmal nocturnal hemoglobinuria. The presenters will focus on the latest advances in the diagnosis, genetic characteristics, pathology and treatment options for these diseases. AUDIENCE The intended audience for the symposium includes hematologists, medical oncologists, internists, pathologists, residents/fellows, physician assistants, nurse practitioners, nurses and allied health professionals treating patients with bone marrow failure diseases. COURSE DIRECTOR JEFFREY J. PU, MD, MS, PHD Assistant Professor of Medicine Penn State Hershey Cancer Institute Penn State College of Medicine Penn State Milton S. Hershey Medical Center Hershey, PA AAMDS.org The Aplastic Anemia & MDS International Foundation is the world's leading nonprofit health organization dedicated to supporting patients and families living with aplastic anemia, myelodysplastic syndromes, paroxysmal nocturnal hemoglobinuria, and related bone marrow failure diseases. This publication is available in alternative media on request. The Pennsylvania State University is committed to the policy that all persons shall have equal access to programs, facilities, admission, and employment without regard to personal characteristics not related to ability, performance, or qualifications as determined by University policy or by state or federal authorities. It is the policy of the University to maintain an academic and work environment free of discrimination, including harassment. The Pennsylvania State University prohibits discrimination and harassment against any person because of age, ancestry, color, disability or handicap, genetic information, national origin, race, religious creed, sex, sexual orientation, gender identity, or veteran status. Discrimination or harassment against faculty, staff, or students will not be tolerated at The Pennsylvania State University. Direct all inquiries regarding the nondiscrimination policy to the Affirmative Action Director, The Pennsylvania State University, 328 Boucke Building, University Park, PA ; Tel /V, /TTY. U.Ed. MED E Z

3 OBJECTIVES Upon completion of this activity, participants should be able to: Provide accurate differential diagnosis of bone marrow failure diseases, including myelodysplastic syndromes, aplastic anemia, large granular lymphocyte leukemia, paroxysmal nocturnal hemoglobinuria and myeloid malignancies Critically appraise recent advances in research on the genetic characteristics of bone marrow failure diseases Summarize recent progress in understanding the pathogenesis of myeloid malignancies and bone marrow failure diseases Describe safety and efficacy data for currently available therapies for bone marrow failure diseases Identify current therapeutic standards, as well as new and emerging therapies used in hematopoietic cell transplantation CREDIT This activity has been planned and implemented in accordance with the accreditation requirements and policies of the Accreditation Council for Continuing Medical Education (ACCME) through the joint providership of Penn State College of Medicine and the Aplastic Anemia & MDS International Foundation. Penn State College of Medicine is accredited by the ACCME to provide continuing medical education for physicians. Penn State College of Medicine designates this live activity for a maximum of 5.5 AMA PRA Category 1 Credit(s) TM. Physicians should claim only the credit commensurate with the extent of their participation in the activity. NURSES USING AMA CREDIT Nurses will receive a certificate from Penn State College of Medicine verifying their participation in 5.5 hours of approved continuing medical education. Continuing education activities which are approved by accredited institutions of higher education and that are relevant to patient care or professional nursing meet the requirements of mandatory continuing education for registered nurses in Pennsylvania, as required by the Pennsylvania State Board of Nursing. The Board of Nursing recommends that nurses retain their certificates for a period of at least five years. Additional information is available through the State Board of Nursing at dos.state.pa.us/nurse. It is our policy to ensure balance, independence, objectivity, and scientific rigor in all of our educational programs. Faculty and course directors have disclosed relevant financial relationships with commercial companies, and Penn State has a process in place to resolve any conflict of interest. Complete faculty disclosure will be provided to program participants at the beginning of the activity.

4 FACULTY MICHAEL G. BAYERL, MD* Associate Professor Division of Anatomic Pathology MONICA BESSLER MD, PHD Buck Family Professor of Hematology Professor of Pediatrics and Internal Medicine Director, Pediatric and Adult Comprehensive Bone Marrow Failure Center The Children s Hospital of Philadelphia and The Hospital of the University of Pennsylvania University of Pennsylvania School of Medicine Philadelphia, Pennsylvania ROBERT A. BRODSKY, MD Johns Hopkins Family Professor of Oncology Research Director, Division of Hematology Johns Hopkins Medicine Institute Baltimore, Maryland DAVID F. CLAXTON, MD* Professor of Medicine RICHARD W. CHILDS, MD Clinical Director Division of Intramural Research National Heart, Lung and Blood Institute Bethesda, Maryland RAYMOND J. HOHL, MD, PHD* Professor of Medicine and Director THOMAS P. LOUGHRAN, MD Professor of Medicine and Director Virginia University Cancer Center Virginia University School of Medicine Charlottesville, Virginia JEFFREY J. PU, MD, MS, PHD* Assistant Professor of Medicine *Penn State Hershey Cancer Institute; Penn State College of Medicine; Penn State Milton S. Hershey Medical Center, Hershey, PA

5 AGENDA 8:00 Registration and Continental Breakfast 8:30 Welcome Jeffrey J. Pu, MD, MS, PhD 8:40 Myelodysplastic Syndromes: Sixty and Maturing Raymond J. Hohl, MD, PhD 9:10 Combined Transplantation with Umbilical Cord Blood and Haploidentical Stem Cells: A New and Effective Treatment Option for Patients with Refractory Aplastic Anemia Who Lack Human Leukocyte Antigen Matched Donors Richard W. Childs, MD 9:40 Morning Break 10:10 Paroxysmal Nocturnal Hemoglobinuria and Bone Marrow Failure Robert A. Brodsky, MD 10:40 Pathologic Considerations in Bone Marrow Failure and Mimics Michael G. Bayerl, MD 11:10 Panel Discussion Noon Lunch in the Circular Dining Room 1:00 Pathogenesis of Large Granular Lymphocytic Leukemia Thomas P. Loughran, MD 1:30 Moving Towards Precision Medicine in Bone Marrow Failure Monica Bessler MD, PhD 2:00 Afternoon Break 2:20 Genomic Analysis and Advances in the Treatment of Acute Myeloid Leukemia David F. Claxton, MD 2:50 The Relevance of Small Population of GPI-AP Deficient Cells in Bone Marrow Failure Diseases Jeffrey J. Pu, MD, MS, PhD 3:20 Panel Discussion 4:00 Wrap Up and Adjournment

6 FEE } $50 PER PERSON Includes: Continental Breakfast Handout Materials Refreshments Lunch CANCELLATION POLICY Cancellations received later than: } SEPTEMBER 22, 2014 will incur a $25 processing fee. No refunds: } AFTER SEPTEMBER 24, 2014 Substitutes are accepted at any time. The University reserves the right to cancel or postpone any course or activity because of insufficient enrollment or other unforeseen circumstances. Please provide a cell phone number so we can reach you in the event of last-minute changes or cancellation. If a program is canceled or postponed, the University will refund registration fees but cannot be held responsible for any related costs, charges, or expenses, including cancellation/change charges assessed by airlines or travel agencies. Preregistered participants will be notified if a program is filled or canceled. REGISTRATION Registrations will be confirmed by upon receipt of payment, authorization to bill, or a credit card guarantee. If you do not receive a confirmation within two weeks, call to confirm your registration. Walk-ins and late registrations will be accepted if space is available. Call Fax ContinuingEd@hmc.psu.edu Mail Registrar, Penn State Hershey Continuing Education G220, P. O. Box 851 Hershey, PA REGISTER ONLINE TODAY! pennstatehershey.org/web/ce

7 BONE MARROW FAILURE DISEASE SYMPOSIUM SATURDAY SEPTEMBER 27, 2014 E Z Name (First Name, MI, Last Name) q Male q Female Degree/Credentials q MD/ DO q PA q NP q PT q AT q Other Address (Print legibly. Used for confirmation/registration/program materials.) Home Address City State Zip Code Daytime Phone Cell Phone (For last-minute change or cancellation) Hospital or Organization Specialty/Position Address (HMC/COM employees dept. name/mail code) City State Zip Code Special Needs (Dietary, access, etc.) } FEE $50 PER PERSON Indicate method of payment: q Check enclosed payable to Penn State q Bill my sponsoring organization attach authorization letter q HMC/COM employee bill department budget Cost Center/Budget # Charge my credit card: q VISA q MasterCard q AMEX Account # Exp. Date Signature

8 THE PENNSYLVANIA STATE UNIVERSITY PENN STATE MILTON S. HERSHEY MEDICAL CENTER DEPARTMENT OF CONTINUING EDUCATION G220 P.O. BOX 851 HERSHEY, PA BONE MARROW FAILURE DISEASE SYMPOSIUM SATURDAY SEPTEMBER 27, 2014 REGISTER ONLINE TODAY! pennstatehershey.org/web/ce NONPROFIT ORG. U.S. POSTAGE P A I D PA STATE UNIVERSITY M.S. HERSHEY MED. CTR.