cincinnati, oh · 2018. 8. 17. · cincinnati, oh september 7, 2018 rgional program asfa 2018...

REGIONAL MEETING

ProgramUC COLLEGE OF MEDICINE,

KRESGE AUDITORIUM, CINCINNATI, OHIO

CINCINNATI, OHSEPTEMBER 7, 2018

REGIONAL PROGRAM

CINCINNATI, OH | SEPTEMBER 7, 2018

WELCOME FROM THE ASFA PRESIDENT

On behalf of the Board of Directors and myself, I would like to extend a warm welcome to the 5th Regional Meeting of the American Society for Apheresis at the UC College of Medi-cine, in Cincinnati, Ohio. I would like to take this opportunity to thank the Regional Meeting Organizing Committee, led by Jose A. Cancelas, MD, PhD, for their hard work in planning a fantastic program for you. The meeting program offers a full day of exciting educational and networking opportunities in apheresis medicine that will appeal to all apheresis prac-titioners. I encourage you to take advantage of networking opportunities at the meeting. Please seek out members of the Regional Meeting Organizing Committee, other ASFA members, and the ASFA Registration Desk to find out about the benefits of ASFA membership and how you can become more involved in the society. As an additional bene fit, the Main Strasse Village Oktoberfest will take place across the river in Covington. The Cincinnati Reds are at home this weekend, and the many great new and old city attractions, award-winning restaurants, shopping opportunities and multi-cultural activities can all be found in the area. I must also men-tion the expertise and tireless effort provided by the ASFA Head Office staff. Finally, I want to extend our thanks to our exhibitors and our hosts at the UC College of Medicine who helped make this Regional Meeting possible. I hope you en-joy the 5th ASFA Regional Meeting and look forward to future regional meetings.

Sincerely,

Joseph Schwartz, MD, MPH

President, American Society for Apheresis

WELCOME FROM THE CINCINNATI REGIONAL MEETING ORGANIZING COMMITTEE

On behalf of the ASFA Regional Meeting Organizing Commit-tee, we warmly welcome you to Cincinnati & Hoxworth Blood Center/University of Cincinnati for the 5th ASFA Regional Meeting. The Organizing Committee has put together a one-day program of didactic and interactive sessions intended to appeal to all apheresis practitioners. We are very excited about this educational and networking opportunity. We look forward to your participation!

We would like to thank the University of Cincinnati College of Medicine for allowing us to use their facilities for this meeting.

Sincerely,

Jose A. Cancelas, MD, PhD & Matthew Montgomery, MD

Cincinnati & Hoxworth Blood Center/University of Cincinnati

UC COLLEGE OF MEDICINE, KRESGE AUDITORIUM, CINCINNATI, OHIOwww.apheresis.org

REGIONAL MEETING

GENERAL INFORMATION

TARGET AUDIENCE

The target audience for this program is physicians, scientists and allied health professionals working in apheresis, includ-ing but not limited to pathology, hematology, immunology, ne-phrology, pediatrics and rheumatology.

LEARNING OBJECTIVES

After participating in this CME activity, participants should be able to:

• Describe the general practice of apheresis medicine and its role in the treatment of diseases in a variety of organ systems

• Describe the emerging field of personal cellular therapy and the importance of good manufacturing practices in the manufacturing of these products

• Describe current knowledge of cellular therapy• Assess the limitations, advantages and technical

aspects of new apheresis instruments along with best practices on the use of central venous catheters for apheresis procedures in children and adults

ACCREDITATION AND DESIGNATION OF CREDIT

Please complete the online evaluation and credit request that will be sent to you via email post-conference to obtain your respective credit certificates.

CONTINUING MEDICAL EDUCATION CREDIT

ACCREDITATIONThis activity has been planned and implemented in accor-dance with the Essential Areas and Policies of the Accred-itation Council for Continuing Medical Education (ACCME) through the joint providership of the American College of Sur-geons and the American Society for Apheresis. The American College of Surgeons is accredited by the ACCME to provide continuing medical education for physicians.

AMA PRA CATEGORY 1 CREDITS™

The American College of Surgeons designates this live ac-tivity for a maximum of 7.50 AMA PRA Category 1 Credits™. Physicians should claim only the credit commensurate with the extent of their participation in the activity.

CEUCEUs have been approved by ASFA along with the California Board of Registered Nursing. A maximum of 7.50 CEUs can be earned through this educational activity (ASFA Provider Number CEP 14122). Completion of the online evaluation sur-vey is required for all conference delegates which, upon com-pletion, will allow you to receive your CEU credits. This survey must be completed within one month after the meeting in or-der to receive your credits. Electronic CEU certificates will be e-mailed within 4-6 weeks following the meeting.

CMLEThis continuing medical laboratory education activity is recog-nized by the American Society for Clinical Pathology (ASCP) as meeting the criteria for a maximum number of 7.50 CMLE credits. ASCP CMLE credit hours are acceptable to meet the continuing education requirement for the ASCP Board of Registry Certification Maintenance Program. (ASFA Provider Number 261-12-11). Completion of the online evaluation survey is required for all conference delegates which, upon comple-tion, will allow you to receive your CMLE credits. This survey must be completed within one month after the meeting in or-der to receive your credits. Electronic CMLE certificates will be e-mailed within 4-6 weeks following the meeting.


REGIONAL PROGRAM

ASFA 2018 REGIONAL MEETING SPEAKERS AND FACILITATORSSPEAKERS

Joseph Schwartz, MD, MPH, Columbia University, New York, NYJose Cancelas, MD, PhD, Hoxworth Blood Center, Cincinnati, OHJeffrey L. Winters, MD, Mayo Clinic College of Medicine, Rochester, MNDavid Oh, MD, Hoxworth Blood Center, Cincinnati, OHJan C. Hofmann, MD, MPH, MSc, UCSF School of Medicine, San Francisco, CAMarty Gerhardt, Hoxworth Blood Center, Cincinnati, OHJoerg Krueger, MD, The Hospital for Sick Children, Toronto, ONAnand Padmanabhan, MD, PhD, QIA, BloodCenter of Wisconsin, Milwaukee, WIStuart Goldstein, MD, FAAP, FNKF, Cincinnati Children’s Hospital, Cincinnati, OHRavindra Sarode, MD, UT Southwestern Medical Center, Dallas, TXDavid Ward, MD, FRCP, HP(ASCP), University of California San Diego, CAMatthew Montgomery, MD, Hoxworth Blood Center, Cincinnati, OHPunam Malik, MD, Cincinnati Children’s Hospital, Cincinnati, OHThomas Leemhuis, PhD, Hoxworth Blood Center, Cincinnati, OHCarolyn Lutzko, PhD, Hoxworth Blood Center, Cincinnati, OH

FACILITATORSAngie Bonavita, BSN, RN, CHTC, Cincinnati Children’s Hospital, Cincinnati, OHMark Mueller, RN, BS, ASN, BMTCN, Cincinnati Children’s Hospital, Cincinnati, OHCharles Quinn, MD, MS, Cincinnati Children’s Hospital, Cincinnati, OHKatye Hogue, RN, BSN, Hoxworth Blood Center, Cincinnati, OHRavindra Sarode, MD, UT Southwestern Medical Center, Dallas, TXAlexander (Alex) Bondoc, MD, Cincinnati Children’s Hospital, Cincinnati, OHJoseph Schwartz MD, MPH, Columbia University, New York, NYJanice (Jan) Habel, MS, Hoxworth Blood Center, Cincinnati, OHMark Folino, RN, Hoxworth Blood Center, Cincinnati, OHWalter Linz, MD, MBA, Texas A&M Health Science Center, Temple, TXDavid Ward, MD, FRCP, HP(ASCP), University of California San Diego, CA

ASFA 2018 REGIONAL MEETING ORGANIZING COMMITTEECHAIRS:

Jose Cancelas, MD, PhD (Chair), Hoxworth Blood Center, Cincinnati, OHMatthew Montgomery, MD (Co Chair), Hoxworth Blood Center, Cincinnati, OH

PLANNING MEMBERS:Laura L Collins, BSN, HP(ASCP), University of Iowa Hospitals and Clinics, Iowa City, IAMark Folino, RN, Hoxworth Blood Center, Cincinnati, OHThomas Leemhuis, PhD, Hoxworth Blood Center, Cincinnati, OHWalter J Linz, MD, MBA, Texas A&M Health Science Center, Temple, TXCarolyn Lutzko, PhD, Hoxworth Blood Center, Cincinnati, OHDavid Oh, MD, Hoxworth Blood Center, Cincinnati, OHJeffrey Papiernik, MD, University of Cincinnati College of Medicine, Cincinnati, OH Joseph Schwartz, MD, MPH, Columbia University, New York, NY

DISCLOSURE INFORMATIONIn accordance with the ACCME Accreditation Criteria, the American College of Surgeons, as the accredited provider of this activity, must ensure that anyone in a position to con-trol the content of the educational activity has disclosed all relevant financial relationships with any commercial interest. Therefore, it is mandatory that both the program planning committee and speakers complete disclosure forms. Mem-bers of the program committee were required to disclose all financial relationships and speakers were required to dis-close any financial relationship as it pertains to the content of the presentations. The ACCME defines a “commercial in-terest” as “any entity producing, marketing, re-selling, or dis-tributing health care goods or services consumed by, or used on, patients”. It does not consider providers of clinical service directly to patients to be commercial interests. The ACCME considers “relevant” financial relationships as financial trans-actions (in any amount) that may create a conflict of interest and occur within the 12 months preceding the time that the individual is being asked to assume a role controlling content of the educational activity.

ACS is also required, through our joint providership partners, to manage any reported conflict and eliminate the potential for bias during the activity. All program committee members and speakers were contacted and the conflicts listed below have been managed to our satisfaction. However, if you per-ceive a bias during a session, please report the circumstanc-es on the session evaluation form.

Please note we have advised the speakers that it is their responsibility to disclose at the start of their presentation if they will be describing the use of a device, product, or drug that is not FDA approved or the off-label use of an approved device, product, or drug or unapproved usage.

The requirement for disclosure is not intended to imply any impropriety of such relationships, but simply to identify such relationships through full disclosure and to allow the audi-ence to form its own judgments regarding the presentation.

EDUCATIONAL GRANTS

American Society for Apheresis wishes to recognize and thank the following company for its ongoing support through educational grants:

• Mallinckrodt Pharmaceuticals


Speakers / Moderators / Discussants

Nothing to

Disclose

Disclosure

Company Role Received

Joseph Schwartz, MD, MPH XJose Cancelas, MD, PhD Cerus Co

TerumoBCT Hemanext Inc US DoD National Institutes of Health

Nothing Nothing Advisor Fees Sent to Employer: Hoxworth Blood Center Nothing Nothing

Research Grant/Contract Research Grant/Contract Research Grant/Contract + Advisor Research Grant/Contract Research Grant/Contract

Jeffrey L Winters, MD Wiley Mayo Clinic

Salary Salary

Editor-in-Chief Journal of Clinical Apheresis Employee

David Oh, MD XJan C. Hofmann, MD, MPH, MSc Fresenius Medical Care Consultant Fees Consultant

Marty Gerhardt XJoerg Krueger, MD XAnand Padmanabhan, MD, PhD, QIA Terumo BCT

Jansen R&D Retham Technologies Veralox Therapeutics

Speaking Fee Consultancy Fee Equity Equity, Consulting Fee

Speaker Consultant President of Company Advisory Board Member, Consulting

Angie Bonavita, BSN, RN, CHTC XMark Mueller, RN, BS, ASN, BMTCN XCharles Quinn, MD, MS Amgen

Global Blood Therapeutics Silver Lake Research Corporation

Research funding to institution Research funding to institution NIH (SBIR) funding

Site P.I. for clinical trial Site P.I. for clinical trial Diagnostic device development

Katye Hogue, RN, BSN XRavindra Sarode, MD Octapharma

Ablynx Alexsion Portola

Self Self Self Self

Consultant Consultant Consultant Consultant

Alexander Bondoc, MD XJanice Habel, MS XMark Folino, RN XWalter Linz, MD, MBA XDavid Ward, MD, FRCP, HP(ASCP) TerumoBCT, Inc ( a manufacturer

of apheresis equipment) I am about to receive ~$5000, but have not received any payments in last 12 months

Consulting, paid by the hour.

Stuart Goldstein, MD, FAAP, FNKF Baxter Healthcare MediBeacon Bioporto Akebia ELOXX Medtronic Astute Renibus Reata

Honorarium/Grant Funding Honorarium Honorarium/Grant funding Honorarium Honorarium Honorarium Honorarium Honorarium Honorarium

Consultant, Educational Course Chair Consultant Consultant Consultant DSMB member Consultant Consultant Consultant DSMB member

Matthew Montgomery, MD XPunam Malik, MD XThomas Leemhuis, PhD XCarolyn Lutzko, PhD X

Planning CommitteeNothing

to Disclose

Disclosure

Company Role Received

Jose Cancelas, MD, PhD (Chair) Cerus Co TerumoBCT Hemanext Inc US DoD National Institutes of Health

Nothing Nothing Advisor fees sent to employer: Hoxworth Blood Center Nothing Nothing

Research Grant/Contract Research Grant/Contract Research Grant/Contract + Advisor Research Grant/Contract Research Grant/Contract

Matthew Montgomery, MD (Co-Chair) XLaura Collins, BSN, HP(ASCP) Terumo BCT Travel Expenses ConsultantMark Folino, RN XThomas Leemhuis, PhD XWalter Linz, MD, MBA XCarolyn Lutzko, PhD XDavid Oh, MD XJeffrey Papiernik XJoseph Schwartz, MD, MPH X


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MORNING SESSION

6:00am – 7:00am Exhibitor Setup

7:00am – 8:00am Breakfast and Registration

8:00am – 8:05am WELCOME REMARKS FROM ASFASpeaker: Joseph Schwartz, MD, MPH

8:05am – 8:10am WELCOME REMARKS FROM HOXWORTH BLOOD CENTERSpeaker: Jose A. Cancelas, MD, PhD

Session 1: Getting Ready for Apheresis: A 2018 PerspectiveModerator: Matthew Montgomery, MD

8:10am – 8:55am KEYNOTE LECTURE: PLASMA EXCHANGE: EVIDENCE, CONCEPTS AND DECISION MAKINGSpeaker: Jeffrey L. Winters, MD

8:55am – 9:20am HEALTHY DONOR APHERESIS PROCEDURES: SAFETY, EFFICACY & COMPLIANCESpeaker: David Oh, MD

9:20am – 9:45amCREDENTIALING/PRIVILEGING - IN MULTI-HOSPITAL SETTINGS: CARE IN THE 21ST CENTURY

Speaker: Jan C. Hofmann, MD, MPH, MSc

9:45am – 10:10am THERAPEUTIC APHERESIS IN CHILDREN: A PARENT’S EXPERIENCESpeaker: Marty Gerhardt

10:10am – 10:40am Break

Session 2: Optimization of Nucleated Cell Product CollectionsModerator: Carolyn Lutzko, PhD

10:40am – 11:05am OPTIMIZATION OF SPECTRA OPTIA FOR GRANULOCYTE COLLECTIONSSpeaker: Jose A. Cancelas, MD, PhD

11:05am – 11:30amHEMATOPOIETIC AND MONONUCLEAR CELL COLLECTIONS FOR ADVANCED THERAPIES IN CHILDREN

Speaker: Joerg Krueger, MD

11:30am – 11:55am LEUKOREDUCTION WITH THE SPECTRA OPTIASpeaker: Anand Padmanabhan, MD, PhD, QIA

11:55am – 1:00pm Lunch

PROGRAM SCHEDULE


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AFTERNOON SESSION

1:00pm – 2:00pm

ROUND TABLE DISCUSSIONModerators: Jose A. Cancelas MD, PhD, Matthew Montgomery, MD, & David Oh, MD

• Role of the Transplant Coordinator in Apheresis: Angie Bonavita, BSN, RN, CHTC & Mark Mueller, RN, BS, ASN, BMTCN

• RBC Exchange Transfusion/FCR Changes in RCX: Charles Quinn, MD, MS

• Pediatric Apheresis: Katye Hogue, RN, BSN

• FCR Goals: Ravindra Sarode, MD

• Vascular Access in Children: Alexander Bondoc, MD

• Challenges in HPC Collections: Joseph Schwartz, MD, MPH

• Quality Assurance of Donor and Patient Apheresis Units: Janice Habel, MS

• Donor Apheresis Operations: Mark Folino, RN

• Quality in Apheresis – How to Connect with ASFA: Walter Linz, MD

• Use of Lipid Exchange Columns in Hyperlipidemia Therapy: David Ward, MD, FRCP, HP(ASCP)

Session 3: Disease-Oriented ApplicationsModerator: David Oh, MD

2:00pm – 2:25pm TPE IN PEDIATRIC KIDNEY DISEASESpeaker: Stuart Goldstein, MD, FAAP, FNKF

2:25pm – 2:50pm ISOVOLEMIC HEMODILUTION-RED BLOOD CELL EXCHANGE IN SICKLE CELL ANEMIA (SCA)Speaker: Ravindra Sarode, MD

2:50pm – 3:15pm ROLE OF APHERESIS IN THE TREATMENT OF HYPERLIPIDEMIA Speaker: David Ward, MD

3:15pm – 3:40pm EXTRACORPOREAL PHOTOPHERESIS: WHAT IS NEW?Speaker: Matthew Montgomery, MD

3:40pm – 4:10pm Break

4:10pm – 4:45pm Exhibitor Move-Out

Session 4: Apheresis Optimization for Cell- & Gene-Therapy ApproachesModerator: Jose A. Cancelas, MD, PhD

4:10pm – 4:35pmGENE THERAPY FOR SICKLE CELL ANEMIA USING A MODIFIED GAMMA GLOBIN LENTIVIRUS VECTOR AND REDUCED INTENSITY CONDITIONING TRANSPLANT

Speaker: Punam Malik, MD

4:35pm – 5:00pmUTILIZING VIRUS-SPECIFIC T CELLS TO AUGMENT ANTIVIRAL IMMUNITY IN IMMUNOCOMPROMISED PATIENTS

Speaker: Thomas Leemhuis, PhD

5:00pm – 5:25pmQUALITY CONTROL FOR APHERESIS PRODUCTS TO BE USED IN ADVANCED IMMUNOTHERAPIES

Speaker: Carolyn Lutzko, PhD

5:25pm – 5:40pm SUMMARY & CLOSING REMARKSSpeaker: Jose A. Cancelas MD, PhD & Matthew Montgomery, MD


REGIONAL PROGRAMKEYNOTE LECTURE: PLASMA EXCHANGE: EVIDENCE, CONCEPTS AND DECISION MAKINGJeffrey L. Winters, MD

The concept of plasma exchange (originally referred to as plasmapheresis by Able, Rowntree and Turner) originated more than 114 years ago. The objectives of this keynote presentation will be to briefly describe the early application of plasma exchange in the treatment of disease, what lead people to consider this treatment? The mechanisms of separation, both by centrifugation and filtration, as well as other essential concepts, such as anticoagulation, that relate to these techniques will be discussed. The possible therapeutic mechanisms of plasma exchange will be examined; it is more than just the removal of “evil humors”, and how these possible mechanisms could inform future use of plasma exchange will be considered. Clinical considerations involved in clinical decision making, such as underlying comorbid conditions and concurrent therapies will be discussed. Clinical decision making will be framed within the context of the ASFA Guidelines. Finally, current and potential future indications for plasma exchange will be reviewed.

HEALTHY DONOR APHERESIS PROCEDURES: SAFETY, EFFICACY & COMPLIANCEDavid Oh, MD

Donor apheresis is an essential component of almost all blood center collection strategies. Donor red cells, plasma, and platelets are standard blood components collected by apheresis in larger amounts than can be collected through whole blood donation. Granulocytes are collected less frequently, but are typically requested for severely ill patients with life threatening infection not responding to antimicrobial therapy. Healthy donors are usually asked to take medication in order to increase the number of circulating neutrophils at the time of collection.

The speaker will review methods used by blood centers to maximize donor collections. A single attempted platelet donation may yield from zero to three apheresis platelet products. Maximizing efficiency in automated collections is essential for a blood center to provide blood products in significant quantities with decreased expenses.

The speaker will review steps taken by blood centers to ensure donor safety. Potential donors must fulfill eligibility requirements. Blood center policies limit donation frequency and collection volume according to AABB standards and federal regulations.

REMOTE APHERESIS TELEMEDICINE: EVOLVING ISSUES OF VIRTUAL CONSULTATION, PRIVILEGING, DOCUMENTATION, AND MEDICAL-LEGAL ASPECTS OF CARE IN THE 21ST CENTURYJan C. Hofmann, MD, MPH, MSc

This session will highlight the changing landscape of telemedicine in the 21st century focusing on the evolving practice of virtual apheresis consultation, with the goal of educating participants about topics such as: models of apheresis telemedicine and mobile apheresis, remote expert consultation, privileging, and electronic documentation. Issues of billing, coding, and reimbursement, and discussion of medical-legal, ethical, and research, privacy, and database concerns will be addressed. This session will explore questions, such as: what are possible pathways for providing expert remote apheresis consultation, and being compensated for such services? What are the medical-legal ramifications of providing virtual physician-to-physician consultation, and how can you minimize potential liabilities? Will established models of telemedicine (such as, teleradiology programs, eICU, and primary care services like MDLive, etc.) serve as useful templates for structuring such consultations? Should ASFA play a central role in constructing guidance documents for “best practices” in remote apheresis consultation, and create guidelines for various aspects of apheresis telemedicine? This presentation is a 50,000-foot view of this evolving, complicated, and “grey” area of medical care in the 21st century. We now have the capability to successfully leverage the expertise of transfusion and apheresis medicine specialists around the country to bring higher quality care to patients with challenging autoimmune diseases through remotely guiding their physicians and providing actionable recommendations in real-time. This session is an opportunity to articulate intelligent questions and conceptualize possible solutions about how to do this.

THERAPEUTIC APHERESIS IN CHILDREN: A PARENT’S EXPERIENCE Marty Gerhardt

Patient family speaking about their experience receiving therapeutic apheresis at the Hoxworth Blood Center.


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OPTIMIZATION OF SPECTRA OPTIA FOR GRANULOCYTE COLLECTIONSJose A. Cancelas, MD, PhD

Functioning granulocytes are a vital component of the defense system against bacterial and fungal infections in humans. For patients undergoing stem cell transplantation or induction for acute leukemia, modern intensive chemotherapy often results in prolonged periods of neutropenia, a major risk factor for severe bacterial and fungal infections. Despite the administration of granulocyte colony-stimulating factor and appropriate antimicrobials, systemic infections in patients with neutropenia are associated with extended hospital admission, organ damage, and a significant mortality, which in some series reaches >20%. Clinical experience and data from animal studies suggest that control of infection in these patients requires the recovery of bone marrow neutrophil production.

Several retrospective and early prospective studies have suggested that the efficacy of granulocyte transfusions in neutropenic patients may be proportional to the dose of granulocytes transfused. Doses of ≥1 × 109 granulocytes/kg body weight in daily transfusions seem to be required to treat or prevent infection. This dose requirement is difficult to achieve when heavier patients are treated because the usual level of granulocytes collected ranges from 10 to 70 × 109 granulocytes per transfusion.

In this presentation, we will analyze the retrospective and prospective, randomized studies on granulocyte efficacy and safety in the context of neutropenia therapy or prophylaxis, and studies comparing different collection efficiencies by older and newer apheresis devices. This presentation will also focus on the granulocyte collection efficiency of health donors subject to dexamethasone and G-CSF treatment, comparing a historic device with a newer version.

HEMATOPOIETIC AND MONONUCLEAR CELL COLLECTIONS FOR ADVANCED THERAPIES IN CHILDRENJoerg Krueger, MD

With advances in graft manipulation techniques and the development of innovative therapies using immune effector cells (IEC) such as CAR T-cells as well as gene therapeutic manipulation of hematopoietic stem cells (HPC), apheresis within the pediatric population is growing in demand. Timing of collection, choice of apheresis device, vascular access and

collection protocol determine success of the cell collection. Whereas timing of HPC collection is well established, timing of IEC collection for leukemia patients is still subject of research and requires consideration of lymphocyte count, blast percentage, prior chemotherapy and availability of vascular access. As the Spectra Optia (Optia) apheresis device has replaced the COBE Spectra (COBE) apheresis device in many centres we compared HSC and IEC collections on the COBE with the secondary chamber protocol (MNC) on the Optia device. We found that the collection efficiency (CE2) for HPCs was similar between the two devices and superior on the Optia for IECs. Adequate mononuclear cell products for CAR T-cells were successfully collected with a single procedure from all patients except one; CE2 was 58% on the Optia.

Recently a new continuous collection protocol (CMNC), which bypasses the second collection chamber, has been approved for the Spectra Optia. Although the CMNC protocol is easier to operate and the collection can be stopped at any time when the calculated target volume is reached, the CMNC tubing volume is significantly higher and requires blood priming of the system for more pediatric patients. In a prospective study we compared the two protocols and found comparable HPC CE2s between MNC and CMNC cohorts, however, the CMNC was more platelet sparing (p<0.01). Both protocols offer distinct advantages in the pediatric population and centers may choose between the protocols based on center preferences. If the CMNC protocol offers an advantage for IEC collections in children as shown in some adult studies remains to be seen.

LEUKOREDUCTION WITH THE SPECTRA OPTIAAnand Padmanabhan, MD, PhD, QIA

Anand Padmanabhan, MD, PhD, QIA will review complications and risks associated with hyperleukocytosis and detail current management strategies with a focus on leukodepletion by apheresis. The rationale and outcomes associated with this therapy will be discussed and technical information related to use of the Spectra Optia apheresis device, recently FDA-approved for this indication, will be presented.

TPE IN PEDIATRIC KIDNEY DISEASEStuart Goldstein MD, FAAP, FNKF

The provision of therapeutic apheresis to children is a technically challenging procedure, requiring trained personnel and an understanding of the disease processes


REGIONAL PROGRAMthat lead to the need for apheresis. Most apheresis protocols are derived from studies in adult patients, even though most studies are of limited sample size. The focus of this presentation will highlight the disease processes commonly treated with therapeutic apheresis in children, and to address the technical considerations pertinent to the provision of safe and effective apheresis in the pediatric setting. This presentation will use a case based format to highlight the unique needs of pediatric patients requiring therapeutic plasma exchange.

ISOVOLEMIC HEMODILUTION-RED BLOOD CELL EXCHANGE IN SICKLE CELL ANEMIA (SCA):Ravindra Sarode, MD

Despite the success of hydroxyurea in reducing complications of SCA, red blood cells (RBC) transfusion therapy remains standard of care for treatment and prevention of several conditions. Currently, indefinite RBC transfusion has been an established therapy based on randomized clinical trials for the primary prevention of cerebrovascular accidents (CVA); and previously it was shown to be effective in secondary prevention as well. Simple RBC transfusions can achieve these goals easily because it can be performed in any hospital, however, it causes severe iron overload despite chelation therapy, which is very expensive and has a poor compliance. Red blood cell exchange (RBCx) therapy can achieve the same goal of primary and secondary prevention, however, has advantage of preventing iron overload, especially if began early in childhood. Unfortunately, RBCx requires special equipment and technical skills, two venous accesses and uses more RBC units. A modified technique of initial isovolemic hemodilution step followed by RBCx (IHD-RBCx) has several advantages over standard RBCx because it can deplete HbS containing red cells more efficiently, uses fewer RBC units, may increase inter-procedure interval, and hence reduce total number of RBCx procedures per year. All these advantages could add up saving millions of dollars and improve quality of life of these patients. Our center has used IHD-RBCx for over 15 years. Currently, 46 adults are in RBCx program; 25 of them on IHD-RBCx and 13/25 are off of chelation. Apart from patients with stroke, we have offered this therapy for chronic pain crises and chronic leg ulcers. The challenge of dual venous access can be addressed by using dual-lumen subcutaneously implanted ports. Thus, IHD-RBCx program started early in the childhood could be a very effective therapy with no iron overload and better quality of life.

ROLE OF APHERESIS IN THE TREATMENT OF HYPERLIPIDEMIADavid Ward, MD

Selective LDL-apheresis, using either of the FDA-approved machines (Kaneka Liposorber® and Braun Plasmat Futura®), is effective and indicated in severe familial hypercholesterolemia (FH), particularly in homozygous FH (which causes coronary disease in childhood), in severe cases of heterozygous FH (which afflicts 1 in 500 adults), and in childhood “double heterozygous” cases. Controlled trials have compared LDL-apheresis plus statins and other conventional drug and dietary therapy, to the same without LDL-apheresis. Proven benefits of the addition of LDL-apheresis include improved longevity, lower rates of major cardiovascular events and regression of coronary atherosclerosis; also improvements in regional myocardial perfusion, exercise stress testing, cerebral blood flow and carotid artery atherosclerosis. However, the recent introduction of newer medications, particularly the PCSK9 inhibitors, has lowered LDL levels in many patients to the point they have been able to discontinue LDL-apheresis. The impact of these new drugs, and their use in combination with LDL-apheresis, will be discussed.

Excess of Lp(a) cholesterol (“lipoprotein little a”) may occur with or without elevated LDL levels, and is an independent risk factor for coronary and cardiovascular disease. Currently there are no medications to reduce Lp(a), but LDL-apheresis is effective.

LDL-apheresis with dextran sulfate adsorption has shown promise in treating focal segmental glomerulosclerosis (FSGS), a kidney disease that can recur after transplantation. The role of cholesterol reduction versus other possible mechanisms will be briefly discussed.

Finally hypertriglyceridemia (“chylomicronemia”) can cause acute pancreatitis and other life-threatening complications. Plasma exchange can acutely reduce excessive levels, but there is controversy regarding the effectiveness and place of apheresis in patient management.

EXTRACORPOREAL PHOTOPHERESIS: WHAT IS NEW?Matthew Montgomery, MD

This presentation will begin with a brief review of the history of photopheresis and transition into current instrumentation. Focus will then turn to current indications and theorized mechanisms of action. Finally it will cover new understanding


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of the mechanism of action and how ECP is being impacted by alternative and adjunctive treatments for a variety of conditions and what lies on the horizon for this powerful treatment modality.

GENE THERAPY FOR SICKLE CELL ANEMIA USING A MODIFIED GAMMA GLOBIN LENTIVIRUS VECTOR AND REDUCED INTENSITY CONDITIONING TRANSPLANTPunam Malik, MD

We generated an antisickling modified g-globin gene encod-ing lentiviral vector for gene therapy for sickle cell anemia (SCA). In preclinical studies, gene transfer into autologous hematopoietic stem and progenitor cells (HSC) in sickle mice following both myeloablative conditioning and reduced inten-sity conditioning showed correction of the sickle cell disease phenotype. Based upon these preclinical data, we embarked upon a Reduced Intensity Conditioning (RIC) Phase I/II Pilot Study on Gene Transfer in Patients with SCA with a modi-fied g-Globin Lentivector (NCT02186418), hypothesizing this approach will be safe, feasib le and efficacious; Moreover, RIC will have significantly less toxicity, costs, and be imple-mentable in many transplant centers, including those in some of the resource-poor countries, where supportive therapies for myeloablative transplants are unavailable and where ma-jority of SCA patients exist.

The study is open and enrolling adult patients with severe SCA. Two patients enrolled had CD34+ HSC were collected via bone marrow harvests and/or plerixafor mobilized leu-kopheresis. Patients received a single dose of IV melphalan (140mg/m2 BSA) followed by IV infusion of the g-globin gene modified-HSC. Patients were monitored for adverse events, engraftment, vector copy number (VCN), modified HbF (HbF*) expression and other parameters of SCA. Early results from SCA patients treated with gene therapy following RIC showed excellent safety, feasibility, with minimal post-transplant toxici-ty, rapid count recovery, and detection of vector-derived fetal hemoglobin (HbF*) expression. While these are early results, additional study data will demonstrate whether a level of HbF* will be reached that will provide consistent clinical ben-efit to patients with severe SCA. If this approach is successful, it will provide a safe and feasible gene therapy for SCA. The bench to bedside conception, preclinical development, and translation of gene therapy for SCA will be presented.

UTILIZING VIRUS-SPECIFIC T CELLS TO AUGMENT ANTIVIRAL IMMUNITY IN IMMUNOCOMPROMISED PATIENTSThomas Leemhuis, PhD

This presentation will review the Cincinnati experience producing a quadrivalent anti-viral cytotoxic T cell product from peripheral blood mononuclear cells in support of a phase 2 clinical trial at Cincinnati Children’s Hospital. After a brief introduction to the rationale for the trial, the procedures utilized for stimulation, expansion, and cryopreservation of this product will be reviewed in detail, as will the methods used for analyzing product safety and efficacy. Outcomes from the first 50 infusions of these cells for the treatment of Adenovirus EBV, CMV, and/or BK virus infections in immunocompromised patients shortly after hematopoietic stem cell transplant will also be reviewed.

QUALITY CONTROL FOR APHERESIS PRODUCTS TO BE USED IN ADVANCED IMMUNOTHERAPIESCarolyn Lutzko, PhD

Cell based personalized immunotherapies are in development. In these new therapies, either the patient’s own, or an independent donor’s immune cells are expanded and directed to target and kill cancer cells or virally infected cells. There are several immunotherapies that are FDA approved to treat cancer including Kymriah and Yescarta for the treatment of diffuse large B-cell lymphoma non-Hodgkin’s lymphoma respectively. In addition, there are many new therapies in clinical trials such as the use of dendritic cells for use as a tumor vaccine, viral specific T-lymphocytes to treat viral infections or post-transplant lymphoproliferative disorders in immunocompromised patients. For these applications, the source of the immune cells is from the peripheral blood or non-mobilized apheresis. The quality of the starting material is crucial to the success of these immunotherapies. This presentation will focus on the use of blood and apheresis products for these applications, and the quality control parameters of the immune cells required for their success.

2019

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OR FOCUS GROUP

An Educational and Networking Forum For Professionals in the Field of Apheresis Medicine

www.apheresis.org

PORTLAND, OREGON MAY 14-18, 2019

HILTON PORTLAND DOWNTOWN, PORTLAND, OREGON

ASFA’s 40th Annual Meeting

cincinnati, oh · 2018. 8. 17. · cincinnati, oh september 7, 2018 rgional program asfa 2018...

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