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Scientific Symposium on Bone Marrow Failure and Myelodysplastic Syndromes: From Discovery to Future Strategies


Scientific Symposium on Bone Marrow Failure and Myelodysplastic Syndromes: From Discovery to Future Strategies Banner

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Add to Calendar Scientific Symposium on Bone Marrow Failure and Myelodysplastic Syndromes: From Discovery to Future Strategies 10/24/2026 6:00:00 PM 10/27/2026 3:00:00 PM America/Chicago For More Details: https://stjude.cloud-cme.com/www.stjude.org/napaac Description: St. Jude Children's Research Hospital will host the Scientific Symposium on Bone Marrow Failure and Myelodysplastic Syndromes: From Discovery to Future Strategies on October 25–27, 2026, in partnership with the North American Pediatric Aplastic Anemia Consortium (NAPAAC). NAPAAC is a research consortium established in 2011 to advance research and therapies in pediatric bone marrow failure and myelodysplastic sy... St. Jude Children's Research Hospital false MM/DD/YYYY


Date & Location
Saturday, October 24, 2026, 6:00 PM - Tuesday, October 27, 2026, 3:00 PM, St. Jude Children's Research Hospital, Memphis, TN

Overview

St. Jude Children's Research Hospital will host the Scientific Symposium on Bone Marrow Failure and Myelodysplastic Syndromes: From Discovery to Future Strategies on October 25–27, 2026, in partnership with the North American Pediatric Aplastic Anemia Consortium (NAPAAC). NAPAAC is a research consortium established in 2011 to advance research and therapies in pediatric bone marrow failure and myelodysplastic syndromes.

About the Symposium

Bone marrow failure (BMF) disorders and myelodysplastic syndromes (MDS) represent over 20 disease entities with at least 150 causative genes. Despite rapid progress in genetic diagnostics, developing targeted therapies and deploying effective surveillance remain a critical challenge.

This symposium brings together >60 academic centers who are members of the NAPAAC consortium. In addition, global leaders in bone marrow failure from Europe, Asia, South America, and Australia will share discoveries and clinical insights on genetics, transplantation, gene therapy, predisposition syndromes, and emerging treatment strategies.

Program Highlights

Sunday, October 25 (from 3pm):

Opening session on international collaboration and patient advocacy.

Monday, October 26 (8am – 6pm) and Tuesday, October 27 (8am – 1pm):

Scientific sessions on hematopoiesis, genomics, disease mechanisms, and emerging therapies including novel transplant strategies and genome engineering. Poster session to showcase basic, translational and clinical research within NAPAAC.

Networking and Collaboration:

This interactive event provides opportunities to network with peers, engage in strategic discussions on emerging treatment approaches, and connect across the global community. Dedicated time will be offered for NAPAAC working groups to discuss progress and define future priorities.

Registration fees and deadlines:

Registration Type (in person)

Early Bird
by August 14, 2026

Late Registration
August 14 – October 10, 2026

St. Jude Employee

Free

Free

Invited Speaker

Free

Free

NAPAAC Center - Trainee (student, postdoc, clinical fellow, resident)

Free

$200

NAPAAC Center - Other (including PI, physician, APP, genetic counselor)

$70

$250

St. Jude Affiliate

$70

$250

External: Academic

$400

$500

External: All Others, Including Industry

$800

$1,000

Virtual participation options will be added in August.


Objectives
After participating in this educational activity, you should be able to:

  1. To bring together expert physicians, scientists, and patient group advocates for a scientific conference that bridges innovation and strategy for BMF research and clinical care for the next decade in the United States. NAPAAC has pledged its support for this initiative, recognizing the critical need for enhanced collaboration and strategic planning. Historically, NAPAAC meetings occurred irregularly as brief sessions before ASPHO annual meetings, with limited scope and no dedicated time for strategic planning or extended collaboration. This symposium represents the first standalone, dedicated NAPAAC conference designed for collaboration and strategic planning.

Accreditation

St. Jude Children's Research Hospital is accredited by the Accreditation Council for Continuing Medical Education (ACCME) to provide continuing medical education for physicians. 

St. Jude Children’s Research Hospital designates this Live Activity for a maximum of 12.50 AMA PRA Category 1 CreditsTM. Physicians should only claim credit commensurate with the extent of their participation in the activity.


Credits
AMA PRA Category 1 Credits™ (12.50 hours), Non-Physician Attendance (12.50 hours)

Target Audience
Specialties - Bone Marrow Transplant/Cellular Therapy, Epidemiology and Cancer Control, Global Pediatric Medicine, Hematology, Hospitalist, Infectious Diseases, Medicine, Nurse Practitioner, Nursing, Oncology, Other, Palliative Care, Pathology, Pharmaceutical Services, Physician Assistant


Keywords: LIVECME

Hotel Accommodations

The Peabody Memphis

149 Union Avenue, Memphis, TN 38103

Group Rate: $199/night

Group Name: St. Jude NAPAAC Scientific Symposium

Reservations: 1-800-PEABODY

Reservation Deadline: October 2, 2026

Please reference the group name when booking to receive the discounted rate.

Booking link: https://reservations.peabodymemphis.com/95096?groupID=5315246

The faculty for this activity are listed below. Click to view the faculty member's financial disclosure information and bio. 

Mitigation of Relevant Financial Relationships


St Jude Children's Research Hospital adheres to the ACCME’s Standards for Integrity and Independence in Accredited Continuing Education. Any individuals in a position to control the content of a CE activity, including faculty, planners, reviewers, or others, are required to disclose all financial relationships with ineligible entities (ie, companies whose primary business is producing, marketing, selling, re-selling, or distributing health care products used by or on patients). All relevant conflicts of interest have been mitigated prior to the commencement of the activity.

Serine Avagyan, MD, PhD
Pediatric Hematology
UCSF Helen Diller Family Comprehensive Cancer Center.
San Francisco, CA
Daira Babushok, MD, PhD
Assistant Professor of Medicine
Perelman School of Medicine, University of Pennsylvania
Philadelphia, PA
Rafael Balceiro, MD
Pediatric Hematologist-Oncologist
Barretos Children’s Cancer Hospital, Hospital de Amor
Barretos, São Paulo, Brazil
Dana Bellissimo, MD, PhD
Pediatric Hematology/Oncology Fellow
Children’s Hospital of Philadelphia
Philadelphia, PA
Alison Bertuch, MD
Chair of Pediatric Hematology-Oncology
Baylor College of Medicine and Texas Children’s Cancer and Hematology Center
Houston, TX
Faculty Photos
Nickhill H Bhakta, MD
Assistant Member
St. Jude Children's Research Hospital
Memphis, TN
Senthil Velan Bhoopalan, MBBS
Instructor
St. Jude Children's Research Hospital
Cordova, TN
Michelle Boals, NP
Nurse Practitioner
St. Jude Children's Research Hospital
Memphis, TN
Carmen Bonfim, MD, PhD
Senior Consultant
Hospital Pequeno Principe
Curitiba, Brazil
Derek Chan, MD, PhD, FRCPC
Pediatric Hematology/Oncology Fellow
The Hospital for Sick Children and University of Toronto
Toronto, Canada
Karen M Chisholm, MD, PhD
Hematopathologist, Professor
Seattle Children's Hospital
Seattle, WA
Seth Corey, MD, MPH
Physician-Scientist
Cleveland Clinic
Cleveland, OH
Agnieszka Czechowicz, MD, PhD
Assistant Professor of Pediatrics
Stanford University School of Medicine
Stanford, CA
Jill de Jong, MD, PhD
Associate Professor of Pediatrics
University of Chicago Medicine and Comer Children’s Hospital
Chicago, IL
Craig Forester, MD, PhD
Boettcher Investigator/Associate Professor
University of Colorado Anschutz Medical Campus
Aurora, CO
Lucy Fox, BCom/BSc, MBBS, FRACP, FRCPA, DMedSci
Associate Professor
Peter MacCallum Cancer Centre
Melbourne, Australia
Anita Frisanco Oliveira, MD
Pediatric Hematologist
Barretos Children´s Cancer Hospital
Barretos, São Paulo, Brazil
Biju George, MBBS, MD, DM
Director & Professor of Haematology
Christian Medical College Vellore
Vellore, India
Nick Gloude, MD, FAAP
Director, Bone Marrow Failure Program
Rady Children’s Hospital–San Diego
San Diego, CA
Lucy Godley, MD, PhD
Professor of Medicine
Northwestern Univerity
Chicago, IL
Nathan Gray, Physician Assistant
Physician Assistant
St. Jude Children's Research Hospital
Memphis, TN
Jane Hankins, MD
Member
St. Jude Children's Research Hospital
Memphis, TN
Eszter Hars, PhD
Founder and CEO, SDS Alliance
Shwachman-Diamond Syndrome Alliance
Shaoyan Hu, MD, PhD
Professor & Director
Children’s Hospital of Soochow University
Suzhou, China
Gaurav Joshi, PhD
Postdoctoral Fellow
University of Texas Southwestern Medical Center
Dallas, TX
Alyssa Kennedy, MD PHD
LLH Conference
St. Jude Children's research hospital
Memphis, TN
Jane Koo, MD
Physician / Assistant Professor of Pediatrics
Cincinnati Children’s Hospital Medical Center
Cincinnati, OH
Sara Lewis, MS, CGC
Genetic Counselor
St. Jude Children's Research Hospital
Memphis, TN
Riccardo Masetti, MD, PhD
Associate Professor of Pediatrics
University of Bologna / Sant'Orsola-Malpighi University Hospital
Bologna, Italy
Wojciech Mlynarski, MD, PhD
Professor & Head of Department
The Medical University of Lódz
Lódz, Poland
Hideki Muramatsu, MD, PhD
Associate Professor
Nagoya University Graduate School of Medicine
Nagoya, Japan
Kasiani Myers, MD
Professor of Pediatrics
Cincinnati Children’s Hospital Medical Center and University of Cincinnati College of Medicine
Cincinnati, OH
Taizo Nakano, MD
Associate Professor of Pediatrics
University of Colorado Anschutz Medical Campus School of Medicine
Aurora, CO
Susana Navarro, PhD
Senior Researcher / Research Scientist
CIEMAT (Center for Energy, Environmental and Technological Research)
Madrid, Spain
Ellis J Neufeld, MD
EVP/Clinical Director
St. Jude Children's Research Hospital
Memphis, TN
Charlotte Niemyer, MD
Professor Emerita
Medical Center – University of Freiburg (University Medical Center Freiburg)
Freiburg, Germany
Bhavisha Patel, MD
Associate Research Physician
National Heart, Lung and Blood Institute
Jessica Pollard, MD
Clinical Director
Dana-Farber/Boston Children’s Cancer and Blood Disorders Center / Harvard Medical School
Boston, MA
Michael Pulsipher, MD
Professor of Pediatrics, Division of Hematology/Oncology
University of Utah / Intermountain Primary Children’s Hospital
Salt Lake City, UT
Kristie Ramos, MD
Instructor of Pediatrics, University of Cincinnati College of Medicine
Cincinnati Children’s Hospital Medical Center
Cincinnati, OH
Raul C Ribeiro, MD
Member
St. Jude Children's Research Hospital
Memphis, TN
Charles Roberts, MD
EVP/Cancer Center Director
St. Jude Children's Research Hospital
Memphis, TN
Sushree Sahoo, PhD
Assistant Professor
Cancer Research Center–D.C., Fralin Biomedical Research Institute at VTC
Washington, DC
Anjali Sharathkumar, MBBS, MD, MS
Clinical Professor of Pediatrics
University of Iowa Carver College of Medicine / University of Iowa Health Care
Iowa City, IA
Faculty Photos
Akshay Sharma, MBBS
Assistant Member
St. Jude Children's Research Hospital
Memphis , TN
Akiko Shimamura, MD, PhD
Professor of Pediatrics; Director, Bone Marrow Failure and Myelodysplastic Syndrome (MDS) Program
Dana-Farber/Boston Children’s Cancer and Blood Disorders Center/Harvard Medical School
Boston, MA
Cristian Sotomayer, MD
Physician, Pediatric Stem Cell Transplant and Oncology Unit
UC CHRISTUS Health Network (Red de Salud UC CHRISTUS)
Santiago, Chile
Brittany Stewart, CGC PhD
Hematology/Oncology Genetic Counselor
Cleveland Clinic
Cleveland, OH
Brigitte Strahm, MD
Deputy Head; Head of Pediatric Stem Cell Transplantation; Senior Physician
Medical Center – University of Freiburg
Freiburg, Germany
Shruthi Suryaprakash, MD
Clinical MD Fellow
St. Jude Children's Research Hospital
Memphis, TN
Jennie Vagher, MD
Senior Manager of Genetic Counseling Research and Adjunct Assistant Professor
Huntsman Cancer Institute, University of Utah
Salt Lake City, UT
Michael Vido, MD
Instructor of Pediatrics
Perelman School of Medicine, University of Pennsylvania
Adrianna Vlachos, MD
Chief of Hematology
Johns Hopkins All Children’s Hospital
St. Petersburg, FL
Yang Wan, MD, PhD
Associate Chief Physician
Chinese Academy of Medical Sciences & Peking Union Medical College
Tianjin, China
Julia Warren, MD, PhD
Assistant Professor of Pediatrics (Hematology)
Children’s Hospital of Philadelphia
Philadelphia, PA
Franziska Watcher, MD
Attending Physician
Dana-Farber/Boston Children’s Cancer and Blood Disorders Center
Boston, MA
Mitchell J. Weiss, MD
Chair/Member
St. Jude Children's Research Hospital
MEMPHIS, TN
Jonathan Yen, PhD
Associate Member, Director, Therapeutic Genome Engineering (TGEn)
St. Jude Children's Research Hospital
Memphis, TN
Helena Yu, MD
Attending Physician and Ning Zhao & Ge Li Endowed Chair
Memorial Sloan Kettering Cancer Center
New York, NY
Haijiao Zhang, MD
Assistant Professor
OHSU Knight Cancer Institute, Oregon Health & Science University School of Medicin
Portland, OR

Saturday, October 24, 2026
Sunday, October 25, 2026
NAPAAC Business Meeting
12:30PM - 1:45PM


12:30–1:45   Closed NAPAAC Business Meeting

By invitation only · MTC auditorium

Strategic vision, collaborations, future of the registry.



Collaborations and Working Groups
1:45PM - 3:00PM


Open for NAPAAC members, EWOG chairs invited ·

MTC auditorium



Patient Advocacy Strategy Session - (non-accredited)
2:00PM - 3:30PM


By invitation only
Nine patient advocacy groups discuss strategy.


Session 1 — Collaborations to Strengthen BMF/MDS Research
4:00PM - 6:00PM
Nickhill H Bhakta, MD
Jane Hankins, MD
Yang Wan, MD, PhD (Speaker)
Charlotte Niemyer, MD (Moderator)
Wojciech Mlynarski, MD, PhD (Moderator)
Craig Forester, MD, PhD (Moderator)
Mitchell J. Weiss, MD (Moderator)


Peabody Ballroom
Chairs: Wojciech Mlynarski & Craig Forester


4:00   Welcome & housekeeping (Mitch Weiss, MD, PhD - Chair, Department of Hematology, St Jude) 
4:10   St Jude Global: Implementation of global genomic diagnostic platform (Nickhil Bhakta, USA)
4:40   St Jude Global: International registry alignment: AI-supported approach (Jane Hankins, USA)  
           Break 
5:10   Childhood Inherited Bone Marrow Failure in China  (Yang Wan, China) 
5:35   Pediatric AA and MDS: learning from collaborations within the EWOG group  (Charlotte Niemeyer, Germany)


 



Welcome Dinner (Peabody Hotel): Past, Present, Future of NAPAAC and Patient Advocacy Collaborations
6:30PM - 9:00PM
Akiko Shimamura, MD, PhD (Speaker)
Taizo Nakano, MD (Moderator)


6:30 reception, followed by dinner from 7:00 · Moderator: Taizo Nakano

Past, present and future of NAPAAC (Akiko Shimamura, USA)

Patient advocacy groups: research directions & collaborations • AAMDS International Foundation

RUNX1 Research Program

GATA2 Patient Advocacy 

SAMD9L Foundation

SDS Alliance

National Neutropenia Network

Team Telomere

DBA Foundation US & DBA Canada



Monday, October 26, 2026
Registration
7:30AM - 8:15AM

Session 2 — Genetics / Genomics
8:15AM - 10:20AM
Lucy Fox, BCom/BSc, MBBS, FRACP, FRCPA, DMedSci (Speaker)
Nick Gloude, MD, FAAP (Moderator)
Lucy Godley, MD, PhD (Speaker)
Julia Warren, MD, PhD (Moderator)
Charles Roberts, MD
Kristie Ramos, MD (Speaker)
Haijiao Zhang, MD (Speaker)
Daira Babushok, MD, PhD (Speaker)
Gaurav Joshi, PhD (Speaker)


Chairs: Julia Warren & Nick Gloude


8:15 Welcome (Charles W. M. Roberts, MD, PhD - St Jude CEO-elect) 
8:30 Increasing the diagnostic yield in BMF using comprehensive genomics (Lucy Fox, Australia) 
8:55 Lessons for pediatricians from adult-onset marrow failure & germline predisposition (Lucy Godley, USA)
9:20 Abnormal telomere maintenance and LINE-1 dysregulation in Fanconi anemia: a 20-year longitudinal study (Kristie Ramos, Cincinnati) 
9:35 NGDN variants impair erythropoiesis in Diamond-Blackfan anemia (Gaurav Joshi, Dallas) 
9:50 Haploinsufficiency of copy number dosage-sensitive essential genes drives bone marrow failure (Haijiao Zhang, Portland)
10:05 Predictive aplastic score system (PASS): a new clinical tool to distinguish acquired AA from IBMFS in adults using readily available clinical data (Daria Babushok, Philadelphia) 



Break
10:20AM - 10:50AM

Session 3 — Disease Mechanisms & Experimental Models
10:50AM - 12:30PM
Hideki Muramatsu, MD, PhD
Serine Avagyan, MD, PhD (Moderator)
Seth Corey, MD, MPH (Moderator)
Craig Forester, MD, PhD
Alison Bertuch, MD
Helena Yu, MD
Michael Vido, MD
Dana Bellissimo, MD, PhD


Chairs: Serine Avagyan & Seth Corey
10:50 Two is better than one: aldehyde clearance in BMF (Hideki Muramatsu, Japan) 
11:15 Implementation of the LoxCode barcoding system as a tool to study normal and clonal hematopoiesis (Dana Bellissimo, Philadelphia) 
11:30 DNA-encoded chemistry library screening to identify telomere-elongating lead small molecules (Alison Bertuch, Houston) 
11:45 Fragmentation patterns of human telomeric chromatin in plasma cfDNA (Craig Forester, Aurora)  
12:00 Proteostasis remodeling across development defines fetal, neonatal and adult hematopoietic stem cell states (Helena Yu, San Diego) 
12:15 New CLPB mouse model revealing mitochondrial proteostatic stress contributes to dysregulated granulopoiesis (Michael Vido, Philadelphia) 


Session 4 — Development of New Therapies
1:40PM - 3:05PM
Susana Navarro, PhD
Alyssa Kennedy, MD PHD (Moderator)
Anjali Sharathkumar, MBBS, MD, MS (Moderator)
Shruthi Suryaprakash, MD
Agnieszka Czechowicz, MD, PhD
Kasiani Myers, MD
Derek Chan, MD, PhD, FRCPC


Chairs: Alyssa Kennedy & Anjali Sharathkumar
1:40 Development of gene therapies in BMF (Susana Navarro, Spain)  
2:05 Development of lentiviral gene therapy for RPS19-mutated Diamond-Blackfan anemia syndrome (Shruthi Suryaprakash, Memphis) 
2:20 In utero therapies for Fanconi anemia (Agnieszka Czechowicz, San Francisco) 
2:35 Prolonged persistence of hematopoiesis with telomere extension after infusion of autologous CD34+ cells exposed to ZSCAN4 in patients with telomere biology disorders (Kasiani C. Myers, Cincinnati) 
2:50 Investigating approaches for recovering thrombopoietin receptor signaling in congenital amegakaryocytic thrombocytopenia (Derek Chan, Toronto) 


Break
3:05PM - 3:25PM

Session 5 — Global Collaboration Towards Better Patient Outcomes
3:25PM - 5:00PM
Biju George, MBBS, MD, DM
Carmen Bonfim, MD, PhD
Senthil Velan Bhoopalan, MBBS (Moderator)
Cristian Sotomayer, MD (Moderator)
Adrianna Vlachos, MD
Brittany Stewart, CGC PhD
Rafael Balceiro, MD


Chairs: Cristian Sotomayor & Senthil Bhoopalan
3:25 Improving transplant outcomes in FA and TBD: the Brazilian experience (Carmem Bonfim, Curitiba, Brazil)
3:50 Old vs new: approaches to transplanting BMF and MDS (Biju George, India) 
4:15 Results of the International Diamond Blackfan Anemia Syndrome Cancer Consortium (Adrianna Vlachos, St. Petersburg, FL)
4:30 Building consensus for the management of RUNX1 familial platelet disorder: results from an international modified-Delphi survey (Brittany Stewart, Cleveland)
4:45 Advanced pediatric MDS: clinical and genetic characterization of patients registered in the Brazilian cooperative group of pediatric MDS (Rafael Balceiro, São Paulo, Brazil)


Poster Session — St Jude
5:00PM - 6:15PM


St Jude Marlo Thomas Center Foyer
Shuttle to brewery
6:30PM - 7:00PM

Dinner: Soul and Spirits brewery
7:00PM - 10:00PM

Tuesday, October 27, 2026
Registration
7:30AM - 7:45AM

Meet Your Expert — Pastries & Coffee
7:45AM - 8:45AM



In person only, no virtual option · Rooms at St Jude MTC · whiteboards with dry-erase markers

  • Genetics of neutropenia disorders (Julia Warren, USA)
  • DNA repair disorders and cancer risk (Wojciech Mlynarski, Poland)
  • Diagnostic approaches for MDS/JMML (Anita Friscanco-Oliveira, Brazil)
  • Writing a successful grant -  tips and tricks (Seth Corey, USA)
  •  Principles of genome engineering: from CRISPR to gene editing (Jonathan Yen, USA)
  • TBA (Eszter Hars, USA)

Session 6 — MDS
8:45AM - 10:50AM
Raul C Ribeiro, MD (Moderator)
Shaoyan Hu, MD, PhD
Alyssa Kennedy, MD PHD
Karen M Chisholm, MD, PhD
Riccardo Masetti, MD, PhD
Franziska Watcher, MD
Sushree Sahoo, PhD
Jessica Pollard, MD (Moderator)


 Chairs: Raul Ribeiro & Jessica Pollard

9:00 Unifying the classification of pediatric MDS (Karen Chisholm, Seattle)

9:20 Pre-HSCT strategies in pediatric MDS (Riccardo Masetti, Italy - virtual)

9:45 Use of novel agents in pediatric MDS/AML (Shaoyan Hu, China)

10:05 Results from a phase II clinical trial of CPX-351 in patients with secondary myeloid malignancies (Alyssa Kennedy, Memphis) 


10:20 Post-HSCT maintenance with low-dose decitabine and G-CSF in children and young adults with high-risk MDS and related myeloid malignancies (Franziska Wachter, Boston)


10:35 Germline and somatic landscape of monosomy 7 in pediatric MDS (Sushree Sahoo, Memphis/Virginia) 



Break
10:50AM - 11:10AM

Session 7 — Aplastic Anemia
11:10AM - 12:40PM
Brigitte Strahm, MD
Serine Avagyan, MD, PhD
Jill de Jong, MD, PhD (Moderator)
Taizo Nakano, MD (Moderator)
Bhavisha Patel, MD
Jane Koo, MD
Akshay Sharma, MBBS


Chairs: Jill De Jong & Taizo Nakano
11:10 Transplant in pediatric SAA and MDS: lessons from EWOG-SAA/MDS (Brigitte Strahm, Germany) 
11:40 Immunosuppressive therapies in SAA: yesterday, today and tomorrow (Bhavisha Patel, Bethesda)  
12:10 Radiation-free reduced-intensity conditioning HSCT achieves excellent outcomes for pediatric severe aplastic anemia: a multi-center experience (Jane Koo, Cincinnati) 
12:25 Haplo-transplants and beyond: what comes next? (Akshay Sharma, Memphis) 


Farewell & Lunch
12:40PM - 2:00PM

Complete a Financial Disclosure

Complete a Presentation Release Form

Start or continue a CME Activity Application

St. Jude Children’s Research Hospital is accredited by the Accreditation Council for Continuing Medical Education (ACCME) to provide continuing medical education for physicians.

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