SCD Curative Therapies
| Select | Protocol | ID |
|---|---|---|
|
#851202 |
|
|
#851201 |
|
|
#850801 |
|
|
#850201 |
|
|
#850901 |
|
|
#850101 |
|
|
#850301 |
|
|
#850601 |
|
|
#850501 |
|
|
#850401 |
|
|
#851101 |
|
|
#851001 |
|
|
#850701 |
Release Date:
August 16, 2021
Scope of Collection
| Element Considered | Final Measures in Toolkit |
|---|---|
| Indications for Transplant | |
| Pretransplant Evalution | Prothrombin Time |
| Left Ventricular Global Longitudinal Strain by Speckle Tracking Echocardiography | |
| Determination of Liver Iron Concentration by R2* | |
| Determination of Liver Iron Concentration by Ferriscan | |
| Determination of Liver Fibrosis by Magnetic Resonance Elastography (MRE) (Supplemental Information) | |
| Recurrent Priapism | |
| Psychosocial Assessment of Candidates for Transplantation | |
| Fibrosis Staging of Liver Biopsy (Supplemental Information) | |
| Determination of Liver Fibrosis by Transient Elastography (Supplemental Information) | |
| Myocardia Iron Estimation Using T2* MRI (Supplemental Information) | |
| Description of transplant conditioning regimen | Sickle Cell Disease Transplant Conditioning Regimen |
| Transplant stem cell source | Sickle Cell Disease Stem Cell Source and Cell Manipulation |
| Effect of transplant on donor derived erythropoiesis | |
| Endpoints for curative therapy (complications) | White Cell Chimerism and Graft Failure |
| Graft versus Host Disease | |
| Pretransfusion evaluation | |
| Endpoints for Transfusion | Pre-transfusion Antibody Testing |
| Transfusion Reaction Reporting | |
| Thyroid Stimulating Hormone (hTSH) in Serum | |
| Endpoints for transplant (complications) | Determination of Liver Iron Concentration by R2* |
| Determination of Liver Iron Concentration by Ferriscan | |
| Determination of Liver Fibrosis by Magnetic Resonance Elastography (MRE) (Supplemental Information) | |
| Fibrosis Staging of Liver Biopsy (Supplemental Information) | |
| Determination of Liver Fibrosis by Transient Elastography (Supplemental Information) | |
| Myocardia Iron Estimation Using T2* MRI (Supplemental Information) |
Working Group Roster:
WG Members:
|
Ross Fasano, MD, Co-Chair Emory University, Children's Healthcare of Atlanta Atlanta, GA |
Matthew Hsieh, MD, Co-Chair National Heart, Lung, and Blood Institute (NHLBI) Bethesda, MD |
|
Stella Chou, MD Children's Hospital of Pennsylvania Philadelphia, PA |
Julie Kanter, MD University of Alabama, Birmingham Birmingham, AL |
|
Emily Meier, MD Indiana Hemophilia and Thrombosis Center Indianapolis, IN |
Rene Romero, MD Emory University, Children's Healthcare of Atlanta Atlanta, GA |
|
Elizabeth Stenger, MD UPMC Children's Hospital of Pittsburgh Pittsburgh, PA |
Santosh Saraf, MD University of Illinois Urbana-Champaign, IL |
Sickle Cell Disease Panel Liaison, WG Manager and WG Supervisor:
|
Wayne Huggins, PhD, WG Supervisor RTI International Research Triangle Park, NC |
Lauren Wood, MS, WG Manager RTI International Research Triangle Park, NC |
Principal Investigator:
|
Carol M. Hamilton, PhD RTI International Research Triangle Park, NC |
Co-Investigator:
|
Tabitha P. Hendershot, BA RTI International Washington, DC |
Consensus Coordinator :
|
Deborah Maiese, MPA RTI International Research Triangle Park, NC |
Project Manager:
|
Amanda Riley, PMP, MBA RTI International Research Triangle Park, NC |
NIH National Heart, Lung and Blood Institute Liaison:
|
Traci Mondoro National Heart, Lung, and Blood Institute (NHLBI) Bethesda, MD |
Project Scientist:
|
Erin M. Ramos, PhD, MPH National Human Genome Research Institute (NHGRI) Bethesda, MD |
Supplemental Information includes protocols that the Working Group or Expert Review Panel considered useful and highly relevant to the domain but do not meet the criteria for inclusion in the Toolkit.
Additional Information:
There is no additional information for this collection.