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Protocol - Sickle Cell Disease Stem Cell Source and Cell Manipulation

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Description

This protocol uses existing medical records to document information about the stem cell donor, the doner’s relationship to the patient, the place in the body from which the stem cells were collected, human leukocyte antigen match or mismatch, and any gene therapy or other drug treatment administered to the stem cells before their transplantation in a patient with sickle cell disease. The first part of the protocol includes six questions from the Center for International Blood and Marrow Transplant Research CIBMTR Data Collection Form 2400 Pre-Transplant Essential Data (Pre-TED). The second part of the protocol includes questions from the Drug Product Case Report Form from the Cure Sickle Cell Disease Initiative (CureSCi) Common Data Element Project.

Specific Instructions

In Question 2 below, PBSC stands for peripheral blood stem cell.

Protocol

Donor Information for this HCT

(from Center for International Blood & Marrow Transplant Research form 2400)

1. Specify donor

   [ ] Autologous

   [ ] Allogenic, related

   [ ] Allogenic, unrelated

2. Specify product type (check all that apply)

   [ ]  Bone marrow

   [ ]  PBSC

   [ ]  Single cord blood unit

   [ ] Other product

                 Specify other product ___________________

3. Is the product genetically modified? (if autologous, go to the Drug Product Case Report Form below. If allogeneic related, go to question 4. If allogeneic unrelated, go to question 7.)

   [ ] Yes

   [ ] No

         4. If allogenic, specify the related donor type

            [ ] Syngeneic (monozygotic twin)

            [ ] HLA-identical sibling (may include non-monozygotic twin)

            [ ] HLA-matched other relative (does NOT include a haplo-identical donor)

            [ ] HLA-mismatched relative

         5. Specify the biological relationship of the donor to the recipient

            [ ] Mother

            [ ] Father

            [ ] Child

            [ ] Sibling

            [ ] Fraternal twin

            [ ] Maternal aunt

            [ ] Maternal uncle

            [ ] Maternal cousin

            [ ] Paternal aunt

            [ ] Paternal uncle

            [ ] Paternal cousin

            [ ] Grandparent

            [ ] Grandchild

            [ ] Other biological relative

                    Specify other biological relative:_______________

         6. Degree of mismatch (related donors only)

            [ ] HLA-mismatched 1 allele

            [ ] HLA-mismatched ≥ 2 alleles (does not include haplo-identical donor)

7. Specify unrelated donor type

   [ ] HLA matched unrelated

   [ ] HLA mismatched unrelated

Drug Product CRF (only for Autologous Donor Products)

(from Cure Sickle Cell Disease Initiative Common Data Element (CureSCi CDE) Project)

Drug Product Starting Material:

      [ ] Hematopoietic Progenitor Cell, Apheresis (HPC-A)

      [ ] Hematopoietic Progenitor Cell, Marrow (HPC-M)

      [ ] Hematopoietic Progenitor Cell, Cord Blood (HPC-C)

Manufacture Information

Date of Drug Product Manufacture__________________

        [ ] Proportion of CD34+ cells _________

        [ ] Total Colonies Formed: ______ per _______ cells

Expression of globin chain of interest:

       [ ] Qualitative Assay: [ ] Positive [ ] Negative

 [ ] Quantitative Assay: ___________

Were any additional drug product potency assays performed on drug product?

        [ ] Yes        [ ] No         

  If yes, describe and report result________________________________

Type of Modification:

  [ ] Integrating Vector

  [ ] Gene Editing Nuclease

  [ ] Stem Cell Expansion

POPULATE SECTION A FOR INTEGRATING VECTOR CELL PRODUCT

POPULATE SECTION B FOR GENE EDITED CELL PRODUCT

Section A

Type of Integrating Vector: [ ] Retrovirus [ ] Lentivirus [ ] Other______________

     [ ] Drug Product VCN______

     [ ] Percentage of cells positive for integrating vector _________

     [ ] Drug Product VCN/cell ________

Section B

Type of Editing: [ ] CRISPR-Cas 9 [ ] ZFN [ ] TALEN  [ ] Cytosine Base Editing

[ ]  Other______________

Mode of nuclease delivery: [ ] mRNA by EP [ ] RNP by EP [ ] AAV [ ] Other

gRNA target gene_________________ Mode of Delivery___________

Donor Template used? [ ] Yes [ ] No

If Yes, mode of delivery: [ ] ssODN [ ] AAV   [ ] Other [ ] Drug Product:

On-target Indels______  Verified off-target indels_________

Conversion _______%

 Copyright© 2021 National Marrow Donor Program and the Medical College of Wisconsin, Inc.

      

Availability

Available

Personnel and Training Required

Personnel who are trained in performing medical record review

Equipment Needs

None

Requirements
Requirement CategoryRequired
Major equipment No
Specialized training No
Specialized requirements for biospecimen collection No
Average time of greater than 15 minutes in an unaffected individual No
Mode of Administration

Medical record abstraction

Lifestage

Infant, Toddler, Child, Adolescent, Adult, Senior, Pregnancy

Participants

All ages

Selection Rationale

The Sickle Cell Disease Curative Therapies Working Group selected questions from the Center for International Blood and Marrow Transplant Research (CIBMTR) and the Cure Sickle Cell Disease Initiative Common Data Element Project as the best standardized methodologies for collecting data on stem cell source and cell manipulation. CIBMTR forms, which are commonly used in stem cell transplantation research, were developed with the international transplant community to establish a standard set of data elements to be collected for all transplant recipients. Additionally, there are comprehensive Forms Instruction Manuals to help investigators complete the questions. The Cure Sickle Cell Initiative Drug Product Case Report Form was developed specifically for use in sickle cell disease and supplements the questions from CIBMTR.

Language

English

Standards
StandardNameIDSource
caDSR Common Data Elements (CDE) Product Tissue Donor Type 5089291 CDE Browser
caDSR Common Data Elements (CDE) Hematopoietic Cell Graft Type Name 2730912 CDE Browser
caDSR Common Data Elements (CDE) Hematopoietic Cell Graft Other Type Specify 2527917 CDE Browser
caDSR Common Data Elements (CDE) Product Genetic Modification Occurrence Indicator 5085374 CDE Browser
caDSR Common Data Elements (CDE) Hematopoietic Cell Tissue Donor Relationship Type 3924953 CDE Browser
caDSR Common Data Elements (CDE) Tissue Donor Recipient Biological Relative Type 3870112 CDE Browser
caDSR Common Data Elements (CDE) Tissue Donor Recipient Biological Relative Specify Text 3972451 CDE Browser
caDSR Common Data Elements (CDE) Relative Tissue Donor MHC Class I Protein MHC Class II Protein Mismatch Type 2686055 CDE Browser
caDSR Common Data Elements (CDE) Unrelated Tissue Donor Type 6436759 CDE Browser
Derived Variables

None

Process and Review

Not applicable.

Protocol Name from Source

Cure Sickle Cell Initiative (CureSCi) Drug Product Case Report Form and Center for International Blood and Marrow Transplant Research (CIBMTR) Form 2400 (R8.0) Pre-Transplant Essential Data, 2021

Source

Center for International Blood and Marrow Transplant Research. (2021, January). Pre-Transplant Essential Data (Pre-TED) Form (CIBMTR Form 2400; Revision 8.0), Questions 46–54. National Heart, Lung, and Blood Institute.

National Heart, Lung, and Blood Institute Cure Sickle Cell Disease Initiative Common Data Element (CDE) Project, Genetics/Assays Working Group, Transplant Conditioning Regimen Case Report Form. (2020). Transplant Conditioning Regimen Case Report Form.

General References

Lee, C. J., Savani, B. N., Mohty, M., Labopin, M., Ruggeri, A., Schmid, C., Baron, F., Esteve, J., Gorin, N. C., Giebel, S., Ciceri, F., & Nagler, A. (2017). Haploidentical hematopoietic cell transplantation for adult acute myeloid leukemia: A position statement from the Acute Leukemia Working Party of the European Society for Blood and Marrow Transplantation. Haematologica, 102(11), 1810–1822.

Panch, S. R., Szymanski, J., Savani, B. N., & Stroncek, D. F. (2017). Sources of hematopoietic stem and progenitor cells and methods to optimize yields for clinical cell therapy. Biology of Blood and Marrow Transplantation, 23(8), 1241–1249.

Protocol ID

850501

Variables
Export Variables
Variable Name Variable IDVariable DescriptiondbGaP Mapping
PX850501_Sickle_Cell_Disease_Stem_Cell_Source_Manipulation_Any_Additional_Drug_Product_Potency_Assays_Performed
PX850501140100 Were any additional drug product potency more
assays performed on drug product show less
N/A
PX850501_Sickle_Cell_Disease_Stem_Cell_Source_Manipulation_Any_Additional_Drug_Product_Potency_Assays_Performed_If_Yes_Describe_Report_Result
PX850501140200 Were any additional drug product potency more
assays performed on drug product: If yes, describe and report result show less
N/A
PX850501_Sickle_Cell_Disease_Stem_Cell_Source_Manipulation_Conversion
PX850501230300 Conversion N/A
PX850501_Sickle_Cell_Disease_Stem_Cell_Source_Manipulation_Date_Drug_Product_Manufacture
PX850501090000 Date of drug product manufacture N/A
PX850501_Sickle_Cell_Disease_Stem_Cell_Source_Manipulation_Degree_Mismatch_Related_Donors_Only
PX850501060000 Degree of mismatch for related donors N/A
PX850501_Sickle_Cell_Disease_Stem_Cell_Source_Manipulation_Donor_Template_Used
PX850501220100 Donor Template Used? N/A
PX850501_Sickle_Cell_Disease_Stem_Cell_Source_Manipulation_Donor_Template_Used_Yes
PX850501220200 Donor Template Used? If yes, mode of delivery N/A
PX850501_Sickle_Cell_Disease_Stem_Cell_Source_Manipulation_Drug_Product_Starting_Material
PX850501080000 Drug product starting material N/A
PX850501_Sickle_Cell_Disease_Stem_Cell_Source_Manipulation_Drug_Product_VCN
PX850501170000 Drug product VCN N/A
PX850501_Sickle_Cell_Disease_Stem_Cell_Source_Manipulation_Drug_Product_VCN_Cell
PX850501190000 Drug product VCN/cell N/A
PX850501_Sickle_Cell_Disease_Stem_Cell_Source_Manipulation_Expression_Globin_Chain_Interest_Qualitative_Assay
PX850501120000 Expression of globin chain of interest: more
Qualitative Assay show less
N/A
PX850501_Sickle_Cell_Disease_Stem_Cell_Source_Manipulation_Expression_Globin_Chain_Interest_Quantitative_Assay
PX850501130000 Expression of globin chain of interest: more
Quantitative Assay show less
N/A
PX850501_Sickle_Cell_Disease_Stem_Cell_Source_Manipulation_Genetically_Modified
PX850501030000 Is the product genetically modified? N/A
PX850501_Sickle_Cell_Disease_Stem_Cell_Source_Manipulation_Genetically_Modified_Allogenic
PX850501040000 Is the product genetically modified? more
Allogenic: Specify related donor type show less
N/A
PX850501_Sickle_Cell_Disease_Stem_Cell_Source_Manipulation_Mode_Nuclease_Delivery
PX850501210100 Mode of nuclease delivery N/A
PX850501_Sickle_Cell_Disease_Stem_Cell_Source_Manipulation_Mode_Nuclease_Delivery_GRNA_Target_Gene
PX850501210200 Mode of nuclease delivery: gRNA target gene N/A
PX850501_Sickle_Cell_Disease_Stem_Cell_Source_Manipulation_Mode_Nuclease_Delivery_Mode_Delivery
PX850501210300 Mode of nuclease delivery: Mode of delivery N/A
PX850501_Sickle_Cell_Disease_Stem_Cell_Source_Manipulation_Off_Target_Indels
PX850501230200 Off-target Indels N/A
PX850501_Sickle_Cell_Disease_Stem_Cell_Source_Manipulation_On_Target_Indels
PX850501230100 On-target Indels N/A
PX850501_Sickle_Cell_Disease_Stem_Cell_Source_Manipulation_Percentage_Cells_Positive_Integrating_Factor
PX850501180000 Percentage of cells positive for integrating more
factor show less
N/A
PX850501_Sickle_Cell_Disease_Stem_Cell_Source_Manipulation_Proportion_CD34_Cells
PX850501100000 Proportion of CD34+ Cells N/A
PX850501_Sickle_Cell_Disease_Stem_Cell_Source_Manipulation_Specify_Donor
PX850501010000 Specify donor N/A
PX850501_Sickle_Cell_Disease_Stem_Cell_Source_Manipulation_Specify_Donor_Relationship_Recipient
PX850501050100 Specify donor relationship to the recipient N/A
PX850501_Sickle_Cell_Disease_Stem_Cell_Source_Manipulation_Specify_Donor_Relationship_Recipient_Other_Biological_Relative
PX850501050200 Specify donor relationship to the recipient: more
Other biological relative - Specity other biological relative: show less
N/A
PX850501_Sickle_Cell_Disease_Stem_Cell_Source_Manipulation_Specify_Product_Type
PX850501020100 Specify product type (check all that apply) N/A
PX850501_Sickle_Cell_Disease_Stem_Cell_Source_Manipulation_Specify_Product_Type_Other
PX850501020200 Specify product type (check all that apply): more
Other product - Specify other product: show less
N/A
PX850501_Sickle_Cell_Disease_Stem_Cell_Source_Manipulation_Specify_Unrelated_Donor_Type
PX850501070000 Specify unrelated donor type N/A
PX850501_Sickle_Cell_Disease_Stem_Cell_Source_Manipulation_Total_Colonies_Formed
PX850501110100 Total Colonies Formed N/A
PX850501_Sickle_Cell_Disease_Stem_Cell_Source_Manipulation_Total_Colonies_Formed_Rate
PX850501110200 Total Colonies Formed (rate) N/A
PX850501_Sickle_Cell_Disease_Stem_Cell_Source_Manipulation_Type_Editing
PX850501200100 Type of editing N/A
PX850501_Sickle_Cell_Disease_Stem_Cell_Source_Manipulation_Type_Editing_Other
PX850501200200 Type of editing: Other N/A
PX850501_Sickle_Cell_Disease_Stem_Cell_Source_Manipulation_Type_Integrating_Vector
PX850501160100 Type of integrating factor N/A
PX850501_Sickle_Cell_Disease_Stem_Cell_Source_Manipulation_Type_Integrating_Vector_Other
PX850501160200 Type of integrating factor: Other N/A
PX850501_Sickle_Cell_Disease_Stem_Cell_Source_Manipulation_Type_Modification
PX850501150000 Type of modification N/A
Curative Therapies
Measure Name

Sickle Cell Disease Stem Cell Source

Release Date

August 16, 2021

Definition

The stem cells used for a transplant may come from a donor either related or unrelated to the patient (allogeneic transplant) or from the patient’s own body (autologous transplant). Stem cells may also be collected from different sources, including directly from the bone marrow, from peripherally mobilized blood, or from umbilical cord blood. Before being used for transplantation, stem cells may be manipulated to further purify a specific cell population (in allogeneic transplants) or for use in gene therapy (including gene editing and gene addition in autologous transplants). 

Purpose

The stem cell donor type, source type, and type of manipulation (if any) influence the outcome of a stem cell transplant for a patient with sickle cell disease, including the quantity of stem cells, speed of engraftment, risk of graft rejection, number of CD34+ cells, risk of graft versus host disease, and overall success or failure of the transplant to treat sickle cell disease.

Keywords

Sickle cell disease, sickle cell curative therapies, stem cell, bone marrow transplant, gene therapy, stem cell donor type, allogenic transplant, autologous transplant, Center for International Blood and Marrow Transplant Research, CIBMTR

Measure Protocols
Protocol ID Protocol Name
850501 Sickle Cell Disease Stem Cell Source and Cell Manipulation
Publications

There are no publications listed for this protocol.