Protocol - Sickle Cell Disease Transplant Conditioning Regimen

Personnel and Training Required

Personnel who are trained in performing medical record review

Equipment Needs

None

Requirements

Requirement Category	Required
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 Cure Sickle Cell Initiative (CureSCi) Transplant Conditioning Regimen Case Report Form was designed for use in sickle cell disease. It was also chosen because it describes specific conditioning agents, dosages, and timing rather than using general terminology for conditioning regimens, such as “myeloablative” or “non-myeloablative.”

Language

English

Standards

Standard	Name	ID	Source

Derived Variables

None

Process and Review

Not applicable.

Protocol Name from Source

Cure Sickle Cell Initiative (CureSCi) Transplant Conditioning Case Report Form (CRF)

Source

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

General References

Gluckman, E., Cappelli, B., Bernaudin, F., Labopin, M., Volt, F., Carreras, J., ... & Eapen, M. (2017). Sickle cell disease: An international survey of results of HLA-identical sibling hematopoietic stem cell transplantation. Blood, 129(11), 1548–1556.

Protocol ID

850401

Variables

Export Variables

Variable Name	Variable ID	Variable Description	dbGaP Mapping
PX850401_Sickle_Cell_Disease_Transplant_Conditioning_Regimen_Average_AUC_1
	PX850401050200	Average AUC: AUC	N/A
PX850401_Sickle_Cell_Disease_Transplant_Conditioning_Regimen_Average_AUC_2
	PX850401060200	Average AUC: AUC	N/A
PX850401_Sickle_Cell_Disease_Transplant_Conditioning_Regimen_Average_AUC_Agent_1
	PX850401050100	Average AUC: Agent	N/A
PX850401_Sickle_Cell_Disease_Transplant_Conditioning_Regimen_Average_AUC_Agent_2
	PX850401060100	Average AUC: Agent	N/A
PX850401_Sickle_Cell_Disease_Transplant_Conditioning_Regimen_Chemotherapy_Agent_Used_1
	PX850401010100	Specify agent/s used	N/A
PX850401_Sickle_Cell_Disease_Transplant_Conditioning_Regimen_Chemotherapy_Agent_Used_2
	PX850401020100	Specify agent/s used	N/A
PX850401_Sickle_Cell_Disease_Transplant_Conditioning_Regimen_Chemotherapy_Agent_Used_3
	PX850401030100	Specify agent/s used	N/A
PX850401_Sickle_Cell_Disease_Transplant_Conditioning_Regimen_Chemotherapy_Agent_Used_Day_1
	PX850401010300	Specify agent/s used: on day	N/A
PX850401_Sickle_Cell_Disease_Transplant_Conditioning_Regimen_Chemotherapy_Agent_Used_Day_2
	PX850401020300	Specify agent/s used: on day	N/A
PX850401_Sickle_Cell_Disease_Transplant_Conditioning_Regimen_Chemotherapy_Agent_Used_Day_3
	PX850401030300	Specify agent/s used: on day	N/A
PX850401_Sickle_Cell_Disease_Transplant_Conditioning_Regimen_Chemotherapy_Agent_Used_Total_Dose_1
	PX850401010200	Specify agent/s used: total dose	N/A
PX850401_Sickle_Cell_Disease_Transplant_Conditioning_Regimen_Chemotherapy_Agent_Used_Total_Dose_2
	PX850401020200	Specify agent/s used: total dose	N/A
PX850401_Sickle_Cell_Disease_Transplant_Conditioning_Regimen_Chemotherapy_Agent_Used_Total_Dose_3
	PX850401030200	Specify agent/s used: total dose	N/A
PX850401_Sickle_Cell_Disease_Transplant_Conditioning_Regimen_Non_Chemotherapy_Conditioning_Agent
	PX850401080100	Non-chemotherapy conditioning: Agent	N/A
PX850401_Sickle_Cell_Disease_Transplant_Conditioning_Regimen_Non_Chemotherapy_Conditioning_Day
	PX850401080500	Non-chemotherapy conditioning: Day	N/A
PX850401_Sickle_Cell_Disease_Transplant_Conditioning_Regimen_Non_Chemotherapy_Conditioning_Dose
	PX850401080200	Non-chemotherapy conditioning: Dose	N/A
PX850401_Sickle_Cell_Disease_Transplant_Conditioning_Regimen_Non_Chemotherapy_Conditioning_Frequency
	PX850401080300	Non-chemotherapy conditioning: Frequency	N/A
PX850401_Sickle_Cell_Disease_Transplant_Conditioning_Regimen_Non_Chemotherapy_Conditioning_Total_Dose
	PX850401080400	Non-chemotherapy conditioning: Total Dose	N/A
PX850401_Sickle_Cell_Disease_Transplant_Conditioning_Regimen_Radiation_Days
	PX850401070200	Radiation: Days of Radiation	N/A
PX850401_Sickle_Cell_Disease_Transplant_Conditioning_Regimen_Radiation_Source
	PX850401070100	Radiation: Source/Type	N/A
PX850401_Sickle_Cell_Disease_Transplant_Conditioning_Regimen_Radiation_Total_Dose
	PX850401070300	Radiation: Total Dose	N/A

Curative Therapies

Measure Name

Sickle Cell Disease Transplant Conditioning Regimen

Release Date

August 16, 2021

Definition

This measure describes the combination of treatments, or conditioning regimen, used to prepare a patient with sickle cell disease for stem cell transplantation, including chemotherapy, monoclonal antibody therapy, other serotherapy, and whole-body radiation therapy.

Purpose

The purpose of a conditioning regimen is to make room in the patient’s bone marrow for new (or donor) blood stem cells to grow, prevent the patient’s body from rejecting the transplanted cells, and increase the chances of a successful transplant. A conditioning regimen used for stem cell transplant plays a significant role in the outcome of the transplant, including potential for success; risk of graft versus host disease; potential for graft failure; and complications including infection, infertility, changes in organ function, and secondary complications such as malignancy.

Keywords

sickle cell disease, Sickle Cell Disease Curative Therapies, stem cell transplant, bone marrow transplant, conditioning, radiation, Chemotherapy, monoclonal antibody, myeloablative

Measure Protocols

Protocol ID	Protocol Name
850401	Sickle Cell Disease Transplant Conditioning Regimen

Publications

There are no publications listed for this protocol.