Informing treatment decision-making for sickle cell disease

Discussing sickle cell disease (SCD) therapy choices with patients and caregivers is an ongoing dialogue throughout the course of treatment. Clinical trials may be an important consideration, especially for patients with an aggressive disease course. A consultation for hematopoietic cell transplantation (HCT), the only potentially curative therapy, may be indicated. 

On this page, you will find educational activities on the latest treatment guidelines and choices, clinical trials and resources. These are available to you, your patients and caregivers to care for and support treatment decisions with pediatric and adult patients with SCD. 

On-Demand Sickle Cell Disease Education Activities

Access current clinical trials for patients with SCD

Clinical trials are key to making progress in treatments for patients with sickle cell disease. Two Blood and Marrow Transplant Clinical Trials Network (BMT CTN) protocols are currently enrolling patients.

Finding and joining clinical trials for blood disorders is made easier with the Jason Carter Clinical Trials Program tool. 

Learn more about clinical trials and search tools          

Dr. Steven Devine discusses Clinical Trials for Sickle Cell Disease

Two ongoing SCD clinical trials, plus a search tool for SCD trials

BMT CTN Protocol 1503

HCT versus standard of care in adolescents and young adults with SCD

Sickle Cell Disease Medicare Study

Study Title: Prospective Assessment of Allogeneic Hematopoietic Cell Transplantation in Patients with Sickle Cell Disease (17-CMS-SCD)

A Phase II, multi-center, CMS-approved CED clinical trial

Note: Patients with Medicare are eligible for coverage of HCT for Sickle Cell Disease (SCD) if they participate in this Coverage with Evidence Determination (CED) clinical trial

Study Objectives:

The primary objective of this observational study is to prospectively compare survival of patients 15 to 50 years of age with SCD who receive allogeneic transplantation compared to those receiving standard of care treatments.

Patient Eligibility:

  • Sickle cell disease (HB SS, HB S beta thalassemia or HB SC) with severe signs or symptoms indicating poor prognosis and warranting transplantation
  • Age 15 – 50 years
  • Donors may be HLA-matched siblings, mismatched siblings or other relatives (parent, offspring or another relative mismatched to the recipient at 1 or more HLA loci), HLA-matched or mismatched unrelated donors
  • Donors with sickle cell trait are eligible to donate

Abbreviated eligibility requirements, see BMT CTN Protocol 1503 for complete list

Sickle Cell Disease Medicare Study

Study Title: Prospective Assessment of Allogeneic Hematopoietic Cell Transplantation in Patients with Sickle Cell Disease (17-CMS-SCD)

A Phase II, multi-center, CMS-approved CED clinical trial

Note: Patients with Medicare are eligible for coverage of HCT for Sickle Cell Disease (SCD) if they participate in this Coverage with Evidence Determination (CED) clinical trial

Study Objectives:

The primary objective of this observational study is to prospectively compare survival of patients 15 to 50 years of age with SCD who receive allogeneic transplantation compared to those receiving standard of care treatments.

Patient Eligibility:

  • Sickle cell disease (HB SS, HB S beta thalassemia or HB SC) with severe signs or symptoms indicating poor prognosis and warranting transplantation
  • Age 15 – 50 years
  • Donors may be HLA-matched siblings, mismatched siblings or other relatives (parent, offspring or another relative mismatched to the recipient at 1 or more HLA loci), HLA-matched or mismatched unrelated donors
  • Donors with sickle cell trait are eligible to donate

Abbreviated eligibility requirements, see BMT CTN Protocol 1503 for complete list

BMT CTN Protocol 1507

Reduced-intensity haploidentical transplantation in patients with symptomatic SCD

Sickle Cell Haploidentical Study

Study Title: Reduced Intensity Conditioning for Haploidentical Bone Marrow Transplantation in Patients with Symptomatic Sickle Cell Disease

A Phase II, single arm, multi-center trial, designed to estimate the efficacy and toxicity of haploidentical bone marrow transplantation in patients with SCD.

Study Objectives:

The primary objective of this study is to estimate EFS at 2 years after haploidentical transplantation in patients with severe SCD.

Patient Eligibility:

  • Patients with severe SCD in two cohorts: children age 5 – 15 years and adults age >15 – 45 years
  • Karnofsky or Lansky performance score ≥60
  • Some eligibility criteria differ by age

Abbreviated eligibility requirements, see BMT CTN Protocol 1507 for complete list

Sickle Cell Haploidentical Study

Study Title: Reduced Intensity Conditioning for Haploidentical Bone Marrow Transplantation in Patients with Symptomatic Sickle Cell Disease

A Phase II, single arm, multi-center trial, designed to estimate the efficacy and toxicity of haploidentical bone marrow transplantation in patients with SCD.

Study Objectives:

The primary objective of this study is to estimate EFS at 2 years after haploidentical transplantation in patients with severe SCD.

Patient Eligibility:

  • Patients with severe SCD in two cohorts: children age 5 – 15 years and adults age >15 – 45 years
  • Karnofsky or Lansky performance score ≥60
  • Some eligibility criteria differ by age

Abbreviated eligibility requirements, see BMT CTN Protocol 1507 for complete list

The Jason Carter Clinical Trials Program

Learn about and access clinical trials for blood cancers and disorders

The Jason Carter Clinical Trials Program was created to make finding and joining clinical trials easier for patients and their families. The tool provides easy-to-read descriptions of blood cancer and blood disorder trials from clinicaltrials.gov. The program also offers a Clinical Trial Patient Education Specialist who can help answer questions and prepare patients for informed conversations with their care teams.
Additional features of the program include:

  • Financial assistance for travel expenses through the Drs. Jeffrey and Isabel Chell Clinical Trial Travel Grant
  • Creating an account to save searches and be notified when new trials are added that meet your search criteria

Explore our search tool to find suitable trials and information to share with your patients.

 

    The Jason Carter Clinical Trials Program was created to make finding and joining clinical trials easier for patients and their families. The tool provides easy-to-read descriptions of blood cancer and blood disorder trials from clinicaltrials.gov. The program also offers a Clinical Trial Patient Education Specialist who can help answer questions and prepare patients for informed conversations with their care teams.
    Additional features of the program include:

    • Financial assistance for travel expenses through the Drs. Jeffrey and Isabel Chell Clinical Trial Travel Grant
    • Creating an account to save searches and be notified when new trials are added that meet your search criteria

    Explore our search tool to find suitable trials and information to share with your patients.

     


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