ASH Releases New Clinical Practice Guidelines on Stem Cell Transplantation in Sickle Cell Disease

Sep 28, 2021

Transplantation guideline completes collection of evidence-based guidelines for sickle cell disease

(WASHINGTON, Sep. 28, 2021) – Today, the American Society of Hematology (ASH), the world’s largest professional society dedicated to furthering the understanding, diagnosis, treatment, and prevention of blood disorders, announced the publication of the American Society of Hematology 2021 Guidelines for Sickle Cell Disease: Stem Cell Transplantation. The guidelines, which are the final installment of the Society’s five evidence-based clinical practice guidelines on sickle cell disease (SCD), round out the full collection of recommendations for improving care for individuals living with SCD.

SCD is the most common inherited red blood cell disorder in the United States, affecting an estimated 100,000 people. According to the Centers for Disease Control and Prevention, SCD affects one out of every 365 Black or African American births and one out of every 16,300 Hispanic American births. The condition can cause severe pain, joint and organ damage, and stroke.

Allogeneic hematopoietic stem cell transplant (HSCT), a treatment in which the individual’s blood-forming stem cells are replaced with healthy cells from a donor, is currently the only potentially curative therapy for SCD. The guidelines, published in ASH’s journal Blood Advances, inform how to use HSCT in clinical practice, including through weighing of risks and benefits of transplantation rather than disease-modifying therapies or potential curative therapies still under development, such as gene therapy.

Care of individuals living with SCD is often the responsibility of primary care, family, and emergency medicine physicians and when surveyed, 69% of family physicians reported that clinical decision support tools would be useful for treating SCD. Together, the guidelines in the SCD collection provide evidence-based recommendations for emergency medicine physicians, primary care and family physicians, hematologists, and patients about how to manage SCD and how to individualize care to help individuals with SCD and their providers make shared and informed decisions. The collection of SCD guidelines covers care topics for which there has been significant uncertainty or variation in clinical practice:

“We have known for decades that transplantation is indeed curative for sickle cell disease, but we have never before had a set of comprehensive recommendations about how the therapy fits into the treatment options for this disease,” said John Tisdale, MD, guideline co-chair and Chief of the Cellular and Molecular Therapeutics Branch at the National Heart, Lung, and Blood Institute (NHLBI). “It is important that discussions about transplantation begin at a young age for all individuals with sickle cell disease as one possible treatment option available over their lifetime. These guidelines inform the continued discussions and risk-benefit analyses between patients and providers.”

In partnership with the Evidence-Based Practice Research Program at Mayo Clinic, the ASH Guidelines on SCD were developed using the GRADE methodology to ensure the highest standards for trustworthiness. ASH brought together 61 clinical experts, five methodologists, and 10 patient representatives to identify best practices for the management of acute and chronic complications of SCD and ways to improve the quality of care for patients.

“The culmination of the full set of clinical practice guidelines for sickle cell disease is an important achievement for ASH, since supporting research and improving care for all people living with the disease is one of the Society’s key priorities,” said ASH President Martin S. Tallman, MD, of Memorial Sloan Kettering Cancer Center. “It is of even greater importance to the SCD community. As treatment for individuals living with SCD has continued to evolve and progress, a comprehensive, evidence-based set of treatment guidelines is invaluable to patients, providers, and caregivers.”

Highlights of the ASH Guideline Recommendations for SCD: Stem Cell Transplantation:

  • HSCT should be considered over standard of care (transfusion) in individuals with SCD who have experienced a stroke or are at very high risk of stroke. Further, transplantation should be considered for all patients with neurologic injury who have a matched, related sibling donor. Recommendations point to evidence suggesting that children under age 13 who receive HSCT from a matched sibling donor have better outcomes than those older than age 13.
  • For patients with frequent pain, as well as those with recurrent episodes of acute chest syndrome, the ASH guidelines suggest transplantation from a matched sibling donor over the standard of care.
  • For individuals with an indication for HSCT who lack a matched sibling donor, the ASH guideline panel suggests transplantation from alternate donors only in the context of a clinical trial.
  • In patients with an indication for transplant, the ASH guideline panel suggests transplantation with cells from a matched donor earlier in life due to the risk of irreversible SCD-related damage to the body that increases with age.

The ASH Guidelines on SCD are part of a larger guideline development initiative for ASH. ASH is committed to the timely update of existing guidelines and the development of new guidelines for a range of hematologic conditions. ASH also has resources for treating SCD in the COVID-19 pandemic, including frequently asked questions about SCD and COVID.

Visit www.hematology.org/SCDguidelines for more information about the ASH Guidelines on SCD, including fact sheets with key takeaways from each guideline, infographics, clinical teaching slide sets, and patient stories.

About ASH’s Work in Sickle Cell Disease

In 2016, ASH launched a multifaceted initiative to address the burden of disease both in the United States and globally. ASH has since developed clinical guidelines for SCD management and care, expanded education and training efforts, and advocated with policymakers to enhance and expand federal SCD programs that include efforts to address pain management in individuals with SCD (learn more about ASH’s advocacy efforts here). Currently, ASH is working with members of Congress to create a pilot program for better access to health care for people living with SCD. In 2016, the Society also founded the Sickle Cell Disease Coalition which has grown to more than 85 members. In addition to these efforts, the ASH Research Collaborative (ASH RC) Data Hub and SCD Clinical Trials Network was developed with the mission to improve outcomes for individuals with SCD by expediting SCD therapy development and facilitating innovation in clinical trial research. It provides the infrastructure for identifying patient cohorts for trials, matching trial sponsors with sites, facilitating recruitment of eligible patients, and ensuring optimally designed trials and an efficient, coordinated approach. Through patient engagement and optimized clinical trial execution, the Clinical Trials Network will help to bring new and more effective therapies to individuals with SCD.

American Society of Hematology

The American Society of Hematology (ASH) (www.hematology.org) is the world’s largest professional society of hematologists dedicated to furthering the understanding, diagnosis, treatment, and prevention of disorders affecting the blood. For more than 60 years, the Society has led the development of hematology as a discipline by promoting research, patient care, education, training, and advocacy in hematology. ASH publishes Blood (www.bloodjournal.org), the most cited peer-reviewed publication in the field, and Blood Advances (www.bloodadvances.org), an online, peer-reviewed open-access journal.

Contact:
 Leah Enser, American Society of Hematology
lenser@hematology.org; 202-552-4927