AT A GLANCE

A new guideline published in Bone Marrow Transplantation offers updated recommendations for use of hematopoietic stem cell transplantation (HSCT) in patients with rheumatologic and musculoskeletal diseases (RMDs).¹

“Immune-mediated RMDs comprise a heterogeneous group of systemic conditions that affect the connective tissues of the musculoskeletal system and internal organs,” explain study authors Alexander et al. “Immune-mediated RMDs are driven by chronic autoimmune responses and typically require continuous or repeated administration of immunosuppressive or biologic disease-modifying drugs. Although generally effective, these therapies can cause both short- and long-term side effects and may fail to control the disease with risk of irreversible tissue damage.”

They add that HSCT has been successfully employed over the past 30 years for such patients, but caution with this approach is required due to significant side effects.

Now, updated recommendations for the use of HSCT in RMDs have been developed in collaboration with an international expert panel from the European Society for Blood and Marrow Transplantation (EBMT). For guideline development, available evidence regarding HSCT application released since 2004 was reviewed by a panel. Then, EBMT expert-based consensus recommendations were formulated.

“These recommendations include detailed indications, contraindications, and cautionary notes specific to each RMD, along with comprehensive protocols for diagnostic work-up. They are intended to support clinicians, scientists, patients, and caregivers in the field of RMDs,” explain the authors.

Reference

1.     Alexander T, Roldan E, Del Papa N. Autologous haematopoietic stem cell transplantation for rheumatic diseases: best practice recommendations from the EBMT Practice Harmonization and Guidelines Committee (online ahead of print August 20, 2025). Bone Marrow Transplant.