Bone Marrow & Stem Cell Transplant

The FDA granted Regenerative Medicine Advanced Therapy designation to NXC-201 for the treatment of relapsed/refractory light chain amyloidosis.

Orca-T improved overall survival vs post-transplant cyclophosphamide in a retrospective analysis.

Orca-T with reduced-intensity stem cell transplantation was a safe, feasible alternative to conventional transplant in blood cancers, per phase 1 data.

A phase 2 study evaluated the use of ruxolitinib plus standard GVHD prophylaxis in patients with myelofibrosis undergoing transplant.

In a retrospective cohort of patients with severe chronic GVHD, combination axatilimab with other GVHD therapies led to clinical benefit.

Defibrotide prophylaxis after HSCT did not reduce sinusoidal obstructive syndrome incidence in high-risk pediatric patients, per retrospective study at 2025 Tandem Meeting.

Unrelated cord blood transplant demonstrated efficacy in patients with blood cancer after microcystic adnexal carcinoma.

The FDA has issued a complete response letter to the biologics license application seeking the approval of tabelecleucel for Epstein-Barr virus–positive post-transplant lymphoproliferative disease treatment.

David Sallman, MD, Moffitt Cancer Center, discussed the trial's impact and the future role of MRD monitoring in treatment strategies for TP53-mutant MDS and AML.

Amandeep Salhotra, MD, discussed the background and findings from a pilot trial evaluating enasidenib as post-hematopoietic stem cell transplant maintenance therapy for IDH2-mutated acute myeloid leukemia treatment.

Following a complete response letter and biologics license application resubmission, the FDA has approved remestemcel-L for the treatment of pediatric patients with steroid-refractory acute graft-vs-host disease.

In this feature, Memorial Sloan Kettering Cancer Center physicians explain what drives the collaborative process in their multidisciplinary graft-vs-host disease clinic.

In an interview, Colton Ladbury, MD, discussed new insights from a study and expanded on total marrow irradiation’s potential to transform transplant conditioning.

In an interview with Targeted Oncology, Josep Maria Ribera, MD, PhD, discussed the evolving treatment protocols for Philadelphia chromosome-positive acute lymphoblastic leukemia.

During a Case-Based Roundtable® event,  Michael R. Bishop, MD, moderated a discussion on monitoring and treatment of chronic graft-vs-host disease.

Findings from the phase 3 SIERRA study showed that Iomab-B was associated with a higher durable complete response rate vs allogeneic cell transplant in older patients with relapsed/refractory acute myeloid leukemia.

Efficacy findings from the phase 2 AGAVE-201 support the use of axatilimab, which was approved by the FDA for recurrent or refractory chronic graft-vs-host disease in August 2024.

In an interview with Targeted Oncology, Sergio A. Giralt, MD, discussed the evolving role of transplant in myelodysplastic syndromes.

Axatilimab is now an FDA-approved treatment option for patients with chronic graft-vs-host disease.

A phase 2 study found that a combination of pacritinib, sirolimus, and tacrolimus successfully suppressed pSTAT3 and Th1/Th17 cells, but failed to prevent acute graft-versus-host disease following allogeneic transplant.

Mayuko Sakae, MD, discusses findings from research presented at the 2024 Multinational Association of Supportive Care in Cancer Annual Meeting on refractory pain in patients who have undergone a bone marrow transplant.

Mayuko Sakae, MD, discussed research on pain management for patients undergoing bone marrow transplants, highlighting the potential buprenorphine shows in offering a valuable alternative to traditional opioids.

Patients who underwent hematopoietic cell transplantation had similar outcomes with matched vs mismatched unrelated donors when posttransplant cyclophosphamide was used.

Alexandra Gomez Arteaga, MD, discusses the rationale behind a retrospective study comparing Orca-T with posttransplant cyclophosphamide-based hematopoietic cell transplantation.

The FDA has accepted and granted priority review to tab-cel, a treatment for Epstein Barr virus-positive posttransplant lymphoproliferative disease.