Lupus & antiphospholipid syndrome

CD19 Chimeric Antigen Receptor (CAR) T-Cell Therapy in Autoimmune Disease — A Case Series with Follow-up

N Engl J MedPublished Case series

Authors' conclusion

In this case series, CD19 CAR T-cell transfer appeared to be feasible, safe, and efficacious in three different autoimmune diseases

Study context

Population
Patients with severe systemic lupus erythematosus (SLE), idiopathic inflammatory myositis or systemic sclerosis receiving a single infusion of CD19 CAR T cells after fludarabine and cyclophosphamide preconditioning
Participants
15 patients
Exposure
SLE vs Idiopathic inflammatory myositis vs Systemic sclerosis
  • SLEn = 8
  • Idiopathic inflammatory myositisn = 3
  • Systemic sclerosisn = 4
Outcome
Efficacy up to 2 years after CAR T-cell infusion
Follow-up
Median 15 months (range, 4 to 29)
Methodological detailssingle infusion, follow-up up to 2 years
  • Single infusion
  • Follow-up up to 2 years

Main finding

As reported in the abstract

  • SLE: all patients had Definition of Remission in SLE (DORIS) remission
  • Idiopathic inflammatory myositis: all patients had an American College of Rheumatology-European League against Rheumatism (ACR-EULAR) major clinical response
  • Systemic sclerosis: all patients had a decrease in EUSTAR activity index score
  • Immunosuppressive therapy completely stopped in all patients

Observational data: associations, not effects.

Further findings

Mean (±SD) duration of B-cell aplasia, days
112±47

Safety

Grade 1 cytokine release syndrome

Patients with grade 1 cytokine release syndrome
10

Grade 2 cytokine release syndrome; grade 1 immune effector cell-associated neurotoxicity syndrome; pneumonia resulting in hospitalisation

One patient each
1

Original publication

CD19 CAR T-Cell Therapy in Autoimmune Disease — A Case Series with Follow-up. N Engl J Med. Published 22 February 2024. doi:10.1056/nejmoa2308917

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