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Autologous ATG-free hematopoietic stem cell transplantation for refractory autoimmune rheumatic diseases: a Latin American cohort

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Abstract

Autologous hematopoietic stem cell transplantation (HSCT) has been recognized as treatment alternative for patients with severe, refractory autoimmune rheumatic diseases (ARDs). Usually, anti-thymocyte globulin (ATG)-containing conditioning regimens are employed; however, ATG is unavailable in some developing nations. We report our 15-year clinical experience autografting patients with ARDs with an ATG-free conditioning regimen and a brief assessment of patient-reported outcomes post-HSCT. All patients had active disease and were resistant to multiple lines of treatment. Event-free survival (EFS) was assessed using the Kaplan-Meier method. Eight patients underwent autologous HSCT. Diagnoses included juvenile idiopathic arthritis (n = 3), systemic lupus erythematosus (n = 2), systemic sclerosis (n = 2), and rheumatoid arthritis (n = 1). Median time from diagnosis to HSCT was 3 years (0.75–19). Hematological recovery was documented in all recipients, and 4 patients (50%) completed the procedure in a completely ambulatory setting. Five (62.5%) patients achieved complete response and 3 (37.5%) partial response. The median EFS was 7 months (95% CI, 4.97–9.02), and the 1-year EFS rate was 21.9% (95% CI, 18.25–25.76). Transplant-related mortality was 0%, and 1 recipient died 8 years post-HSCT due to chronic kidney disease. Six (75%) patients presented steroid dosage reduction post-HSCT, and 2 (25%) perceived improvement in functionality despite having relapsed. HSCT is a viable treatment alternative for selected patients with severe therapy-resistant ARDs, as an improvement in disease management and quality of life was documented. The need remains to elucidate the characteristics of the optimal HSCT candidate, as well as the adequate conditioning regimen when ATG is not available.

Key Points

• Despite advances in the treatment of autoimmune rheumatic diseases, some patients remain refractory. In this context, autologous hematopoietic stem cell transplantation (HSCT) rises as a viable alternative.

• Of 8 HSCT recipients with autoimmune rheumatic diseases, 5 (62.5%) patients achieved complete response and 3 (37.5%) partial response, with a 1-year event-free survival of 21.9%.

• Transplant-related mortality was 0%, with 4 (50%) patients autografted in a completely outpatient setting.

• Even when relapse presented, patients reported an improvement in functionality and quality of life; also, a better response to DMARDs and a reduction in steroid dependency post-HSCT were documented.

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Fig. 1

Data availability

The datasets generated and/or analyzed during the current study are available from the corresponding author on reasonable request.

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Acknowledgements

We thank Sergio Lozano-Rodriguez, MD, for his critical review of this manuscript.

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Authors and Affiliations

  1. Hematology Department, Internal Medicine Division, Dr. José Eleuterio González University Hospital and School of Medicine, Universidad Autónoma de Nuevo León, Monterrey, Mexico

    José Carlos Jaime-Pérez, Mariana González-Treviño, Jesús D. Meléndez-Flores, Eugenia M. Ramos-Dávila, Olga G. Cantú-Rodriguez, César H. Gutiérrez-Aguirre & David Gómez-Almaguer

  2. Rheumatology Department, Internal Medicine Division, Dr. José Eleuterio González University Hospital and School of Medicine, Universidad Autónoma de Nuevo León, Monterrey, Mexico

    Dionicio A. Galarza-Delgado

Authors
  1. José Carlos Jaime-Pérez
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  2. Mariana González-Treviño
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  8. David Gómez-Almaguer
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Correspondence to José Carlos Jaime-Pérez.

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Ethical approval

The study was approved by the local Research Ethics Committee (approval number: HE21–00020), and all patients provided an informed consent in accordance with the Declaration of Helsinki of 1964.

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Jaime-Pérez, J.C., González-Treviño, M., Meléndez-Flores, J.D. et al. Autologous ATG-free hematopoietic stem cell transplantation for refractory autoimmune rheumatic diseases: a Latin American cohort. Clin Rheumatol 41, 869–876 (2022). https://doi.org/10.1007/s10067-021-05931-0

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