Azathioprine or mycophenolate mofetil for pediatric autoimmune cytopenia: a propensity score–matched study

Data on the immunosuppressants azathioprine (AZA) and mycophenolate mofetil (MMF) in autoimmune cytopenia (AIC) are limited, and no direct comparison exists. We analyzed the failure-free survival (FFS; time from AZA/MMF initiation to another non-first-line treatment, or death) of both treatments in the prospective nationwide pediatric OBS'CEREVANCE cohort. We included 343 patients (chronic immune thrombocytopenia, n = 161; autoimmune hemolytic anemia, n = 74; Evans syndrome, n = 108). They received AZA (n = 276) or MMF (n = 104; 37 sequentially received both) as monotherapy for a median duration of 11.3 (range, 0.01-149.0) months. Older age was associated with higher FFS for AZA, whereas secondary AIC was associated with higher FFS for MMF. AIC type had no effect. In a propensity score (PS)-matched cohort, AZA and MMF showed similar FFS (adjusted hazard ratio, 0.91; 95% confidence interval, 0.54-1.52; P = .71), with 1-year FFS rates of 73% and 76%, respectively. In subgroup analyses, AZA was associated with higher FFS in PS-matched patients diagnosed at an age of ≥10 years, whereas MMF was associated with higher FFS in PS-matched patients diagnosed at an age of <10 years and in those with confirmed secondary AIC (although with suboptimal matching). Rates of grade ≥3 infection were similar between the 2 drugs, at ∼2% new cases per year. In summary, AZA and MMF demonstrated comparable overall FFS and infection risk. However, our data suggest that AZA may be more beneficial in children diagnosed at an age of ≥10 years, whereas MMF may be more beneficial in those diagnosed at an age of <10 years and possibly in patients with secondary AIC.

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Publication Details

Journal
Blood Advances
Published
2025-10-15
DOI
https://doi.org/10.1182/bloodadvances.2025017536
Citations
1
Primary Topic
Immunodeficiency and Autoimmune Disorders
Type
article
Field-Weighted Citation Impact
0.80

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article

Azathioprine or mycophenolate mofetil for pediatric autoimmune cytopenia: a propensity score–matched study

Isabelle Pellier, Catherine Paillard, Pascal Chastagner, Caroline Thomas et al.
1 citations
Blood Advances
Immunodeficiency and Autoimmune Disorders
0.80
article

Azathioprine or mycophenolate mofetil for pediatric autoimmune cytopenia: a propensity score–matched study

Isabelle Pellier, Catherine Paillard, Pascal Chastagner, Caroline Thomas, Corinne Guitton, Sandrine Thouvenin Doulet, M Deparis, Hélder Miguel Fernandes, Nathalie Cheikh, Claire Pluchart, Nathalie Garnier, Nathalie Aladjidi, Marlène Pasquet, Anne Pagnier, Julien Lejeune, Wadih Abou Chahla, Vincent Barlogis, Thomas Pincez, Claire Desplantes, Joy Benadiba, Martin Castelle, Christophe Piguet, Sébastien Heritier, Éric Jeziorski, Philippe Vic, Frédéric Millot, Thierry Leblanc, Aude Marie‐Cardine, G Leverger, Mony Fahd, Sophie Bayart, Valérie Li Thiao Te
article en
1 citations

Abstract

Data on the immunosuppressants azathioprine (AZA) and mycophenolate mofetil (MMF) in autoimmune cytopenia (AIC) are limited, and no direct comparison exists. We analyzed the failure-free survival (FFS; time from AZA/MMF initiation to another non-first-line treatment, or death) of both treatments in the prospective nationwide pediatric OBS'CEREVANCE cohort. We included 343 patients (chronic immune thrombocytopenia, n = 161; autoimmune hemolytic anemia, n = 74; Evans syndrome, n = 108). They received AZA (n = 276) or MMF (n = 104; 37 sequentially received both) as monotherapy for a median duration of 11.3 (range, 0.01-149.0) months. Older age was associated with higher FFS for AZA, whereas secondary AIC was associated with higher FFS for MMF. AIC type had no effect. In a propensity score (PS)-matched cohort, AZA and MMF showed similar FFS (adjusted hazard ratio, 0.91; 95% confidence interval, 0.54-1.52; P = .71), with 1-year FFS rates of 73% and 76%, respectively. In subgroup analyses, AZA was associated with higher FFS in PS-matched patients diagnosed at an age of ≥10 years, whereas MMF was associated with higher FFS in PS-matched patients diagnosed at an age of <10 years and in those with confirmed secondary AIC (although with suboptimal matching). Rates of grade ≥3 infection were similar between the 2 drugs, at ∼2% new cases per year. In summary, AZA and MMF demonstrated comparable overall FFS and infection risk. However, our data suggest that AZA may be more beneficial in children diagnosed at an age of ≥10 years, whereas MMF may be more beneficial in those diagnosed at an age of <10 years and possibly in patients with secondary AIC.

Blood AdvancesVol. 10(3)
Université de Tours (FR), Hôpital Necker-Enfants Malades (FR), Centre National de la Recherche Scientifique (FR), Université de Bordeaux (FR), Inserm (FR), Université Paris Cité (FR), Centre Hospitalier Universitaire de Grenoble (FR), Centre Hospitalier Universitaire Sainte-Justine (CA), Centre Hospitalier Universitaire de Bordeaux (FR), Centre Hospitalier Universitaire de Lille (FR), Centre Hospitalier Universitaire de Nice (FR), Centre Hospitalier Universitaire de Nantes (FR), Université de Poitiers (FR), Sorbonne Université (FR), Hôpital Armand-Trousseau (FR), Assistance Publique Hôpitaux de Marseille (FR), Centre Hospitalier Universitaire de Besançon (FR), Institut d’Hématologie et d’Oncologie Pédiatrique (FR), Sorbonne Paris Cité (FR), Centre de Recherche en Cancérologie et Immunologie Intégrée Nantes Angers (FR), Centre Hospitalier Universitaire de Montpellier (FR), Centre Hospitalier Universitaire de Tours (FR), Centre Hospitalier Universitaire Amiens-Picardie (FR), Assistance Publique – Hôpitaux de Paris (FR), Centre Hospitalier Régional et Universitaire de Nancy (FR), Hospices Civils de Lyon (FR), Hôpital Jeanne de Flandre (FR), Hôpital d'Enfants (FR), Centre Hospitalier Universitaire de Reims (FR), Laboratoire de Thermique et Energie de Nantes (FR), Centre Hospitalier Universitaire de Poitiers (FR), Laboratoire National de Référence (MA), Hôpital Pellegrin (FR), Centre Hospitalier Universitaire d'Angers (FR), Centre Hospitalier Universitaire de Caen Normandie (FR), CHU Dijon Bourgogne (FR), Centre Hospitalier de Cornouaille (FR), Centre Hospitalier Universitaire de Limoges (FR), Hôpital Robert-Debré (FR), Centre d'Investigation Clinique de Nantes (FR), Hôpital Mère-Enfant (FR), Hôpital Purpan (FR), Maison des Sciences sociales et des Humanités de Dijon (FR), Hôpitaux Universitaires de Strasbourg (FR), Hôpital des Enfants (FR), Hôpital Arnaud de Villeneuve (FR), Centre d’études des Mondes Russe, Caucasien et Centre-Européen (FR), Institut Jean Godinot (FR), Centre Hospitalier Universitaire de Rouen (FR), Centre Hospitalier Universitaire de Rennes (FR), Centre Hospitalier Universitaire de Saint-Étienne (FR), Hôpital d'Hautepierre (FR), Hôpital de la Timone (FR), Bicêtre Hospital (FR), Université de Rouen Normandie (FR), Université Grenoble Alpes (FR), Université de Reims Champagne-Ardenne (FR), Université de Caen Normandie (FR)
Amgen, GlaxoSmithKline, Assistance publique-Hôpitaux de Paris
Openalex Percentile: Top 22%
Immunodeficiency and Autoimmune Disorders
0.80
DOIOpen Access PDFOpenAlex
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