Mycophenolate Mofetil Use in Pediatric Immune Thrombocytopenia Refractory to First-line Therapy: a Single-center Experience

Goldberg, Tracie; Levy, Carolyn Fein

doi:10.1097/mph.0000000000002688

Cited by 2 publications

(1 citation statement)

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“…Over the last decade, efficacy and safety of SR and mycophenolate therapy in patients with ALPS, ALPS-like, and other AICs have been described in several reports ( 1 , 2 , 5 – 7 , 9 , 12 , 15 – 17 , 21 – 33 ). However, there are no studies reporting the actual risks of infections in patients undergoing these treatments over the years.…”

Section: Discussionmentioning

confidence: 99%

Infection risk in patients with autoimmune cytopenias and immune dysregulation treated with mycophenolate mofetil and sirolimus

Comella,

Palmisani,

Mariani

et al. 2024

Front. Immunol.

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IntroductionAutoimmune cytopenias (AICs) are a group of disorders characterized by immune-mediated destruction of blood cells. In children, they are often secondary to immune dysregulation that may require long-lasting immunosuppression. Mycophenolate mofetil and sirolimus represent two well-tolerated options to treat these disorders, often as a steroid-sparing option. However, no data are available on the infection risk for patients undergoing long-lasting treatments.Patients and methodsThe rate of severe infective events was calculated in episodes per 100 persons/months at risk (p/m/r) documented by the analysis of hospitalization charts between January 2015 and July 2023 of patients treated with mycophenolate mofetil or sirolimus given for isolated AIC or AICs associated with autoimmune lymphoproliferative syndrome (ALPS)/ALPS-like syndromes in two large Italian pediatric hematology units.ResultsFrom January 2015 to July 2023, 13 out of 96 patients treated with mycophenolate mofetil or sirolimus developed 16 severe infectious events requiring hospitalization. No patients died. Overall infection rate was 0.24 person/*100 months/risk (95% CI 0.09–0.3). Serious infectious events incidence was higher in patients with ALPS-like compared to others (0.42 versus 0.09; p = 0.006) and lower in patients who underwent mycophenolate treatment alone compared to those who started sirolimus after mycophenolate failure (0.04 versus 0.29, p = 0.03). Considering only patients who started treatment at the beginning of study period, overall cumulative hazard was 18.6% at 60 months (95% CI 3.4–31.4) with higher risk of infectious events after 5 years in ALPS-like patients (26.1%; 95% CI 3.2–43.5) compared to other AICs (4%; 95% CI 0–11.4; p = 0.041).DiscussionTo the best of our knowledge, this is the first study to describe the infectious risk related to mycophenolate and sirolimus chronic treatment in patients with AICs and immune dysregulation. Our data highlight that infection rate is very low and mainly related to the underlying hematological condition.ConclusionsMycophenolate and sirolimus represent a safe immunosuppressive therapy in AICs and immune dysregulation syndromes.

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