Safety
immunodeficiency disorder, infections, [Immunodeficiency disorder
IMMUNODEFICIENCY DISORDER, PRIMARY IMMUNODEFICIENCY, INFECTIONS, ADVERSE EFFECTS, ADVERSE EVENT, ULCERATION, IMMUNODEFICIENCY DISORDER
The frequency of adverse effects was in line with EXIST-1–3 [Notably, patients with adverse effects ≥ grade 3 were younger than patients without adverse effects ≥ grade 3, this is in line with results in the extension study of EXIST-3 [The frequency of stomatitis/oral ulceration was slightly lower than those reported in EXIST-1–3 [The upper respiratory tract infections in our study were quite similar to those in EXIST-1–3 [Dose reductions and interruptions were reported in 50%, as reported in EXIST-2 [Immunodeficiency disorder was described in one patient. This is a serious condition and is as far as we know not previously described as an everolimus related adverse event. Immunoglobulins were not measured before initiation of everolimus in this patient, and consequently it was not possible to determine for certain whether the patient that developed immunodeficiency disorder has a primary immunodeficiency, or if this is drug related. Clinically infections increased significantly after treatment initiation, making everolimus treatment a probable cause.Data collection was finished for most patients before the outbreak of covid-19, only 14 had follow-up for days up to a month after the outbreak. For two of the nine patients that discontinued everolimus treatment, the COVID-19 pandemic was part of the reason, but apart from that it is not likely that the study result was influenced by the pandemic.