Time to CR, to relapse, and on sirolimus, in ALPS and non-ALPS patients. (A) Overall time to achieve a CR on sirolimus in patients with ALPS compared with non-ALPS patients separated by multilineage vs single-lineage autoimmune cytopenias. For patients whose best response was PR or NR, the curve represents time on sirolimus plus 1 year of observation after discontinuation. The time to CR was not significantly different in ALPS (median, 4 weeks; range, 2 to 60) compared with non-ALPS patients with multilineage cytopenias (median, 9 weeks; range, 2 to 260) (P = .135). In contrast, the time to CR was significantly different in ALPS compared with non-ALPS patients with single-lineage cytopenias (P = .0065). Asterisk (*) represents 1 patient with non-ALPS single-lineage disease who achieved a PR and remained on drug for 260 weeks. (B) Relapse incidence in ALPS and non-ALPS patients with multilineage and single-lineage cytopenias, for all patients enrolled. Events include patients who achieved a CR and then relapsed, and patients who discontinued sirolimus for lack of efficacy. Most of the ALPS patients remained disease-free while on sirolimus, including 1 patient who stopped sirolimus after 3 years but maintained a durable CR. The median time to an event in non-ALPS patients was 13 months. Of note, 2 non-ALPS patients with multilineage cytopenias electively stopped sirolimus, had a transient drop in counts, and then were re-challenged with sirolimus, reverting back to a CR. These patients were not counted as having relapsed. (C) Histogram demonstrating the total time on sirolimus separated by ALPS, non-ALPS multilineage vs non-ALPS–single lineage. The different colors reflect whether they achieved a CR (black), PR (gray), or NR (white).