Table 1_A stepwise institutional approach to refractory autoimmune cytopenia after allogeneic stem cell transplantation in children: a retrospective case series.xlsx

The primary endpoint was sustained remission; the secondary endpoint was optimal outcome (sustained remission without relapse and without Grade II–IV graft-versus-host disease [GVHD]).</p>Results<p>All 20 children achieved sustained remission (100%; 95% CI 83.2–100%) at a median follow-up of 22.2 months. Optimal outcomes occurred in 80% of Step 1 patients versus 62.5% in Step 2 and 28.6% in Step 3 (Cochran-Armitage trend test, P = 0.070).

GVHD incidence increased with treatment intensity: 20% after Step 1, 50% after Step 2, and 100% after Step 3 (P<0.001); all events were acute and self-limited.</p>Conclusion<p>All children achieved sustained remission with this stepwise protocol, and the most favourable outcomes occurred in the group that withdrew immunosuppressants alone. Because treatment was not randomised, this apparent advantage may reflect patient selection rather than true efficacy.

These observations are exploratory and require prospective validation.</p>