MALIGNANCY IN WAS
ALLOGENEIC STEM CELL TRANSPLANTATION OFFERS AN EFFECTIVE TREATMENT STRATEGY FOR LYMPHOPROLIFERATIVE DISORDERS IN PRIMARY IMMUNODEFICIENCIES
Background and Aims
Patient affected by lymphoproliferative disorders in the context of PID have a dismal outcome. HSCT has been reported as treatment strategy in this context, but outcome data are scanty. This retrospective study aims to describe the clinical characteristics and outcomes of paediatric patients affected by PIDs and LPD between 2000 and 2016.
Patients <18 years, with a histologically proven LPD and a diagnosis of PID, who were referred for HSCT between 2000 and 2016 were eligible. LPD histological phenotype was reviewed by an haematopathologist . Radiographic imaging was blindly reviewed by two radiologists to establish LPD disease stage at presentation and pre HSCT.
39 children were identified and 32 had undergone HSCT. Median age at LPD diagnosis was 5.5 years. The most common histological diagnosis was B cell lymphoma (59%), HL (15.4%), polymorphic LPD (10.3%), plasmacytic hyperplasia (7.7%) T-cell lymphoma (5%). Patients had undergone a median of 2 lines of treatment before HSCT. Pre HSCT, 46% were in CR, 21% were in PR and 33% were NR. Median follow-up was 3.8 years. 3-year OS was 75.8%. The outcome for patients who did not receive HSCT was poor and significantly worse than children who received HSCT (18.0% v 87.3%, p<0.001). Only 2 pts relapsed post HSCT. LPD status pre HSCT was significantly associated with 3-year OS (94.3%, 76.5% and 50% respectively for patients in CR, PR and NR, p=0.030).
The study describes the largest cohort of patients with LPD and PID treated with HSCT so far, and supports HSCT as curative strategy.