Giulia Prunotto, Italy

University Milano Bicocca Paediatrics

Presenter of 1 Presentation

Meet the Expert Malignancy and PID

ALLOGENEIC STEM CELL TRANSPLANTATION OFFERS AN EFFECTIVE TREATMENT STRATEGY FOR LYMPHOPROLIFERATIVE DISORDERS IN PRIMARY IMMUNODEFICIENCIES

Lecture Time
08:15 - 08:25
Room
Copper
Date
19.09.2019, Thursday
Session Time
07:45 - 08:45
Presentation Topic
Malignancy and PID

Abstract

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.

Methods

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.

Results

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).

Conclusions

The study describes the largest cohort of patients with LPD and PID treated with HSCT so far, and supports HSCT as curative strategy.

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