Pediatric Immune Thrombocytopenia in Lebanon: Treatment and Predictors of Outcome

Abstract

To the Editor: A retrospective analysis was conducted on 105 pediatric patients (60% males; median age at presentation 5 years) with immune thrombocytopenia (ITP) attending Rafic Hariri University and Nini Hospitals, Lebanon, between 1985 and 2012. The presenting symptom in 89% of cases was mucocutaneous bleeding; none had gastrointestinal or intracranial bleeding; 22.9% were taking medications, 24.8% had preceding infections, and 2.85% recent vaccination. Mean platelet count at presentation was 16 Â 10 9 /L. Bone marrow aspirates (BMAs) at diagnosis (60%) and after treatment failure (20%) confirmed ITP diagnosis.Main treatment modalities were steroids (61.9%), intravenous immunoglobulin (IVIG) (43.8%), and close observation (29.5%) (Supplemental Figure 1). Median time to splenectomy was 6.75 months after presentation. Patients who received platelet transfusions had platelet counts <10 Â 10 9 /L and wet purpura; none had life-threatening bleeding. Newly diagnosed, persisting, chronic, relapsing, and refractory forms were present in 40%, 16.2%, 27.7%, 1.9%, and 0.9% of patients, respectively; 13.3% were lost to followup. Urgent hospitalization was necessary in 27.6%. Life-threatening complications occurred in two patients: rituximab associated anaphylactic shock and gastrointestinal bleeding-associated hypotension. One patient developed mild systemic lupus erythematosis.Significant correlations are summarized in Table I. Positive correlations included chronicity with older age at presentation or treatment with steroids, rituximab, or splenectomy; urgent hospitalization with severe ITP and platelet transfusion; BMA at diagnosis with steroid use and platelet transfusion; BMA after treatment failure correlated with chronicity, IVIG use, and splenectomy; platelet transfusion with persistent, relapsing, and severe ITP; IVIG treatment with severe ITP, urgent hospitalization, and chronicity; both life-threatening and non-life-threatening complications with chronic ITP, treatment with rituximab, and splenectomy. We observed negative correlations of both urgent hospitalization and IVIG treatment with increased age.Time to platelet recovery and life-threatening complications were not significantly decreased by corticosteroids (P ¼ 0.1, P ¼ 0.2) or IVIG (P ¼ 0.7, P ¼ 0.

Defining curative endpoints for transfusion‐dependent β‐thalassemia in the era of gene therapy and gene editing

“…The pace and ease of pre‐existing iron removal are also affected by the timing of initiation of iron removal therapy after transplant, whether the option of phlebotomy is practical, and patient adherence to iron reduction treatment in the post‐transplant setting. It is expected that removal of pre‐existing iron will occur slowly over time after gene therapy and gene editing approaches, potentially taking years to normalize, consistent with observations from the allogeneic HSCT experience 61,62 . As such, iron overload and the use of iron chelation therapy should also be monitored in long‐term follow‐up studies to best inform achievement of a functional cure.…”

“…It is expected that removal of pre-existing iron will occur slowly over time after gene therapy and gene editing approaches, potentially taking years to normalize, consistent with observations from the allogeneic HSCT experience. 61,62 As such, iron overload and the use of iron chelation therapy should also be monitored in long-term follow-up studies to best inform achievement of a functional cure.…”