Treatment and outcomes of high-risk neuroblastoma in Southeast Asia: a single-institution experience and review of the literature

INTRODUCTION@#In Europe and North America, the majority of children with high-risk neuroblastoma survive the disease. Elsewhere, the treatment outcomes are poor.@*METHODS@#A retrospective review of children treated for high-risk neuroblastoma in a single institution in Singapore from 2007 to 2019 was carried out. Treatment consisted of intensive chemotherapy, surgery aimed at gross total resection of residual disease after chemotherapy, consolidation with high-dose therapy followed by autologous stem cell rescue, and radiotherapy to the primary and metastatic sites followed by maintenance treatment with either cis-retinoic acid or anti-disialoganglioside monoclonal antibody therapy. Survival data were examined on certain clinical and laboratory factors.@*RESULTS@#There were 57 children (32 male) treated for high-risk neuroblastoma. Their mean age was 3.9 (range 0.7-14.9) years. The median follow-up time was 5.5 (range 1.8-13.0) years for the surviving patients. There were 31 survivors, with 27 patients surviving in first remission, and the five-year overall survival and event-free survival rates were 52.5% and 47.4%, respectively. On log-rank testing, only the group of 17 patients who were exclusively treated at our centre had a survival advantage. Their five-year overall survival rate compared to patients whose initial chemotherapy was done elsewhere was 81.6% versus 41.1% (P = 0.011), and that of event-free survival was 69.7% versus 36.1% (P = 0.032). Published treatment results were obtained from four countries in Southeast Asia with five-year overall survival rates from 13.5% to 28.2%.@*CONCLUSION@#Intensified medical and surgical treatment for high-risk neuroblastoma proved to be effective, with superior survival rates compared to previous data from Southeast Asia.

Isolated thrombocytopenia in childhood: what if it is not immune thrombocytopenia?

<p><b>INTRODUCTION</b>Childhood immune thrombocytopenia (ITP) remains a diagnosis of exclusion when isolated thrombocytopenia is not part of another disease process. In practice, the diagnosis of ITP can only be confirmed when thrombocytopenia resolves or is excluded after the recognition of a primary cause.</p><p><b>METHODS</b>The records of 87 consecutive children with isolated thrombocytopenia seen over a nine-year period in a private paediatric haematology practice were reviewed retrospectively. Children in whom a primary cause was eventually found were the subjects of a further descriptive study.</p><p><b>RESULTS</b>9 (10%) children with isolated thrombocytopenia were not diagnosed with ITP because a primary disease was found. Of these nine cases, four had thrombocytopenia recognised during the neonatal period, consisting of perinatal cytomegalovirus infection (n = 2), meconium aspiration pneumonia (n = 1) and transient abnormal myelopoiesis associated with Down syndrome (n = 1). The remaining five children were each found to have familial thrombocytopenia, portal hypertension, cutaneous mastocytosis, May-Hegglin anomaly and systemic lupus erythematosus. Two of them had a history of failure of response to corticosteroid therapy.</p><p><b>CONCLUSION</b>Secondary thrombocytopenia is not uncommon in a tertiary paediatric specialty practice with adequate evaluation. Thrombocytopenia occurring during the newborn period and failure of steroid therapy are predictive of secondary cases.</p>

Haematologist-reviewed peripheral blood smear in paediatric practice

Manual examination of the peripheral blood smear (PBS) is currently performed on a fraction of samples sent for automated complete cell count. 39 children (age range 0-16.2 years) referred to a private paediatric practice during a 16-month period were retrospectively reviewed. Clinical scenarios, haematological features, laboratory-initiated PBS review, haematologist's PBS review and final diagnosis were described. Clinical indications included isolated thrombocytopenia (n = 10), unexplained bruises (n = 5), acute febrile illnesses (n = 11), anaemia (n = 8) and others (n = 5). The laboratory reviewed the PBS in 30 cases according to preset criteria and made no conclusive remarks. All slides were reviewed by a haematologist and a diagnosis was made in 27 (69%) cases, including 7 (78%) of the nine slides the laboratory did not review. The practice of laboratory-initiated PBS review requires re-evaluation. Haematologist-reviewed PBS is an important diagnostic tool for children with anaemia, bleeding disorders and acute febrile illnesses.