Donor lymphocyte infusion to eradicate recurrent host hematopoiesis after allogeneic BMT for sickle cell disease.

peer reviewedBACKGROUND: Donor lymphocyte infusion (DLI) is currently standard therapy for relapse of malignancies after allogeneic BMT. Several observations suggest that both normal and leukemic progenitor cells of host origin constitute effective target cells for donor-derived lymphocytes. To prevent relapse of sickle cell disease (SCD), a child with evidence of decreasing mixed chimerism received DLIs 8 months after allogeneic BMT for SCD. CASE REPORT: A 4-year-old child who was homozygous for SCD underwent a transplantation of bone marrow from his fully HLA-matched sister. Routine detection of sex chromosomes in bone marrow cells evidenced decreasing mixed chimerism, which heralded a probably imminent recurrence of the disease. The patient received two DLIs in graded incremental doses on Days 234 and 267. One month later, he developed grade 2 acute GVHD that responded well to corticosteroids and cyclosporine. RESULTS: DLI resulted in complete donor chimerism within 2 months of the second infusion. Now, 2 years after the second DLI, the patient is in excellent condition, with normal Hb and excellent growth and development. CONCLUSION: This is the first report of successful use of DLI in a patient with probable imminent SCD recurrence after allogeneic BMT. It shows that DLI can displace residual host HPCs in case of recurrence of nonmalignant disease after allogeneic BMT

Pancytopenia Due to Vitamin B12 and Folic Acid Deficiency—A Case Report

peer reviewedWe report a case of severe pancytopenia in a 15-year-old patient due to a severe deficiency in vitamin B12 and folic acid, probably of nutritional origin. The clinical and biological course was favorable after vitamin supplementation. With this case, we discuss the diagnostic approach of pancytopenia with megaloblastic anemia in children and adolescents, as well as the mechanisms involved in vitamin B12 and B9 deficiency. Hypovitaminosis B12 is known in its severe form but its diagnosis is often made difficult by insidious signs and symptoms. Traditional intramuscular replacement therapy has now proven to be effective orally. The clinical manifestations of folic acid deficiency are relatively similar to those of vitamin B12 deficiency, reflecting their intricate co-enzymatic functions. Its supplementation is administered orally

Schizencephaly associated with a severe prothrombotic syndrome caused by antithombin III deficiency

peer reviewedaudience: professional, studen

A SUCCESSFUL COLLABORATION BETWEEN ADULT AND PEDIATRIC INTENSIVISTS: VENO-VENOUS EXTRACORPOREAL MEMBRANE OXYGENATION FOR A PEDIATRIC PATIENT WITH LIFE-THREATENING TRACHEAL OBSTRUCTION

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Immunoglobulin Heavy Chain High-Throughput Sequencing in Pediatric B-Precursor Acute Lymphoblastic Leukemia: Is the Clonality of the Disease at Diagnosis Related to Its Prognosis?

peer reviewedHigh-throughput sequencing (HTS) of the immunoglobulin heavy chain (IgH) locus is a recent very efficient technique to monitor minimal residual disease of B-cell precursor acute lymphoblastic leukemia (BCP-ALL). It also reveals the sequences of clonal rearrangements, therefore, the multiclonal structure, of BCP-ALL. In this study, we performed IgH HTS on the diagnostic bone marrow of 105 children treated between 2004 and 2008 in Belgium for BCP-ALL in the European Organization for Research and Treatment of Cancer (EORTC)-58951 clinical trial. Patients were included irrespectively of their outcome. We described the patterns of clonal complexity at diagnosis and investigated its association with patients' characteristics. Two indicators of clonal complexity were used, namely, the number of foster clones, described as clones with similar D-N2-J rearrangements but other V-rearrangement and N1-joining, and the maximum across all foster clones of the number of evolved clones from one foster clone. The maximum number of evolved clones was significantly higher in patients with t(12;21)/ETV6:RUNX1. A lower number of foster clones was associated with a higher risk group after prephase and t(12;21)/ETV6:RUNX1 genetic type. This study observes that clonal complexity as accessed by IgH HTS is linked to prognostic factors in childhood BCP-ALL, suggesting that it may be a useful diagnostic tool for BCP-ALL status and prognosis

Quality of life of long-term childhood acute lymphoblastic leukemia survivors:Comparison with healthy controls

peer reviewed[en] OBJECTIVE: Improved treatment landscape has led to better outcomes for paediatric acute lymphoblastic leukemia (ALL) survivors. As the number of survivors increase, we need to elucidate the long-term quality of life (QoL) and domains of complaints in these patients. Furthermore, the main priorities of these patients need to be clarified. We assessed long-term QoL outcomes of survivors of childhood ALL compared to matched population controls. METHODS: QoL data were collected from survivors recruited in France and Belgium between 2012 and 2017, including the Short Form Health Survey (SF-12) and the Quality of Life Systemic Inventory (QLSI). The Wilcoxon test was used to compare SF-12 scale scores between survivors and matched population controls. For the QLSI, comparisons were mainly descriptive. RESULTS: One hundred and eighty-six survivors (mean age: 27.6 years; range: 18.1-52.8) at follow-up completed QoL measures, amongst whom 180 were matched to controls. Overall, survivors had higher QoL on all SF12 scale scores, indicating that they had better functioning compared to controls. Statistically significant differences on the SF12 were observed for Vitality, Social Functioning, Role Limitations due to Emotional Problems and Mental Health scales. QLSI outcomes suggested that survivors were happier than controls with Couple and Social Relations. Controls were unhappiest compared to survivors with Money, Love life, Self-esteem, Nutrition and Paid Work. CONCLUSIONS: Our findings suggest that survivors of childhood ALL have better QoL outcomes on some domains compared to the general population, specifically around social and emotional functioning, and that they tend to prioritize their relationships more. Interventions for improving QoL outcomes, might build on existing positive experiences with family, friends and partners