Late-onset pulmonary complications following allogeneic hematopoietic cell transplantation in pediatric patients: a prospective multicenter study.

The primary objective of our multicenter prospective study was to describe the incidence of late-onset non-infectious pulmonary complications (LONIPCs) in children undergoing hematopoietic cell transplantation (HCT) using sensitive criteria for pulmonary function test (PFT) abnormalities including the non-specific pattern of airflow obstruction. Secondary objectives were to assess the factors associated with LONIPC occurrence and the sensitivity of the 2014 NIH-Consensus Criteria of bronchiolitis obliterans syndrome (BOS). PFT and clinical assessment were performed prior to HCT and at 6, 12, 24, and 36 months post-HCT.

Sebelipase alfa enzyme replacement therapy in Wolman disease: a nationwide cohort with up to ten years of follow-up.

Background: Wolman disease (WD), the rapidly progressive phenotype of lysosomal acid lipase (LAL) deficiency, presents in neonates with failure to thrive and hepatosplenomegaly, and leads to multi-organ failure and death before 12 months of age. In clinical trials, enzyme replacement therapy (ERT) with sebelipase alfa led to improved survival, growth and biological parameters in WD patients followed up to 5 years. Long-term follow-up and health-related quality of life (HRQoL) evaluation are lacking.

Generalized Verrucosis Revealing a Life-Threatening and Unlabeled T-Cell Lymphopenia Associated With Autoimmune Hemolytic Anemia: A Case Report and Review of Literature.

Primary immunodeficiencies are inherited disorders, which may be revealed in the context of autoimmune hemolytic anemia (AIHA). We report the case of a girl presenting with an enterovirus-related AIHA. Despite being in complete remission for her anemia after treatment, the initial CD4/CD8 lymphopenia dramatically worsened with time.

Major Issues of Care in Thalassemia Major Children Refugees.

Beta thalassemia major (βTM) is the most common inherited hemoglobinopathy. Management essentially focuses on preventing and treating complications. Conventional treatment is based on a regular blood transfusion program, and chelation therapy.

Juvenile myelomonocytic leukaemia and Noonan syndrome.

Background: Infants with Noonan syndrome (NS) are predisposed to developing juvenile myelomonocytic leukaemia (JMML) or JMML-like myeloproliferative disorders (MPD). Whereas sporadic JMML is known to be aggressive, JMML occurring in patients with NS is often considered as benign and transitory. However, little information is available regarding the occurrence and characteristics of JMML in NS.

Liposomal daunorubicin (Daunoxome) and polyethylated glycol conjugated asparaginase (PEG-ASPA) in children with relapsed and refractory acute lymphoblastic leukemia treated on compassionate basis.

Background: Daunoxome (DNX) is an encapsulated form of daunorubicin in liposomal vesicles with suggested better pharmacokinetics, pharmacodynamics and lesser cardio toxicity than the free form. Polyethyl glycol asparaginase (PEG-ASPA), a modified form of L-asparaginase, has better activity and lesser immunogenicity than its native molecule.

Aim: To evaluate on a compassionate basis, the combination of Daunoxome and PEG-ASPA, as regard to efficacy and toxicity, as a salvage treatment in refractory/relapsed childhood ALL.