Granulocyte transfusions in life-threatening infections of children with hemato-oncological diseases.

Granulocytes are the most important cells for host defense during infections. Granulocyte suspension transfusions (GTx) may be given as additional treatment in severely neutropenic patients with life-threatening infections when antimicrobial therapy is inadequate. The aim of this study was to evaluate the effectiveness and safety of GTx for the treatment of children with hemato-oncological disease, febrile neutropenia and serious life-threatening infections. Patients who underwent GTx between July 2020 and September 2022 were evaluated retrospectively. Hematologic and clinical response rates, adverse effects, characteristics of infection episodes and survival data of the patients were analyzed. During the study period, 60 patients received a total of 313 GTx for 81 infection episodes with a median number of GTx/infection episode of 3 (range 1-29). The median neutrophil count per bag was 20.8 (range 7.9-68.3) × 109 and the median neutrophil count per kg body weight was 0.82 (range 0.17-9.2) × 109. Clinical response was 85 %. Clinical response decreased significantly as the duration of neutropenia increased (p = 0.002). Hematologic response was calculated in 198 GTx (GTx given with pre-transfusion neutrophil count ≤ 0.5 × 109/L); hematologic response rate was 34 %. The infection-related mortality was 15 % and overall survival rate was 87 % and 70 % on days 30 and 90, respectively. No serious side effects were observed in any patient. Granulocyte transfusions appear to be safe and effective supportive treatment in neutropenic children with hematologic/oncologic diseases and severe infections.

The effect of granulocyte colony-stimulating factors on survival parameters in pediatric patients with acute lymphoblastic leukemia: A retrospective study.

OBJECTIVE: There is a paucity of data concerning the use of granulocyte colony-stimulating factors (G-CSFs) in pediatric patients with acute lymphoblastic leukemia (ALL). The aim of the present study was to evaluate the effect of G-CSF use on relapse-free and overall survival in 358 consecutive, newly diagnosed pediatric ALL patients uniformly treated at the same institution between April 2012 and April 2020. MATERIALS AND METHODS: Patients were evaluated in two separate periods, based on the G-CSF treatment approach. All patients who underwent ALL treatment between April 2012 and December 2016 received G-CSF (G-CSF+ arm; n: 245) in the course of the protocol for reducing the risk of febrile neutropenia and/or inducing neutrophil recovery to prevent any treatment delay. No patients after December 2016 received G-CSF, even if they belonged to the high-risk group, and these were included in the G-CSF- arm (n: 113). RESULTS: Estimated mean relapse-free (106.5 months; 95 % CI 102-110.8 vs 82 months 95 % CI 75.2-88.9; p: 0.794) and overall survival (111.4 months; 95 % CI 108-114.8 vs 85 months 95 % CI 80.4-89.8; p: 0.431) rates were similar between the G-CSF+ and G-CSF- groups. CONCLUSIONS: Our findings indicate that G-CSF use during ALL treatment had no effect on relapse rates or overall survival.

Therapeutic plasma exchange in non-hematooncological disorders in pediatrics: A single center experience.

The use of therapeutic plasma exchange in the pediatric age group is mostly based on retrospective, single-center experiences. The decision to implement apheresis in pediatric patients is usually adopted from the results of studies on adult patients. In order to expand the limited data on pediatric TPE in general and non-hematooncological disorders in particular, we retrospectively evaluated TPE experience in pediatric patients who underwent the procedure for reasons other than hematooncological disorders. A total of 160 sessions in 34 patients (21 females and 13 males) with a median age of 7 (1-17) were analyzed. Most of the patients had sepsis and organ failure (12 patients, 35 procedures). In only one patient (2.9%) with methyl malonic aciduria (MMA) and sepsis, the procedure was terminated due to a grade 3 allergic reaction. Among the study cohort, 4 patients passed away. No patient died due to complications of TPE. The relatively low discontinuation rate and the lack of procedure-related mortality indicate that TPE is generally well tolerated in the pediatric age group similar to the adult population. However, since there are very limited evidence-based data on TPE use, especially in the pediatric age group, retrospective case series may also be helpful for clinicians in the decision-making process.

Vitamin D receptor polymorphisms in immune thrombocytopenic purpura.

BACKGROUND: Vitamin D receptor (VDR) polymorphisms have been studied in immune-mediated disorders, but not yet in immune thrombocytopenic purpura (ITP). We investigated whether VDR variants were associated with ITP in children. METHODS: The study included 44 children with a diagnosis of ITP and 100 healthy controls. Five VDR polymorphisms (Cdx-2, FokI, BsmI, ApaI and TaqI) were genotyped and used to evaluate the association of VDR variants with ITP. RESULTS: The distribution of the three Cdx-2 genotype groups (GG, GA, and AA) was significantly different between ITP patients and controls (P = 0.025); the homozygous GG genotype of Cdx-2 was overrepresented in ITP patients. The frequency of the A allele of Cdx-2 was significantly different between patients and controls (P = 0.01). The A allele of Cdx-2 was associated with a decreased risk of ITP (OR, 0.343; 95% CI: 0.150-0.782). No statistically significant difference was found between the ITP group and control group for Fok1, Bsm1, Apa1, and Taq1 polymorphisms (P > 0.5). CONCLUSION: There appears to be an interaction between the Cdx-2 variant of VDR and childhood immune thrombocytopenia.

ABO blood group mismatched hematopoietic stem cell transplantation.

Apart from solid organ transplantations, use of ABO-blood group mismatched (ABO-mismatched) donors is acceptable in hematopoietic stem cell transplantation (HSCT) patients. About 20-40% of allogeneic HSCT recipients will receive grafts from ABO-mismatched donors. ABO incompatible HSCT procedures are associated with immediate and late consequences, including but not restricted to acute or delayed hemolytic reactions, delayed red blood cell recovery, pure red cell aplasia and graft-versus-host disease. This review summarizes the current knowledge about consequences of ABO-mismatched HSCT in terms of associated complications and will evaluate its impact on important outcome parameters of HSCT.

Successful Treatment of Macroglossia Due to Lymphatic Malformation With Sirolimus.

OBJECTIVE: To evaluate the effectiveness and safety of sirolimus therapy in a child with macroglossia due to lymphatic malformation. METHODS: Sirolimus treatment was applied to the patient with an initial dosing of 0.8 mg/m2 per dose, administered orally, twice daily at approximately 12-hour intervals. RESULTS: After 9 months of sirolimus therapy, there was a nearly complete resolution of lymphatic malformation. The last evaluation was performed 6 months after withdrawal of treatment, and the lesion had almost completely resolved. CONCLUSION: This article presents a novel approach to the treatment of lymphatic malformation of the tongue using sirolimus, which appears to be safe and effective for the management of complex cases.