Ruxolitinib for the treatment of acute and chronic graft-versus-host disease in children: a systematic review and individual patient data meta-analysis.

Steroid-refractory graft-versus-host disease (SR-GvHD) represents a major complication of pediatric allogenic hematopoietic stem cell transplantation. Ruxolitinib, a selective JAK 1-2 inhibitor, showed promising results in the treatment of SR-GvHD in adult trial, including patients >12 years old. This systematic review aims to evaluate ruxolitinib use for SR-GvHD in the pediatric population.

Case report: Venetoclax therapy in a boy with acute myeloid leukemia in Shwachman Diamond syndrome.

Shwachman-Diamond syndrome (SDS) is a rare bone marrow failure syndrome characterized by exocrine pancreatic insufficiency, bone abnormalities, progressive cytopenia, and predispositions to myelodysplastic syndrome (MDS) and acute myeloid leukemia (AML). AML, in these patients, is associated with a poor prognosis and with an increased risk of organ toxicity and infectious complications from chemotherapy and hematopoietic stem cell transplantation (HSCT), thus leading to high rates of treatment-related morbidity and mortality. The BCL-2 inhibitor venetoclax has revolutionized the treatment of AML in elderly adults, especially for treatment-naive elderly patients who are ineligible for intensive chemotherapy.

Pre-Transplant Total Lymphocyte Count Determines Anti-Thymocyte Globulin Exposure, Modifying Graft-versus-Host Disease Incidence and Post-Transplant Thymic Restoration: A Single-Center Retrospective Study.

The use of anti-thymocyte globulin (ATG) as part of conditioning to prevent graft-versus-host disease (GVHD) may severely impair immune reconstitution (IR). We analyzed relationships between ATG exposure, the recipient lymphocyte count, IR, and transplant outcome. We retrospectively reviewed patients aged ≤ 18 years who underwent allogeneic HSCT between April 2005 and April 2020.

New Applications of JAK/STAT Inhibitors in Pediatrics: Current Use of Ruxolitinib.

Janus kinases (JAK) are a family of tyrosine kinases (JAK1, JAK2, JAK3, and TYK2) that transduce cytokine-mediated signals through the JAK-STAT metabolic pathway. These kinases act by regulating the transcription of specific genes capable of inducing biological responses in several immune cell subsets. Inhibition of Janus kinases interferes with the JAK-STAT signaling pathway.

Glucocorticoids inhibit human hematopoietic stem cell differentiation toward a common ILC precursor.

Background: Innate lymphoid cells (ILCs) comprise cytotoxic natural killer (NK) cells and helper ILCs (hILCs). Human hILC development is less characterized as compared with that of NK cells, although all ILCs are developmentally related. It has been reported that the immunosuppressive drugs glucocorticoids (GCs) regulate ILC function, but whether they control ILC differentiation from hematopoietic stem cells (HSCs) is unknown.

Mechanical, Wear and Thermal Behavior of Polyethylene Blended with Graphite Treated in Ball Milling.

Additive manufacturing, civil, and biomechanical applications are among the most important sectors, where the filler's presence can significantly improve the quality of polymeric products blends. The high market demand of new low-cost material to be used as shock absorbers and mechanical joints arouses our curiosity to study a relatively common commercial polymer and filler. The possible improvement by blending high-density polyethylene (HDPE) and graphite was investigated for these sectors.

How Age Matters in the Assessment of Vertigo in the Pediatric Emergency Department: A 10-Year Age-Stratified Etiology Survey.

Vertigo is a relatively frequent cause for referral to the pediatric emergency department, and it is usually caused by benign or self-limiting etiology. However, it could be difficult to evaluate especially in the younger child and could also conceal serious illness as encephalitis or cerebellitis. Our survey collected in a 10-year period 757 children assessed in pediatric emergency department for vertigo and stratified this population for etiology and for group of age: younger than 6 years (113, 14.

Selumetinib in the Treatment of Symptomatic Intractable Plexiform Neurofibromas in Neurofibromatosis Type 1: A Prospective Case Series with Emphasis on Side Effects.

Background: Plexiform neurofibromas (PN) are congenital tumors that affect up to 50% of individuals with neurofibromatosis type 1. Despite their benign nature, they can grow rapidly and cause severe morbidities. Selumetinib, an inhibitor of mitogen-activated protein kinase (MEK) 1 and 2, was reported to induce a clinical response in pediatric subjects with inoperable PN.

Treatment of hepatitis C in two paediatric patients using sofosbuvir during haematopoietic stem cell transplantation.

We report the first two paediatric cases of sofosbuvir treatment during high-intensity myeloablative conditioning and engraftment phases of haematopoietic stem cell transplantation. These reports highlight the safety of sofosbuvir during all phases of transplantation and the lack of interaction between sofosbuvir and alkylating or immunosuppressive agents.

Complete Remission of a Refractory Acute Myeloid Leukemia with Myelodysplastic- and Monosomy 7-Related Changes after a Combined Conditioning Regimen of Plerixafor, Cytarabine and Melphalan in a 4-Year-Old Boy: A Case Report and Review of Literature.

Acute myeloid leukemia with myelodysplastic changes and monosomy 7 is a rare form of pediatric leukemia associated with very poor disease-free survival. The refractoriness of the disease is due to the protection offered by the bone marrow niche, making leukemic stem cells impervious to whatever chemotherapy or myeloablative regimen is chosen. Using a mobilizing agent for haematopoietic stem cells, Plerixafor, could sensitise leukemic cells to the myeloablative therapy.