Hemolytic Anemia With Acanthocytes During Alectinib Treatment of Anaplastic T-Cell Lymphoma: A Case Report and Literature Review.

Alectinib, an ALK inhibitor used for ALK+ non-small cell lung cancer and other malignancies, has been associated with anemia and RBC abnormalities, including acanthocytosis. We report the first case of alectinib-induced acanthocytosis and hemolysis causing anemia during treatment for anaplastic large cell lymphoma in an 11-year-old boy. Extensive testing, including next-generation sequencing, and a specific indirect antiglobulin test conducted with alectinib, was performed to document this hemolytic anemia.

Incidence and risk factors of graft failure in allogeneic hematopoietic stem cell transplantation for mucopolysaccharidosis in a nationwide pediatric cohort. A study on behalf of the Francophone Society of Bone Marrow Transplantation and Cellular Therapy.

Context: Mucopolysaccharidosis (MPS) requires urgent treatment to prevent neurological damage. While gene therapy holds promise for effectively treating these diseases with minimal toxicity, access remains limited for most patients. Consequently, advancing allogeneic hematopoietic stem cell transplantation (HSCT) for young children is crucial.

Four Cases of Myeloproliferative Disorders Associated With Down Syndrome: Distinguishing ML-DS From TAM-DS: Distinguishing TAM-DS and ML-DS: Report of 4 Cases.

Down syndrome (DS) is defined by an extra copy of chromosome 21 and confers an increased susceptibility to hematological disorders. Transient abnormal myelopoiesis (TAM) and myeloid-leukemia associated with Down syndrome (ML-DS) are two conditions that need to be accurately diagnosed to provide appropriate management. Both TAM and ML-DS are characterized by proliferation of megakaryoblasts carrying a mutation in the GATA1 gene.

Experience and andrological follow-up after testicular tissue cryopreservation.

Research Question: What is the experience and mid- and long-term andrological health follow-up of (pre)pubertal males who have undergone testicular tissue freezing (TTF)?

Design: This single-centre longitudinal retrospective cohort study reports on the mid- and long-term andrological health follow-up of (pre)pubertal males and young adults who underwent TTF for fertility preservation between January 2007 and December 2018. Medical characteristics and questionnaire data collected more than 18 months after TTF were analysed.

Results: Thirty-six patients were revisited during a medical follow-up consultation.

Chemo-, Regio-, and Stereoselective -Hydroboration of 1,3-Enynes: Copper-Catalyzed Access to ()- and ()-2-Boryl-1,3-dienes.

A copper-catalyzed alkyne-selective hydroboration of 1,3-enynes is disclosed, providing access to the previously elusive 2-boryl-1,3-dienes. Using CuOAc, Xantphos, and HBpin, Bpin was installed on the internal carbon of a series of symmetric and nonsymmetric 1,3-enynes, affording products with excellent : selectivity. The utility of the 2-boryl-1,3-diene products was demonstrated by transformation to useful functional groups.

Childhood myelodysplastic syndromes: Is cytoreductive therapy useful before allogeneic hematopoietic stem cell transplantation?

For most patients with childhood myelodysplastic syndrome (cMDS), allogeneic hematopoietic stem cell transplantation (allo-HSCT) remains the only curative option. In the case of increased blasts (cMDS-IB), the benefit of pretransplant cytoreductive therapy remains controversial. In this multicenter retrospective study, the outcomes of all French children who underwent allo-HSCT for cMDS reported in the SFGM-TC registry between 2000 and 2020 were analyzed ( = 84).

[Preventive and therapeutic strategies for relapse after hematopoietic stem cell transplant for pediatric AML (SFGM-TC)].

Treatment of pediatric high-risk acute myeloid leukemia (AML), defined either on molecular or cytogenetic features, relies on bone marrow transplant after cytologic remission. However, relapse remains the first post-transplant cause of mortality. In this 13 session of practice harmonization of the francophone society of bone marrow transplantation and cellular therapy (SFGM-TC), our group worked on recommendations regarding the management of post-transplant relapse in AML pediatric patients based on international literature, national survey and expert opinion.

Researching COVID to enhance recovery (RECOVER) pediatric study protocol: Rationale, objectives and design.

Importance: The prevalence, pathophysiology, and long-term outcomes of COVID-19 (post-acute sequelae of SARS-CoV-2 [PASC] or "Long COVID") in children and young adults remain unknown. Studies must address the urgent need to define PASC, its mechanisms, and potential treatment targets in children and young adults.

Observations: We describe the protocol for the Pediatric Observational Cohort Study of the NIH's REsearching COVID to Enhance Recovery (RECOVER) Initiative.

Phosphine-Catalyzed 1,2-cis-Diboration of 1,3-Butadiynes.

Trialkyl phosphines PMe and PEt catalyze the 1,2-cis-diboration of 1,3-butadiynes to give 1,2-diboryl enynes. The products were utilized to synthesize 1,1,2,4-tetraaryl enynes using a Suzuki-Miyaura protocol and can readily undergo proto-deborylation.

Design, Synthesis, and Antifungal Activity of 3-Substituted-2()-Oxaboroles.

Next generation antimicrobial therapeutics are desperately needed as new pathogens with multiple resistance mechanisms continually emerge. Two oxaboroles, tavaborole and crisaborole, were recently approved as topical treatments for onychomycosis and atopic dermatitis, respectively, warranting further studies into this privileged structural class. Herein, we report the antimicrobial properties of 3-substituted-2()-oxaboroles, an unstudied family of medicinally relevant oxaboroles.

A predictive classifier of poor prognosis in transplanted patients with juvenile myelomonocytic leukemia: a study on behalf of the Société Francophone de Greffe de Moelle et de Thérapie Cellulaire.

Juvenile myelomonocytic leukemia (JMML) is an aggressive pediatric myeloproliferative neoplasm requiring hematopoietic stem cell transplantation (HSCT) in most cases. We retrospectively analyzed 119 JMML patients who underwent first allogeneic HSCT between 2002 and 2021. The majority (97%) carried a RAS-pathway mutation, and 62% exhibited karyotypic alterations or additional mutations in SETBP1, ASXL1, JAK3 and/or the RAS pathway.

Deep brain stimulation of thalamic nucleus reuniens promotes neuronal and cognitive resilience in an Alzheimer's disease mouse model.

The mechanisms that confer cognitive resilience to Alzheimer's Disease (AD) are not fully understood. Here, we describe a neural circuit mechanism underlying this resilience in a familial AD mouse model. In the prodromal disease stage, interictal epileptiform spikes (IESs) emerge during anesthesia in the CA1 and mPFC regions, leading to working memory disruptions.

Regio- and Stereoselective Copper-Catalyzed Borylation-Protodeboronation of 1,3-Diynes: Access to ()-1,3-Enynes.

A facile method to access ()-1,3-enynes is realized via sequential copper-catalyzed regio- and stereoselective borylation-protodeboronation of 1,3-diynes. Pinacolborane, copper(II) acetate, and Xantphos as the ligand efficiently install hydrogen and Bpin in a fashion, which is followed by rapid hydrolysis with water. The reaction has wide substrate scope and occurs in a chemoselective fashion.

Development of an Oral Liquid Formulation of Nicardipine Hydrochloride Compounded with Simple Excipients for the Treatment of Pediatric Hypertension.

Nicardipine hydrochloride is an anti-hypertensive drug that is used off-label to treat hypertension in children. A previous oral formulation of nicardipine hydrochloride was developed using a commercial vehicle as an excipient. However, ready-to-use vehicles are prone to supply shortages, and their composition may undergo substantial modifications.

Disrupted neural correlates of anesthesia and sleep reveal early circuit dysfunctions in Alzheimer models.

Dysregulated homeostasis of neural activity has been hypothesized to drive Alzheimer's disease (AD) pathogenesis. AD begins with a decades-long presymptomatic phase, but whether homeostatic mechanisms already begin failing during this silent phase is unknown. We show that before the onset of memory decline and sleep disturbances, familial AD (fAD) model mice display no deficits in CA1 mean firing rate (MFR) during active wakefulness.

Biomimetic matrix for the study of neuroblastoma cells: A promising combination of stiffness and retinoic acid.

Neuroblastoma is the third most common pediatric cancer composed of malignant immature cells that are usually treated pharmacologically by all trans-retinoic acid (ATRA) but sometimes, they can spontaneously differentiate into benign forms. In that context, biomimetic cell culture models are warranted tools as they can recapitulate many of the biochemical and biophysical cues of normal or pathological microenvironments. Inspired by that challenge, we developed a neuroblastoma culture system based on biomimetic LbL films of physiological biochemical composition and mechanical properties.