Diagnosis and Management of Prenatal Hereditary Pyropoikilocytosis
ABSTRACT Hereditary pyropoikilocytosis (HPP) is a severe hemolytic anemia caused by variants in SPTA1, SPTB, and EPB41. These weaken horizontal interactions in the erythrocyte cytoskeleton, causing membrane fragmentation and splenic sequestration. It will readily cause fetal anemia and often hydrops fetalis. Prenatal diagnosis requires first ruling out
Connor Hartzell +6 more
wiley +1 more source
Progression from Carbapenem-Resistant Enterobacterales Colonization to Bacteremia in Hematopoietic Stem Cell Transplant Recipients: A Single-Center Cohort Study. [PDF]
Rodríguez-Feria D +3 more
europepmc +1 more source
Attitudes Toward Prenatal Interventions in the Fanconi Anemia Community
ABSTRACT Objective In‐utero cell and gene therapies may offer prenatal treatment options for inherited diseases. Preclinical data suggests in‐utero (IU) hematopoietic stem cell transplantation (HSCT) could prevent Fanconi anemia (FA) related bone marrow failure without genotoxic conditioning or immune suppression.
Tony Lum +4 more
wiley +1 more source
ABSTRACT Sickle cell disease (SCD) affects millions worldwide but has limited treatment options, most of which carry significant side effects. At present, the only curative treatment for SCD is allogeneic or gene‐modified autologous hematopoietic stem cell (HSC) transplantation (Tx).
Oluwaseun O. Babatunde +4 more
wiley +1 more source
Calcineurin inhibitor-induced pain syndrome: an uncommon side effect of immunosuppressive therapy after allogeneic hematopoietic stem cell transplant. a case report and review of the literature. [PDF]
De Domenico R +8 more
europepmc +1 more source
Sickle Cell Disease: Historical Overview and Current Therapies
ABSTRACT Sickle cell disease (SCD) affects millions worldwide, yet the limited treatment options currently available do not always adequately control the disease and carry significant side effects. At present, the only curative treatment is hematopoietic stem cell (HSC) transplantation, a procedure that carries considerable challenges and numerous ...
Oluwaseun O. Babatunde +4 more
wiley +1 more source
Single‐center retrospective data showed that ATG‐based myeloablative haploidentical hematopoietic stem cell transplantation provides durable remission and favorable long‐term survival in children with refractory/relapsed non‐Hodgkin lymphoma, achieving high engraftment rates, low relapse incidence, and acceptable toxicity.
Chenguang Jia +12 more
wiley +1 more source
Cognitive Functioning in Vorinostat-Treated Pediatric and Young Adult Patients Over the First 180 Days After Hematopoietic Stem Cell Transplant. [PDF]
Votruba KL +11 more
europepmc +1 more source
Precision immunomodulation for pediatric hemophagocytic lymphohistiocytosis in intensive care
This review presents a bedside framework for recognizing pediatric hemophagocytic lymphohistiocytosis and cytokine storm, stabilizing organ dysfunction, identifying the underlying phenotype, selecting targeted immunomodulation, considering extracorporeal adjuncts, and reassessing response within 24–72 h. ABSTRACT Hemophagocytic lymphohistiocytosis (HLH)
Weerapong Lilitwat, Prakreeti Bhandari
wiley +1 more source

