Allogeneic hematopoietic cell transplantation (HCT) in patients with myelodysplastic syndrome (MDS) improves overall survival. The authors evaluated the impact of MDS genetics on the benefit of HCT in a biological assignment (donor v no donor) study. https://pubmed.ncbi.nlm.nih.gov/37607457/ ALSO AS VIDEO ON https://youtu.be/JbHS_-2sJQc CONNECT WITH US https://twitter.com/TheEBMT_Trainee https://www.ebmt.org/trainee-committee https://twitter.com/CarmeloGurnari

In this study, Nathanial and colleagues systematically analyzed CD19 CAR-T cells of 10 children with R/R B-ALL enrolled in the CARPALL trial via high-throughput single-cell gene expression and T cell receptor sequencing of infusion products and serial blood and bone marrow samples up to 5 years after infusion. Article: https://www.nature.com/articles/s41591-023-02415-3 As VIDEO on https://youtu.be/z_aJgRHpEaI

Chimeric antigen receptor (CAR) T-cell therapy is a promising option for patients with heavily treated multiple myeloma. Point-of-care manufacturing can increase the availability of these treatments worldwide. Aina and colleagues assessed the safety and activity of ARI0002h, a BCMA-targeted CAR T-cell therapy developed by academia, in patients with relapsed or refractory multiple myeloma. https://pubmed.ncbi.nlm.nih.gov/37414060/ ALSO AS VIDEO ON https://youtu.be/Ymgg22roWGU CONNECT WITH US https://twitter.com/TheEBMT_Trainee https://www.ebmt.org/trainee-committee https://twitter.com/CarmeloGurnari

Immunotherapy with chimeric antigen receptor (CAR)–expressing T cells that target the disialoganglioside GD2 expressed on tumor cells may be a therapeutic option for patients with high-risk neuroblastoma. In an academic, phase 1–2 clinical trial, the investigators enrolled patients (1 to 25 years of age) with relapsed or refractory, high-risk neuroblastoma in order to test autologous, third-generation GD2-CAR T cells expressing the inducible caspase 9 suicide gene (GD2-CART01). https://www.nejm.org/doi/full/10.1056/NEJMoa2210859 ALSO AS VIDEO ON YOUTUBE https://youtu.be/o1DVDcrrHFA CONNECT WITH US https://twitter.com/TheEBMT_Trainee https://www.ebmt.org/trainee-committee https://twitter.com/CarmeloGurnari

The presence of TP53 mutations is associated with an unfavorable outcome  in patients allografted for acute myeloid leukemia (AML), leading some  to question the benefit of an allogeneic stem cell transplantation  (allo-SCT) for this patient group, although this has not been studied in  a large cohort.  Article: https://acsjournals.onlinelibrary.wiley.com/doi/10.1002/cncr.34268  ALSO AS AUDIO ON https://podcasts.apple.com/us/podcast/trainee-pearls/id1596786132 https://open.spotify.com/show/2M1PWPZGW9Bz7PL5eh103Y CONNECT WITH US https://twitter.com/TheEBMT_Trainee https://www.ebmt.org/trainee-committee https://twitter.com/CarmeloGurnari

Richter transformation (RT) is a paradigmatic evolution of chronic  lymphocytic leukemia (CLL) into a very aggressive large B cell lymphoma  conferring a dismal prognosis. The mechanisms driving RT remain largely  unknown.   Article: https://www.nature.com/articles/s41591-022-01927-8   ALSO AS AUDIO ON https://podcasts.apple.com/us/podcast/trainee-pearls/id1596786132  https://open.spotify.com/show/2M1PWPZGW9Bz7PL5eh103Y   CONNECT WITH US  https://twitter.com/TheEBMT_Trainee  https://www.ebmt.org/trainee-committee https://twitter.com/CarmeloGurnari

Risk stratification and therapeutic decision-making for myelodysplastic syndromes (MDS) are based on the International Prognostic Scoring System–Revised (IPSS-R), which considers hematologic parameters and cytogenetic abnormalities. Somatic gene mutations are not yet used in the risk stratification of patients with MDS. Combining genomic profiling with hematologic and cytogenetic parameters, the IPSS-M improves the risk stratification of patients with MDS and represents a valuable tool for clinical decision-making. CONNECT WITH US  https://twitter.com/TheEBMT_Trainee  https://www.ebmt.org/trainee-committee  https://twitter.com/CarmeloGurnari

Schimke immuno-osseous dysplasia (SIOD) is a multisystem disorder that  is inherited in an autosomal recessive pattern. It usually manifests  first with growth failure. Other features of the disease are generally  noted in the ensuing evaluation of the growth failure or develop in the  following years.    Article:  https://www.nejm.org/doi/10.1056/NEJMoa2117028?url_ver=Z39.88-2003&rfr_id=ori:rid:crossref.org&rfr_dat=cr_pub%20%200pubmed    ALSO AS AUDIO ON  https://podcasts.apple.com/us/podcast/trainee-pearls/id1596786132  https://open.spotify.com/show/2M1PWPZGW9Bz7PL5eh103Y   YOUTUBE  "EBMT Trainees" CONNECT WITH US  https://twitter.com/TheEBMT_Trainee  https://www.ebmt.org/trainee-committee  https://twitter.com/CarmeloGurnari

Oncogenic alterations underlying B-cell acute lymphoblastic leukemia  (B-ALL) in adults remain incompletely elucidated. To uncover novel  oncogenic drivers, Marie and her team performed RNA-seq and whole-genome  analyses in a large cohort of unresolved B-ALL. Marie and colleagues  identified a novel subtype characterized by a distinct gene expression  signature and the unique association of two genomic microdeletions.   Article link: https://ashpublications.org/blood/article/doi/10.1182/blood.2021014723/484449/Concurrent-CDX2-cis-deregulation-and-UBTF-ATXN7L3

Hemophagocytic lymphohistiocytosis (HLH; hemophagocytic syndrome) is a  rare syndrome of potentially fatal, uncontrolled hyperinflammation.  Allogeneic hematopoietic stem cell transplantation is indicated in  primary, recurrent or progressive HLH, but information about its  outcomes in the adult population is limited. Rafal works at the Department of Hematology, Transplantation and  Internal Medicine, Medical University of Warsaw, Warsaw, Poland. And he  is part of YoungEHA.