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Maternal administration of busulfan before in utero hematopoietic cell transplantation improves congenic bone marrow cells engraftment in murine model
In utero hematopoietic cell transplantation (IUHCT) is a nonmyeloablative procedure that leads to donor cell chimerism and donor-specific tolerance[1]. IUHCT offers the ability to cure a variety of congenital diseases, including sickle cell disease and thalassemia[2]. Under experiment al conditions, IUHCT has been performed successfully in murine[3,4], sheep[5,6], pig[7,8], dog[9,10] and nonhuman primate models[11,12]. The theoretical advantages of IUHCT (na ïve fetal immune system, potentially expanding hematopoietic niches, and small fetal size) have been used to describe the fetus as the perfect recipient of hematopoie...
Source: Biology of Blood and Marrow Transplantation - February 6, 2024 Category: Hematology Authors: Chunyu Shi, Zhongmin Li, Zhanwei Sun, Lu Pan Source Type: research

Comparison of melphalan dose in patients with myelodysplastic syndrome undergoing allogeneic transplantation with reduced-intensity conditioning
The utilization of allogenic hematopoietic stem cell transplantation (allo-HSCT) as a therapeutic intervention for myelodysplastic syndrome (MDS) presents the potential for curative outcomes. However, allo-HSCT also incurs non-relapse mortality (NRM) due to complications such as graft-versus-host disease (GVHD), severe infections, and regimen-related toxicities1-3. Initially, allo-HSCT was restricted to younger patients who were appropriate for myeloablative conditioning (MAC)4-6. However, the advent of reduced-intensity conditioning (RIC) has expanded the pool of eligible elderly patients for allo-HSCT7-9. (Source: Biolog...
Source: Biology of Blood and Marrow Transplantation - February 6, 2024 Category: Hematology Authors: Shuhei Kurosawa, Yoshimitsu Shimomura, Hidehiro Itonaga, Yuta Katayama, Makoto Onizuka, Masatsugu Tanaka, Hikaru Kobayashi, Yukiyasu Ozawa, Masashi Sawa, Junya Kanda, Noriko Doki, Shin Fujisawa, Naoyuki Uchida, Takahiro Fukuda, Yoshiko Atsuta, Ken Ishiyam Source Type: research

Cytomegalovirus Reactivations in Allogeneic Hematopoietic Stem Cell Transplantation from HLA-Matched and Haploidentical Donors with Post-Transplantation Cyclophosphamide
Viral infections, particularly cytomegalovirus (CMV), contribute significantly to morbidity and mortality in recipients of allogeneic hematopoietic stem cell transplantation (HSCT) [1]. Post-transplantation cyclophosphamide (PTCy)-based platforms for graft-versus-host disease (GVHD) prophylaxis are increasingly used in HSCT, but they may be associated with a higher risk of CMV reactivation [2,3], potentially increasing morbidity and nonrelapse mortality (NRM). Studies of CMV infection in the context of PTCy have focused on comparisons with calcineurin inhibitors or have contrasted HSCT from haploidentical (Haplo) donors wi...
Source: Biology of Blood and Marrow Transplantation - February 5, 2024 Category: Hematology Authors: Pedro Chor ão, Marta Henriques, Marta Villalba, Juan Montoro, Aitana Balaguer-Roselló, Eva María González, María Dolores Gómez, Inés Gómez, Pilar Solves, Marta Santiago, Pedro Asensi, Brais Lamas, Ana Bataller, Pablo Granados, Juan Eiris, David Ma Tags: Full Length Article Source Type: research

Cmv reactivations in allogeneic hematopoietic stem cell transplant from hla-matched and haploidentical donors with post-transplant cyclophosphamide
Viral infections, particularly cytomegalovirus (CMV), significantly contribute to morbidity and mortality in recipients of allogeneic hematopoietic stem cell transplant (HSCT)1. Post-transplant cyclophosphamide (PTCy)-based platforms for graft-versus-host disease (GVHD) prophylaxis are increasingly used in HSCT, but they may be associated with a higher risk of CMV reactivation2,3, potentially increasing morbidity and non-relapse mortality (NRM). Studies on CMV infections within the context of PTCy have focused on comparisons with calcineurin inhibitors or contrasted HSCT from haploidentical donors (HAPLO) with matched sibl...
Source: Biology of Blood and Marrow Transplantation - February 5, 2024 Category: Hematology Authors: Pedro Chor ão, Marta Henriques, Marta Villalba, Juan Montoro, Aitana Balaguer-Roselló, Eva María González, María Dolores Gómez, Inés Gómez, Pilar Solves, Marta Santiago, Pedro Asensi, Brais Lamas, Ana Bataller, Pablo Granados, Juan Eiris, David Ma Source Type: research

A day 14 endpoint for acute GVHD clinical trials
Acute graft versus host disease (GVHD) is the leading cause of non-relapse mortality (NRM) in patients undergoing hematopoietic cell transplantation (HCT). High doses of systemic steroids are the primary treatment of acute GVHD, resulting in responses in around 50% of patients 1,2. The overall response rate (ORR) of clinical symptoms after four weeks of treatment (standard clinical response) is a validated surrogate for NRM that has been widely adopted as the primary endpoint for clinical trials of acute GVHD treatment following a joint consensus statement by experts in 2009 3,4. (Source: Biology of Blood and Marrow Transplantation)
Source: Biology of Blood and Marrow Transplantation - February 4, 2024 Category: Hematology Authors: Nikolaos Spyrou, Yu Akahoshi, Steven Kowalyk, George Morales, Rahnuma Beheshti, Paibel Aguayo-Hiraldo, Monzr M. Al Malki, Francis Ayuk, Peter Bader, Janna Baez, Alexandra Capellini, Hannah Choe, Zachariah DeFilipp, Matthias Eder, Gilbert Eng, Aaron Etra, Source Type: research

Phase II Study of Pharmacokinetic Model-Based ATG Dosing to Improve Survival through Enhanced Immune Reconstitution in Pediatric and Adult Patients Undergoing Ex Vivo CD34-Selected Allogeneic HCT (PRAISE-IR)
Ex vivo CD34-selected allo-HCT is associated with favorable CRFS but limited by delayed immune reconstitution (IR) and in some trials higher non-relapse mortality (NRM) (BMT CTN 1301, JCO 2023). We recently reported that the use of traditional weight-based ATG dosing in this setting led to high post-HCT ATG pharmacokinetic (PK) exposure that was associated with delayed CD4+ T cell IR (CD4+IR) and increased NRM risk (Lakkaraja et al., Blood Adv 2022). (Source: Biology of Blood and Marrow Transplantation)
Source: Biology of Blood and Marrow Transplantation - February 1, 2024 Category: Hematology Authors: Michael Scordo, Miguel-Angel Perales, Audrey Mauguen, Andrew Lin, Binni Kunvarjee, Linh Khanh Nguyen, Jennifer Bieler, Maria Paes Pena, Christina Cho, Boglarka Gyurkocza, Andrew C. Harris, Ann A. Jakubowski, Dr. Richard J. Lin, Esperanza B. Papadopoulos, Tags: LBA-1 Source Type: research

Absence of Evidence for Pervasive CAR19 Driven T-Cell Lymphomagenesis Revealed By Comprehensive Genomic Profiling of an Index Tumor.
Despite the success of chimeric antigen receptor (CAR) T-cell therapies, concerns over toxicity remain. Recent reports indicate development of post-infusion T-cell lymphoma (TCL) after CAR therapy. There is minimal data regarding TCL development after commercially available CAR19 products. We analyzed 234 cases of lymphoma (189 LBCL, 20 FL, 25 MCL) treated with commercial CAR19 (100% axi-cel and brexu-cel). One patient, a 59-year-old female with CD19+/CD20+/EBV+ DLBCL, developed a post-infusion CD3+/CD4+/EBV+ T-cell lymphoma (Fig 1) diagnosed in the bone marrow (BM) on day 55 (D55). (Source: Biology of Blood and Marrow Transplantation)
Source: Biology of Blood and Marrow Transplantation - February 1, 2024 Category: Hematology Authors: Mark P. Hamilton, Takeshi Sugio, Troy Noordenbos, Shuyu Shi, Max Diehn, Dita Gratzinger, David B. Miklos, Ash A. Alizadeh, Zinaida Good Tags: LBA-2 Source Type: research

Reducing Ptcy Dose in Patients Receiving CAST for GvHD Prevention Results in Accelerated Engraftment at the Cost of Higher Incidence of GvHD
We previously reported excellent results of post-transplant cyclophosphamide (PTCy), abatacept (A) and a short course of tacrolimus (T) (CAST) for graft-versus-host (GvHD) prevention following peripheral blood (PB) haploidentical transplant. Mechanistic and preliminary clinical data suggest that the dose of PTCy can be decreased to diminish its toxicity while maintaining its efficacy. We sought to explore the feasibility and efficacy of reduced-dose PTCy in patients (pts) receiving CAST. (Source: Biology of Blood and Marrow Transplantation)
Source: Biology of Blood and Marrow Transplantation - February 1, 2024 Category: Hematology Authors: J Andres-Suarez Londono, Kelli Cole, Frank Cirrone, Stephanie Wo, M. Maher Abdul-Hay, Jingmei Hsu, Dr. Sharon Gardner, Mohammad Abu-Zaid, Gloria Contreras Yametti, Benjamin A Levinson, Judith D Goldberg, A. Samer Al-Homsi Tags: LBA-3 Source Type: research

Use of Teduglutide in the Management of Gastrointestinal Graft-versus-Host Disease in Children and Young Adults
Gastrointestinal (GI) graft-versus-host-disease (GVHD) is a life-threatening complication of allogeneic hematopoietic stem cell transplantation (HSCT) [1]. Current treatment options include glucocorticoids, antimetabolites, and JAK1-JAK2 inhibitors [2], which are immune suppressive and carry the risk of significant adverse effects [3]. More recently, glucagon-like peptide-2 (GLP-2) has been proposed as a therapeutic target for managing GI GVHD [4]. (Source: Biology of Blood and Marrow Transplantation)
Source: Biology of Blood and Marrow Transplantation - February 1, 2024 Category: Hematology Authors: Kristie N. Ramos, Daniel Leino, Nathan Luebbering, Michael S. Grimley, Priscila Badia, Stella M. Davies, Pooja Khandelwal Source Type: research

Phase 2 Trial of Ruxolitinib Cream for Chronic Cutaneous GvHD
Oral ruxolitinib is approved to treat aGvHD and cGvHD, while ruxolitinib cream is approved for atopic dermatitis and vitiligo. Topical corticosteroids are the mainstay of skin-directed therapy but may incompletely treat cutaneous cGvHD.We conducted a phase 2 prospective, randomized, double-blind trial evaluating the efficacy and safety of ruxolitinib 1.5% cream in patients ≥12 years old with cutaneous nonsclerotic and superficially sclerotic cGvHD with ≥2% of body surface area (BSA) affected. Patients were eligible if any systemic therapy was stable for ≥4 weeks and concurrent topical/photo- therapy was not used. (So...
Source: Biology of Blood and Marrow Transplantation - February 1, 2024 Category: Hematology Authors: Alina Markova, Esperanza B. Papadopoulos, Stephen Dusza, Veronica Rotemberg, Jelena Ostojic, Rizi Ai, Tara Maier, Mario E. Lacouture, Roni Tamari, Boglarka Gyurkocza, Brian C. Shaffer, Michael Scordo, Miguel-Angel Perales, Andrew C. Harris, Doris M. Ponce Tags: 1 Source Type: research

TSC-100 and TSC-101, TCR-T Cell Therapies That Target Residual Recipient Cells after Reduced Intensity Conditioning Transplantation, Induce Complete Donor Chimerism with Favorable Prognosis: Early Results of a Phase 1 Trial
Disease relapse after allogeneic hematopoietic cell transplantation (HCT) affects ∼40% of patients and has high mortality. A potential solution is to target antigens mismatched between transplant recipients and donors. TSC-100 and TSC-101 are allogeneic donor derived T-cell receptor-engineered T cells that target HA-1 and HA-2 hematopoietic cell antigens respectively, both pres ented on HLA-A*02:01. By choosing donors who are either HLA-A*02:01 or HA-1 negative, TSC-100 or TSC-101 selectively eliminate all residual patient hematopoietic cells post-HCT and prevent relapse (figure 1). (Source: Biology of Blood and Marrow Transplantation)
Source: Biology of Blood and Marrow Transplantation - February 1, 2024 Category: Hematology Authors: Monzr M. Al Malki, Alla Keyzner, Hyung C. Suh, Uday R. Popat, Nishant Dwivedi, Ashish S Kothari, Erica Buonomo, Yun Wang, Nina Abelowitz, Jim Murray, Gavin MacBeath, Debora Barton, Shrikanta Chattopadhyay, Ran Reshef Tags: 2 Source Type: research

Interim Results of a Phase I/II Trial of Intermediate-Dose Post-Transplantation Cyclophosphamide after Reduced Intensity Conditioned HLA-Mismatched Bone Marrow Transplantation for Older or Unfit Patients
High-dose post-transplantation cyclophosphamide (PTCy) is standard of care graft-versus-host disease (GVHD) prophylaxis for HLA-mismatched related (HLA-haploidentical, haplo) and HLA-mismatched unrelated (MMUD) donor hematopoietic cell transplantation (HCT). However, the optimal dosing of PTCy has not been defined. Our studies in murine HCT models support that intermediate-dose PTCy may provide superior protection against acute GVHD (aGVHD). A phase I/II study at the National Institutes of Health (NIH) of 25 mg/kg/day PTCy on days +3/+4 after haplo HCT with myeloablative conditioning (MAC) effectively prevented aGVHD, prov...
Source: Biology of Blood and Marrow Transplantation - February 1, 2024 Category: Hematology Authors: Shannon R. McCurdy, Mustafa A. Hyder, Ruby Sabina, Ashley DeVries, Kirstin Chalupa, Natasha Berryman, Jessica Bernhardt, Alison Christina Cusmano, Amy Chai, Rebecca Schwartz, Dimana Dimitrova, Kamil Rechache, Christi McKeown, Anita Stokes, Jennifer Sponau Tags: 3 Source Type: research

Triggered: How Damage Induced IL-18 Suppresses Thymus Regeneration
The thymus is highly sensitive to acute injury such as the cytoreductive conditioning given pre-hematopoietic cell transplant (HCT). The thymus is capable of a remarkable degree of regeneration, however its reparative capacity and T cell productivity decline with age leaving HCT recipients vulnerable to relapse of malignancy and opportunistic infection. Better understanding endogenous mechanisms of thymus regeneration may inform therapeutic interventions to improve T cell reconstitution in these patients. (Source: Biology of Blood and Marrow Transplantation)
Source: Biology of Blood and Marrow Transplantation - February 1, 2024 Category: Hematology Authors: David Granadier, Kirsten Cooper, Lorenzo Iovino, Dante V Acenas II, Paul deRoos, Jarrod A Dudakov Tags: 4 Source Type: research

CAR19 Therapy Drives Expansion of Clonal Hematopoiesis and Associated Cytopenias
For patients with relapsed/refractory large B-cell lymphomas (rrLBCL), CD19-directed chimeric antigen receptor T-cells (CAR19) improve survival compared to autologous hematopoietic cell transplantation (HCT). However, major toxicities of CAR19 therapy include prolonged cytopenias and associated infections. To better understand the impact of CAR19 on such toxicities, we studied a cohort of LBCL patients achieving durable remissions to assess immune recovery after CAR19 treatment.We first profiled bone marrow aspirates from patients requiring biopsy due to prolonged cytopenia and/or clinical concern for myeloid neoplasms. (S...
Source: Biology of Blood and Marrow Transplantation - February 1, 2024 Category: Hematology Authors: Mark P. Hamilton, Brian Sworder, Stefan Alig, Zinaida Good, Jan Boegeholz, Joseph Schroers-Martin, John Tamaresis, Mohammad Esfahani, Ying Lu, Mari Olsen, Chih Long Liu, Zachary Ehlinger, Moksha Desai, Felicia Liu-Fei, Lori S Muffly, Robert S. Negrin, Sal Tags: 5 Source Type: research

Plasma Proteome Analyses Identify Predictors of Graft Rejection Prior to Stem Cell Infusion and Support Interferon-Mediated Ferroptosis As a Novel Mechanism of Graft Rejection
No established biomarkers or effective interventions exist for graft rejection. We previously reported that CXCL9, a downstream marker of interferon, differentiates graft rejection from other complications but is only useful at the time of rejection. We hypothesized that biological differences in the host proteome exist prior to HSCT and that interferon-mediated pathways drive host elimination of donor cells during graft rejection. (Source: Biology of Blood and Marrow Transplantation)
Source: Biology of Blood and Marrow Transplantation - February 1, 2024 Category: Hematology Authors: Dr. Anthony Sabulski, Lucille Langenberg, Nathan Luebbering, Towia A Libermann, Simon T Dillon, Jane Koo, Kasiani C Myers, Sonata Jodele, Stella M. Davies Tags: 6 Source Type: research