Jeremy D. Rubinstein
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View article: A real-world multi-institution experience of standard vs alternative dosing of inotuzumab ozogamicin for Relapsed/Refractory pediatric B-cell acute lymphoblastic leukemia
A real-world multi-institution experience of standard vs alternative dosing of inotuzumab ozogamicin for Relapsed/Refractory pediatric B-cell acute lymphoblastic leukemia Open
Background: Inotuzumab ozogamicin (InO) is an active agent in children and adults with relapsed/refractory (R/R) B-cell acute lymphoblastic leukemia (B-ALL). The FDA-approved dosing is 1.8 mg/m2/cycle fractionated into 3 doses, although re…
View article: Outcomes with Third Party Virus Specific T-cells After the Use of Single Antigen Cell Lines to Predict HLA Restriction
Outcomes with Third Party Virus Specific T-cells After the Use of Single Antigen Cell Lines to Predict HLA Restriction Open
Patients with significant T-cell dysfunction from chemotherapy or hematopoietic stem cell transplant are at significant risk for complications of viral infections. Off-the-shelf third-party virus specific T-cells (TP VSTs) are an effective…
View article: BK polyomavirus genotype IV isolates from virus-specific T-cell therapy recipients
BK polyomavirus genotype IV isolates from virus-specific T-cell therapy recipients Open
BK polyomavirus (BKPyV) can cause disease in transplant recipients. Virus-specific T-cell (VST) therapy is effective in decreasing virus levels in many individuals. BKPyV genetic variation can impact replication kinetics and immune respons…
View article: Project EVOLVE: an international analysis of postimmunotherapy lineage switch, an emergent form of relapse in leukemia
Project EVOLVE: an international analysis of postimmunotherapy lineage switch, an emergent form of relapse in leukemia Open
Lineage switch (LS), defined as the immunophenotypic transformation of acute leukemia, has emerged as a mechanism of relapse after antigen-targeted immunotherapy, which is associated with dismal outcomes. Through an international collabora…
View article: Posttransplant EBV‐Positive Smooth Muscle Tumors in Children, Adolescents, and Young Adults: A Multi‐Institution Experience
Posttransplant EBV‐Positive Smooth Muscle Tumors in Children, Adolescents, and Young Adults: A Multi‐Institution Experience Open
Epstein–Barr virus (EBV)‐positive smooth muscle tumors (SMTs) are rare tumors seen in immunocompromised patients. There is no clear standard of care for the management and treatment of EBV‐SMTs. Patients are often treated with chemotherapy…
View article: WU Hoo! The Promise of Allogeneic WU-CART-007 in Relapsed/Refractory T-Cell Malignancies
WU Hoo! The Promise of Allogeneic WU-CART-007 in Relapsed/Refractory T-Cell Malignancies Open
View article: Clinical outcomes of hypomethylating agents and venetoclax in newly diagnosed unfit and relapsed/refractory paediatric, adolescent and young adult acute myeloid leukaemia patients
Clinical outcomes of hypomethylating agents and venetoclax in newly diagnosed unfit and relapsed/refractory paediatric, adolescent and young adult acute myeloid leukaemia patients Open
Summary Venetoclax (VEN) combined with hypomethylating agents (HMA) decitabine or azacitidine is used for adult acute myeloid leukaemia (AML), but its application in paediatric, adolescent and young adult (AYA) AML lacks prospective studie…
View article: Blinatumomab as maintenance therapy for pediatric acute B-lymphoblastic leukemia in the setting of asparaginase-associated pancreatitis
Blinatumomab as maintenance therapy for pediatric acute B-lymphoblastic leukemia in the setting of asparaginase-associated pancreatitis Open
Modern chemotherapy for pediatric acute B-lymphoblastic leukemia (B-ALL), including a maintenance phase on the backbone of oral antimetabolite administration, has resulted in a generally excellent prognosis for newly diagnosed disease. How…
View article: Virus-Specific T Cells for the Treatment of Systemic Infections Following Allogeneic Hematopoietic Cell and Solid Organ Transplantation
Virus-Specific T Cells for the Treatment of Systemic Infections Following Allogeneic Hematopoietic Cell and Solid Organ Transplantation Open
Viral infections are a major source of morbidity and mortality in the context of immune deficiency and immunosuppression following allogeneic hematopoietic cell (allo-HCT) and solid organ transplantation (SOT). The pharmacological treatmen…
View article: Inotuzumab ozogamicin in B-cell precursor acute lymphoblastic leukemia: efficacy, toxicity, and practical considerations
Inotuzumab ozogamicin in B-cell precursor acute lymphoblastic leukemia: efficacy, toxicity, and practical considerations Open
Inotuzumab ozogamicin (InO) is an antibody drug conjugate composed of a humanized monoclonal antibody targeting the cell surface receptor CD22 coupled to a cytotoxic calicheamicin payload via an acid labile linker. InO has shown significan…
View article: P561: REVUMENIB IN PATIENTS WITH ACUTE LEUKEMIAS: COMPASSIONATE USE PROGRAM EXPERIENCE
P561: REVUMENIB IN PATIENTS WITH ACUTE LEUKEMIAS: COMPASSIONATE USE PROGRAM EXPERIENCE Open
Background: Revumenib (REV; SNDX-5613), a menin inhibitor, is under investigation in patients (pts) with relapsed/refractory (R/R) acute leukemia harboring a KMT2A rearrangement (KMT2Ar) or NPM1 mutation. A phase 1/2 study, AUGMENT-101 (NC…
View article: Needle in a haystack or elephant in the room? Identifying germline predisposition syndromes in the setting of a new myeloid malignancy diagnosis
Needle in a haystack or elephant in the room? Identifying germline predisposition syndromes in the setting of a new myeloid malignancy diagnosis Open
View article: Post‐transplant lymphoproliferative disease in children, adolescents, and young adults
Post‐transplant lymphoproliferative disease in children, adolescents, and young adults Open
Post‐transplant lymphoproliferative disease (PTLD) remains a major complication of transplantation. PTLD is a rare entity and very heterogenous making consensus on diagnosis and treatment very challenging. The majority are Epstein‐Barr vir…
View article: Post CAR T-cell therapy outcomes and management in HSCT-naive patients: a single-center experience
Post CAR T-cell therapy outcomes and management in HSCT-naive patients: a single-center experience Open
Background Tisagenlecleucel (tisa-cel) is increasingly being used in hematopoietic stem cell transplantation (HSCT)-naive patients. Outcomes for HSCT patients following chimeric antigen receptor (CAR) T-cell therapy demonstrate low relapse…
View article: Third-Party and Patient-Specific Donor-Derived Virus-Specific T Cells Demonstrate Similar Efficacy and Safety for Management of Viral Infections after Hematopoietic Stem Cell Transplantation in Children and Young Adults
Third-Party and Patient-Specific Donor-Derived Virus-Specific T Cells Demonstrate Similar Efficacy and Safety for Management of Viral Infections after Hematopoietic Stem Cell Transplantation in Children and Young Adults Open
Infections with double-stranded DNA viruses are a common complication after hematopoietic stem cell transplantation (HSCT) and cause significant morbidity and mortality in the post-transplantation period. Both donor-derived (DD) and third-…
View article: Multicenter study of pediatric Epstein‐Barr virus–negative monomorphic post solid organ transplant lymphoproliferative disorders
Multicenter study of pediatric Epstein‐Barr virus–negative monomorphic post solid organ transplant lymphoproliferative disorders Open
Background Pediatric Epstein‐Barr virus–negative monomorphic post solid organ transplant lymphoproliferative disorder [EBV(−)M‐PTLD] comprises approximately 10% of M‐PTLD. No large multi‐institutional pediatric‐specific reports on treatmen…
View article: An exceptional case of durable remission achieved with reinfusion of CD19-directed CAR-T despite failure to induce B-cell aplasia and review of institutional experience with reinfusion of tisagenlecleucel
An exceptional case of durable remission achieved with reinfusion of CD19-directed CAR-T despite failure to induce B-cell aplasia and review of institutional experience with reinfusion of tisagenlecleucel Open
CD19-directed chimeric antigen receptor T lymphocytes (CAR-T) have led to durable remissions in children with refractory and/or multiply relapsed B-lymphoblastic leukemia. For those who relapse or lose B-cell aplasia post-CAR-T, the role o…
View article: Burkitt lymphoma after solid‐organ transplant: Treatment and outcomes in the paediatric <scp>PTLD</scp> collaborative
Burkitt lymphoma after solid‐organ transplant: Treatment and outcomes in the paediatric <span>PTLD</span> collaborative Open
Summary Burkitt lymphoma arising in paediatric post‐solid‐organ transplantation‐Burkitt lymphoma (PSOT‐BL) is a clinically aggressive malignancy and a rare form of post‐transplant lymphoproliferative disorder (PTLD). We evaluated 35 patien…
View article: Acute myeloid leukemia in <i>SRP54</i>‐mutated congenital neutropenia
Acute myeloid leukemia in <i>SRP54</i>‐mutated congenital neutropenia Open
SRP54 mutations have recently been implicated in congenital neutropenia (CN) and the in‐frame deletion, p.Thr117del, is the most common pathogenic mutation reported. The largest study of SRP54 ‐mutated CN to‐date followed 23 patients for a…
View article: Scheduled administration of virus-specific T cells for viral prophylaxis after pediatric allogeneic stem cell transplant
Scheduled administration of virus-specific T cells for viral prophylaxis after pediatric allogeneic stem cell transplant Open
Infections with double-stranded DNA viruses are a significant cause of morbidity and mortality in pediatric patients following allogeneic hematopoietic stem cell transplantation (HSCT). Virus-specific T-cell therapies (VSTs) have been show…
View article: Virus-specific T cells for adenovirus infection after stem cell transplantation are highly effective and class II HLA restricted
Virus-specific T cells for adenovirus infection after stem cell transplantation are highly effective and class II HLA restricted Open
Infection with adenoviruses is a common and significant complication in pediatric patients after allogeneic hematopoietic stem cell transplantation. Treatment options with traditional antivirals are limited by poor efficacy and significant…
View article: Virus-specific T-cell therapy to treat BK polyomavirus infection in bone marrow and solid organ transplant recipients.
Virus-specific T-cell therapy to treat BK polyomavirus infection in bone marrow and solid organ transplant recipients. Open
BK polyomavirus (BKPyV) infection is a major complication of hematopoietic stem cell transplant (HSCT) and solid organ transplant (SOT). Treatment options are limited, poorly effective, and have significant toxicities. Cellular therapy usi…
View article: Complement inhibition does not impair the clinical antiviral capabilities of virus-specific T-cell therapy
Complement inhibition does not impair the clinical antiviral capabilities of virus-specific T-cell therapy Open
Key Points The use of terminal complement blockade is compatible with virus-specific T-cell (VST) expansion and clinical effectiveness. VST and complement-blocking agent concurrent therapy may be safely used in patients with thrombotic mic…
View article: Endothelial injury, F-actin and vitamin-D binding protein after hematopoietic stem cell transplant and association with clinical outcomes
Endothelial injury, F-actin and vitamin-D binding protein after hematopoietic stem cell transplant and association with clinical outcomes Open
Endothelial injury after hematopoietic stem cell transplant is an important initiating factor for early transplant toxicities of thrombotic microangiopathy and acute graft versus host disease. We hypothesized that release of the angiopathi…
View article: Pre-Transplant Baseline Platelet Count and the Number of Peri-HSCT Transfusions Are Associated with an Increased Disease-Free Mortality Rate in a Large Pediatric Cohort
Pre-Transplant Baseline Platelet Count and the Number of Peri-HSCT Transfusions Are Associated with an Increased Disease-Free Mortality Rate in a Large Pediatric Cohort Open
IntroductionPlatelets play a key role in the innate immune system as a source of pro-inflammatory cytokines. Lower platelet counts are associated with higher mortality in hospitalized adults with a variety of diseases, and platelet count <…
View article: Characterization of Viral Epitopes and the HLA Restriction That Govern Anti-Adenoviral Response to Viral Specific T-Lymphocyte Therapy in a Pediatric Cohort
Characterization of Viral Epitopes and the HLA Restriction That Govern Anti-Adenoviral Response to Viral Specific T-Lymphocyte Therapy in a Pediatric Cohort Open
IntroductionAdenoviral (AdV) infections are common after HSCT and are a major cause of complications. Antiviral medications are frequently ineffective and toxic. At CCHMC, viral specific T cells (VST) from 3rd party donors are generated by…
View article: PCN369 WHAT IS THE REGULATORY AND HTA LANDSCAPE OF NON-SMALL CELL LUNG CANCER DRUGS ACROSS THE EU5 COUNTRIES?
PCN369 WHAT IS THE REGULATORY AND HTA LANDSCAPE OF NON-SMALL CELL LUNG CANCER DRUGS ACROSS THE EU5 COUNTRIES? Open
View article: Pediatric and Young Adult Vulvovaginal Graft-versus-Host Disease
Pediatric and Young Adult Vulvovaginal Graft-versus-Host Disease Open
Vulvovaginal graft-versus-host disease (GVHD) is an underdiagnosed and poorly recognized complication of hematopoietic stem cell transplantation (HSCT). Previous studies have reported findings restricted to predominantly adult populations.…
View article: CD40 Ligand Levels in Pediatric Cohorts Correlate with Baseline Platelet Count and Transplant-Associated Mortality but Not with Endothelial Injury
CD40 Ligand Levels in Pediatric Cohorts Correlate with Baseline Platelet Count and Transplant-Associated Mortality but Not with Endothelial Injury Open
IntroductionCD40 ligand (CD40L), primarily expressed on activated CD4+ T lymphocytes, is also secreted in soluble form by T cells and platelets. CD40L modulates the immune response, largely via binding to CD40 on B lymphocytes. CD40L is al…