A5073892799- Hemophilia Treatment and Research
- Platelet Disorders and Treatments
- Hematopoietic Stem Cell Transplantation
- Blood Coagulation and Thrombosis Mechanisms
- Immune Cell Function and Interaction
- Virus-based gene therapy research
- Blood disorders and treatments
- Blood groups and transfusion
- Chronic Myeloid Leukemia Treatments
- CAR-T cell therapy research
- Acute Myeloid Leukemia Research
- Coagulation, Bradykinin, Polyphosphates, and Angioedema
- Chronic Lymphocytic Leukemia Research
- Myeloproliferative Neoplasms: Diagnosis and Treatment
- Cancer-related gene regulation
- Acute Lymphoblastic Leukemia research
- Heparin-Induced Thrombocytopenia and Thrombosis
- Glycosylation and Glycoproteins Research
- Lymphoma Diagnosis and Treatment
- Parvovirus B19 Infection Studies
- Monoclonal and Polyclonal Antibodies Research
- Mesenchymal stem cell research
- Biomedical and Engineering Education
- Lymphatic System and Diseases
- Ethics and Legal Issues in Pediatric Healthcare
Cornell University
2016-2025
Weill Cornell Medicine
2016-2025
Indiana Hemophilia and Thrombosis Center
2024
Presbyterian Hospital
2013-2023
Columbia University
2007-2019
New York Hospital Queens
2013-2015
NewYork–Presbyterian Hospital
2013-2015
Columbia University Irving Medical Center
2011
Morgan Stanley Children's Hospital
2008-2010
The prevention of bleeding with adequately sustained levels clotting factor, after a single therapeutic intervention and without the need for further medical intervention, represents an important goal in treatment hemophilia.
Congenital fibrinogen deficiency (CFD) is a rare bleeding disorder caused by mutations in FGA, FGB, and FGG. We sought to comprehensively characterize patients with CFD using PRO-RBDD (Prospective Rare Bleeding Disorders Database). Clinical phenotypes, laboratory, genetic features were investigated retrospective data from the PRO-RBDD. Patients classified asymptomatic grade 3 based on their severity. In addition, FGG sequenced find causative variants. A total of 166 cases 16 countries...
The North American Pediatric Aplastic Anemia Consortium (NAPAAC) is a group of pediatric hematologist-oncologists, hematopathologists, and bone marrow transplant physicians from 46 institutions in America with interest expertise aplastic anemia, inherited failure syndromes, myelodysplastic syndromes. NAPAAC Bone Marrow Failure Diagnosis Care Guidelines Working Group was established the charge harmonizing approach to diagnostic workup anemia an effort standardize best practices field. This...
Abstract Severe aplastic anemia (SAA) is a rare potentially fatal hematologic disorder. Although overall outcomes with treatment are excellent, there variations in management approach, including differences between adult and pediatric patients. Certain aspects of under active investigation clinical trials. Because the rarity disease, some hematologists may have relatively limited experience complex SAA. The following recommendations reflect an up‐to‐date evidence‐based approach to children...
Preventing factor VIII (FVIII) inhibitors following replacement therapies with FVIII products in patients hemophilia A remains an unmet medical need. Better understanding of the early events evolving is essential for risk identification and design novel strategies to prevent inhibitor development. The Hemophilia Inhibitor Previously Untreated Patients (PUPs) Study (HIPS; www.clinicaltrials.gov #NCT01652027) first prospective cohort study evaluate comprehensive changes immune system during 50...
Treatment with fidanacogene elaparvovec, a recombinant adeno-associated virus (AAV) vector developed for the treatment of hemophilia B, led to sustained expression high-activity factor IX variant (FIX-R338L, or FIX-Padua) in phase 1-2a study. The long-term safety and efficacy this are not known. In 12-month study, 15 participants severe moderately B (factor coagulant activity, ≤2% normal value) received elaparvovec at dose 5×1011 genomes (vg) per kilogram body weight; thereafter, could...
Abstract PCDH10 has been implicated as a tumor suppressor, since epigenetic alterations of this gene have noted in multiple types. However, to date, studies regarding its role acute and chronic leukemias are lacking. Here, we investigated the presence promoter hypermethylation two CpG islands by methylation‐specific PCR 215 cases various subsets myeloid‐ lymphoid‐lineage leukemias. We found that was frequent both B‐cell (81.9%) T‐cell (80%) lymphoblastic leukemia (ALL), while it present low...
Previous studies reported the efficacy and safety profile of extended half-life PEGylated recombinant factor VIII (FVIII) rurioctocog alfa pegol (TAK-660, SHP660, BAX 855) in preventing bleeding haemophilia A patients.This study evaluated long-term for prophylaxis treatment previously treated children adults.In this phase 3b, prospective, open-label, multicentre (NCT01945593), eligible patients ≤ 75 years with severe (FVIII < 1%) received prophylactic a fixed dose (FD, twice-weekly or less...
Background Cyclophosphamide‐based conditioning regimens and allogeneic hematopoietic stem cell transplantation (AlloHSCT) from matched related donors (MRD) has resulted in the highest survival rates children adolescents with acquired severe aplastic anemia (SAA). Time to transplant consistently been associated decreased overall survival. Reduced toxicity AlloHSCT used successfully other pediatric non‐malignant diseases. Procedure We piloted a risk‐adapted approach, using fludarabine...
The B-Natural study is a multicentre, multinational, observational of haemophilia B (HB) designed to increase understanding clinical manifestations, treatment and quality life (QoL). To characterise compare QoL in HB across disease severity groups individuals with inhibitors identify gaps treatment. A total 224 from 107 families were enrolled 24 centres North America (n = 16), Europe 7) Asia 1). Of these, 68 (30.4%) subjects had severe (<1 IU/dL), median age 15.6 years, 114 (50.9%) moderate...
Abstract Severe aplastic anemia (SAA) is a rare potentially fatal hematologic disorder. Although overall outcomes with treatment are excellent, there variations in management approach, including differences between adult and pediatric patients. Certain aspects of under active investigation clinical trials. Because the rarity disease, some hematologists may have relatively limited experience complex SAA. The following recommendations reflect an up‐to‐date evidence‐based approach to children...
Unrelated cord blood (CB) is an excellent alternative as allogeneic donor source for stem cell transplantation. CB transplantation associated with lower incidence of severe acute graft versus host disease (GVHD) and chronic GVHD but similar rates malignant relapse compared other unrelated transplants. NK cells are critical innate immune components the comparison vs. peripheral (PB) relatively unknown. receptor expression, function, maturation may play a role in risk after transplant. We...
Haemophilia B (HB) is less well studied than haemophilia A (HA); despite similarities between the two inherited bleeding disorders, important differences remain that require further research.B-Natural a multi-centre, prospective, observational study of HB, designed to increase understanding clinical manifestations, treatment, quality-of-life (QoL), inhibitor development, immune tolerance induction (ITI) outcome, renal function and create biorepository for future investigations.Participants...