A5053532854- Hematopoietic Stem Cell Transplantation
- Hemoglobinopathies and Related Disorders
- Blood groups and transfusion
- Blood disorders and treatments
- Immunodeficiency and Autoimmune Disorders
- Platelet Disorders and Treatments
- Prenatal Screening and Diagnostics
- Acute Myeloid Leukemia Research
- Acute Lymphoblastic Leukemia research
- Polyomavirus and related diseases
- Erythrocyte Function and Pathophysiology
- Blood Coagulation and Thrombosis Mechanisms
- Immune Cell Function and Interaction
- Hemophilia Treatment and Research
- Iron Metabolism and Disorders
- DNA Repair Mechanisms
- RNA modifications and cancer
- Cancer-related gene regulation
- Ethics and Legal Issues in Pediatric Healthcare
- Myeloproliferative Neoplasms: Diagnosis and Treatment
- Cytomegalovirus and herpesvirus research
- Venous Thromboembolism Diagnosis and Management
- Chronic Lymphocytic Leukemia Research
- Cardiovascular Issues in Pregnancy
- Virus-based gene therapy research
Leiden University Medical Center
2016-2025
Leiden University
2013-2023
Loyola University Medical Center
2016
Willem-Alexander Kinderziekenhuis
2014
Haga Hospital
2009
Sanquin
2009
Erasmus MC - Sophia Children’s Hospital
1995-1996
Thalassemia major and sickle cell disease are the two most widely disseminated hereditary hemoglobinopathies in world. The outlook for affected individuals has improved recent years due to advances medical management prevention treatment of complications. However, hematopoietic stem transplantation is still only available curative option. use been increasing, outcomes today have substantially compared with past three decades. Current experience world-wide that more than 90% patients now...
The life expectancy of non-severe hemophilia A (HA) patients equals the non-hemophilic population. However, data on effect inhibitor development mortality and hemophilia-related causes death are scarce. neutralizing factor VIII antibodies in HA may dramatically change their clinical outcome due to severe bleeding complications.We assessed association between occurrence inhibitors with HA.In this retrospective cohort study, vital status were collected for 2709 (107 inhibitors) who treated...
Highlights•Autoimmune cytopenia after stem cell transplantation is a rare complication.•Nonmalignant disease, alemtuzumab use, and CMV reactivation are risk factors.•The cytokine profile of AIC patients appears to favor Th 2 response.•Rituximab, bortezomib, sirolimus promising step-up therapies.AbstractAutoimmune or alloimmune (AIC) known complication hematopoietic (SCT). SCT considered difficult treat associated with high morbidity mortality. In this retrospective study in pediatric we...
The bleeding phenotype of children with von Willebrand disease (VWD) needs to be characterized in detail facilitate diagnosis during childhood and aid the planning assessment treatment strategies. objective was evaluate occurrence, type, severity a large cohort moderate severe VWD. We included 113 (aged 0-16 years) Type 1 (n = 60), 2 44), 3 9) VWD factor (VWF) antigen and/or VWF ristocetin cofactor levels ≤ 30 U/dL from nation-wide cross-sectional study ("Willebrand Netherlands" study)....
Objective . To determine the incidence, management, complications, and outcome in neonates with thrombotic events. Study Design We performed a retrospective study of all events admitted to our neonatal intensive care unit from January 2004 July 2010. Results Thrombotic were identified 32 4734 (0.7%). Seven managed expectantly 25 received anticoagulant treatment. Complete resolution clot within 3 months age was found 68% (17/25) treated 86% (6/7) nontreated neonates. Major complications due...
Summary Treosulfan‐based conditioning is increasingly employed in paediatric haematopoietic stem cell transplantation ( HSCT ). Data on treosulfan pharmacokinetics children are scarce, and the relationship between exposure, toxicity clinical outcome unresolved. In this multicentre prospective observational study, we studied drug's with regimen‐related early 77 patients. Treosulfan dose was 30 g/m 2 , administered over 3 consecutive days infants <1 year old n = 12) 42 ≥1 65). Mean day 1...
Background: High-dose treosulfan is used in conditioning regimens before hematopoietic stem cell transplantation children. Pharmacokinetic data to optimize dosing are scarce this patient population. The aims of study were the development and validation an analytical method for human serum a pharmacokinetic model pediatric patients. Furthermore, we aimed develop limited sampling strategy estimate systemic exposure with minimum inconvenience risk patient. Methods: A reversed phase...
Summary In nonsevere haemophilia A (HA) patients the presence of an inhibitor may exacerbate bleeding phenotype dramatically. There are very limited data on optimal therapeutic approach to eradicate inhibitors in these patients. We aimed describe eradication treatment a large cohort unselected HA with inhibitors. included 101 from source population 2,709 (factor VIII 2–40 IU/dl), treated Europe and Australia (median age 37 years, interquartile range (IQR) 15–60; median peak titre 7 BU/ml,...
Cidofovir is frequently used to treat life-threatening human adenovirus (HAdV) infections in immunocompromised children after hematopoietic stem cell transplantation (HSCT). However, the antiviral effect irrespective of T reconstitution remains unresolved. Plasma HAdV DNA levels were monitored by real-time quantitative PCR during 42 cidofovir treatment episodes for viremia 36 pediatric allogeneic HSCT recipients. load dynamics related and natural killer (NK) measured flow cytometry. To...
Summary This article presents the haematopoietic stem cell transplantation ( SCT ) results of complete Dutch Fanconi anaemia FA patient cohort. Sixty‐eight patients have been transplanted since 1972. In total, 63 (93%) engrafted, 54 after first and 9 second . Fludarabine (FLU)‐based conditioning was associated with decreased graft failure (odds ratio 0·21, P = 0·01), early mortality (hazard 0·25, 0·01) improved 5‐year overall survival (FLU 87·8% [standard error SE 5·1%] versus non‐FLU 59·3%...
Treosulfan-based conditioning has gained popularity in pediatric allogeneic hematopoietic stem cell transplantation (HSCT) because of its presumed favorable efficacy and toxicity profile. Treosulfan is used standardized dosing regimens based on body surface area. The relationships between systemic treosulfan exposure early long-term clinical outcomes patients undergoing HSCT for nonmalignant diseases remain unclear. In this a multicenter, prospective observational study, we assessed the...