Kooperativer Bibliotheksverbund

In: BMC Medicine, Springer Science and Business Media LLC, Vol. 18, No. 1 ( 2020-12)

Kurzfassung: Silent cerebral infarcts (SCIs) are the most common neurological complication in children and adults with sickle cell disease (SCD). In this systematic review, we provide an overview of studies that have detected SCIs in patients with SCD by cerebral magnetic resonance imaging (MRI). We focus on the frequency of SCIs, the risk factors involved in their development and their clinical consequences. Methods The databases of Embase, MEDLINE ALL via Ovid, Web of Science Core Collection, Cochrane Central Register of Trials via Wiley and Google Scholar were searched from inception to June 1, 2019. Results The search yielded 651 results of which 69 studies met the eligibility criteria. The prevalence of SCIs in patients with SCD ranges from 5.6 to 80.6% with most studies reported in the 20 to 50% range. The pooled prevalence of SCIs in HbSS and HbSβ 0 SCD patients is 29.5%. SCIs occur more often in patients with the HbSS and HbSβ 0 genotype in comparison with other SCD genotypes, as SCIs are found in 9.2% of HbSC and HbSβ + patients. Control subjects showed a mean pooled prevalence of SCIs of 9.8%. Data from included studies showed a statistically significant association between increasing mean age of the study population and mean SCI prevalence. Thirty-three studies examined the risk factors for SCIs. The majority of the risk factors show no clear association with prevalence, since more or less equal numbers of studies give evidence for and against the causal association. Conclusions This systematic review and meta-analysis shows SCIs are common in patients with SCD. No clear risk factors for their development were identified. Larger, prospective and controlled clinical, neuropsychological and neuroimaging studies are needed to understand how SCD and SCIs affect cognition.

Materialart: Online-Ressource

ISSN: 1741-7015

URL: Article

DOI: 10.1186/s12916-020-01864-8

Sprache: Englisch

Verlag: Springer Science and Business Media LLC

Publikationsdatum: 2020

ZDB Id: 2131669-7

In: American Journal of Hematology, Wiley, Vol. 94, No. 5 ( 2019-05), p. 575-584

Kurzfassung: In sickle cell disease (SCD), sickle hemoglobin (HbS) polymerizes upon deoxygenation, resulting in sickling of red blood cells (RBCs). These sickled RBCs have strongly reduced deformability, leading to vaso‐occlusive crises and chronic hemolytic anemia. To date, there are no reliable laboratory parameters or assays capable of predicting disease severity or monitoring treatment effects. We here report on the oxygenscan, a newly developed method to measure RBC deformability (expressed as Elongation Index ‐ EI) as a function of pO 2 . Upon a standardized, 22 minute, automated cycle of deoxygenation (pO 2 median 16 mmHg ± 0.17) and reoxygenation, a number of clinically relevant parameters are produced in a highly reproducible manner (coefficients of variation 〈 5%). In particular, physiological modulators of oxygen affinity, such as, pH and 2,3‐diphosphoglycerate showed a significant correlation (respectively R = ‑0.993 and R = 0.980) with Point of Sickling (PoS 5% ), which is defined as the pO 2 where a 5% decrease in EI is observed during deoxygenation. Furthermore, in vitro treatment with antisickling agents, including GBT440, which alter the oxygen affinity of hemoglobin, caused a reproducible left‐shift of the PoS, indicating improved deformability at lower oxygen tensions. When RBCs from 21 SCD patients were analyzed, we observed a significantly higher PoS in untreated homozygous SCD patients compared to treated patients and other genotypes. We conclude that the oxygenscan is a state‐of‐the‐art technique that allows for rapid analysis of sickling behavior in SCD patients. The method is promising for personalized treatment, development of new treatment strategies and could have potential in prediction of complications.

Materialart: Online-Ressource

ISSN: 0361-8609 , 1096-8652

URL: Issue

URL: Article

DOI: 10.1002/ajh.v94.5

DOI: 10.1002/ajh.25443

Sprache: Englisch

Verlag: Wiley

Publikationsdatum: 2019

ZDB Id: 1492749-4

In: Pediatric Blood & Cancer, Wiley, Vol. 69, No. 1 ( 2022-01)

Kurzfassung: Little is known about health‐related quality of life (HRQoL) in young children with sickle cell disease living in a European country. Methods A retrospective cross‐sectional evaluation of TNO‐AZL Preschool Children Quality of Life questionnaire (TAPQOL, 0–1 year) and Pediatric Quality of Life Inventory (PedsQL, 2–7 years) data was conducted. Study participants included caregivers of children with sickle cell disease aged 0–7 years attending the sickle cell centre at the Erasmus Medical Center or the Amsterdam University Medical Centers between April 2012 and October 2020. Comparisons were made with normative data on HRQoL in the general paediatric population. Results The study enrolled 136 caregivers of 136 children. In children aged 0–5 years, no significant differences emerged between children with sickle cell disease and the general population. However, in children aged 5–7 years, children with sickle cell disease scored significantly lower on all subscales except for emotional functioning. Multiple regression models showed a negative association between age and HRQoL. No association was found between HRQoL and disease severity or sociodemographic characteristics. Conclusions This study demonstrates that HRQoL is negatively correlated with age in young children with sickle cell disease with a significantly lower HRQoL in 5‐ to 7‐year‐olds when compared to the general population. Our study underlines the importance of measuring HRQoL in young children to identify patients with impaired HRQoL early in life in order to be able to intervene accordingly. Future research should focus on deepening the knowledge of factors influencing HRQoL in children with sickle cell disease.

Materialart: Online-Ressource

ISSN: 1545-5009 , 1545-5017

URL: Issue

URL: Article

DOI: 10.1002/pbc.v69.1

DOI: 10.1002/pbc.29358

Sprache: Englisch

Verlag: Wiley

Publikationsdatum: 2022

ZDB Id: 2130978-4

In: American Journal of Hematology, Wiley, Vol. 96, No. 1 ( 2021-01)

Materialart: Online-Ressource

ISSN: 0361-8609 , 1096-8652

URL: Issue

URL: Article

DOI: 10.1002/ajh.v96.1

DOI: 10.1002/ajh.26031

Sprache: Englisch

Verlag: Wiley

Publikationsdatum: 2021

ZDB Id: 1492749-4

In: Haemophilia, Wiley, Vol. 28, No. 2 ( 2022-03), p. 197-214

Kurzfassung: Currently, it is unknown which patient‐reported outcomes are important for patients with autosomal inherited bleeding disorders. Therefore, the purpose of this study is to systematically review the available literature assessing patient‐reported outcomes and their measurement methods in autosomal inherited bleeding disorders. Methods The Embase, Medline ALL, Web of Science Core Collection, Cochrane Central Register of Controlled Trails and Google Scholar databases were searched from inception until 14 August 2020. Studies on patient‐reported outcomes in patients with von Willebrand disease, inherited platelet function disorders and coagulation factor deficiencies were included. Results Twenty‐one articles met the inclusion criteria. Three studies were assessed as having poor quality, and therefore a high risk of bias. Nineteen studies had fair quality rating. Different measurements methods were used, ranging from predefined to self‐developed questionnaires. The majority of included studies focused on von Willebrand disease. Patients with von Willebrand disease reported lower health‐related quality of life compared to the general population. Overall, this trend was especially visible in the following domains: vitality, physical and social functioning and pain. Women with inherited bleeding disorders scored lower on health‐related quality of life compared to men, especially women with heavy menstrual bleeding. Patients with joint bleeds or heavy menstrual bleeding reported an increased level of pain. Conclusion Patients with autosomal inherited bleeding disorders report lower health related quality of life, especially those with joint bleeds or heavy menstrual bleeding. Numerous measurement methods are used in patients with autosomal inherited bleeding disorders, highlighting the need for studies using established, standardized measurement methods.

Materialart: Online-Ressource

ISSN: 1351-8216 , 1365-2516

URL: Issue

URL: Article

DOI: 10.1111/hae.v28.2

DOI: 10.1111/hae.14492

Sprache: Englisch

Verlag: Wiley

Publikationsdatum: 2022

ZDB Id: 2006344-1

In: Blood, American Society of Hematology, Vol. 134, No. Supplement_1 ( 2019-11-13), p. 4841-4841

Kurzfassung: Introduction Sickle cell disease (SCD) is a hereditary red blood cell disorder characterized by severe anemia, acute and painful vaso-occlusive crises and progressive organ failure. Success of health management of children with SCD is highly contingent on caregivers' capabilities. Caregivers of SCD affected children must perform a variety of tasks including communication with healthcare providers, reading and understanding of health information, interpretation of acute symptoms and administration of medication and complex decision making with regard to treatment options. A construct which describes the knowledge and competences of persons to meet the complex demands of health is 'health literacy' (HL) (1, 2). The measurement and assessment of HL is of growing importance due to expected and reported relationships between inadequate HL and health outcomes (3-5). In addition, caregivers with higher HL levels feel more confident in their ability to perform caregiving tasks, e.g. have a higher self-efficacy, often associated with higher quality of life (6, 7). Information on literacy levels and the relationship between HL, disease knowledge and self-efficacy may guide interventions in comprehensive SCD care. The aims of this study were to: (a) gain insight into levels of HL in caregivers of children with SCD using objective and subjective measures and to (b) assess the relationship between HL, caregivers self-efficacy' in communication with healthcare professionals and knowledge of SCD on different topics related to the illness. This abstract reports preliminary results. Methods In this cross-sectional, observational study, we included caregivers of children with SCD who attended the outpatient clinic of the Sickle Cell Comprehensive Care Center in the Erasmus Medical Center for a routine follow-up visit. Caregivers included in the study had to be able to communicate verbally in Dutch with professionals. HL was measured using the Short Assessment of Health Literacy in Dutch (SAHL-D) (8, 9); self-reported HL was evaluated by the Set of Brief Screening Questions (SBSQ) (10, 11). Self-efficacy was measured using the Perceived Self-Efficacy in Caregiver-Patient Interactions (PECPI) scale (12). Knowledge of SCD was assessed using a structured 13-item questionnaire (SCD-K) based on information and education provided in our clinic. Since data were not-normally distributed, they were analyzed using non-parametric statistics. Results To date, a total of 42 caregivers were included. Study inclusion will continue until at least 75 caregivers have been included. Demographics are presented in Table 1. Caregivers were mainly the child's mother (81.0%) often the single head of household (66.7%). The mean age of caregivers was 34.4 years. Educational level ranged from never attended school to post college Almost a quarter (23.8%) of caregivers was born in the Netherlands, others the rest were non-western migrants (76.2%). 78.6% of caregivers had low HL according to the SAHL-D. Caregivers with lower HL were more likely to have lower education (p=0.012) and to have been born outside the Netherlands (p=0.002). Only four caregivers (9.5%) reported having difficulties in understanding and applying health information (measured by SBSQ). The correlation between the SAHL-D and the SBSQ scores was weak (r=0.39). Mean scores on the SBSQ and PECPI were high, indicating that caregivers perceived their abilities for self-efficacy and their ability to read and understand medical information as quite high. Responses to individual SCD-K items however suggest large knowledge deficits: only 64.3% of caregivers knew which temperature is considered as fever [ 〉 38.0 °C or 〉 38.5 °C] and only 14.3% was aware which risk factors are able to provoke sickle cell crises. The relationship between literacy status and item responses on SCD-K assessment scale was also examined. Caregivers with low literacy scored significantly lower on almost every individual item. Discussion Inadequate HL is highly prevalent in caregivers of children with SCD. Not being born in the Netherlands and lower education levels are strong predictors of low HL. Our study suggests that healthcare professionals should be attentive to possible low HL. In addition, these results underline that health information should be tailored to the HL skills and specific context of patients and their families. Disclosures Cnossen: NWO: Other: Governmental grants , ZonMW, Innovation fund and Nationale Wetenschapsagenda 2018; Roche: Other: Travel Grants; Takeda: Other: Travel Grants, Research Funding; Shire: Other: Travel Grants, Research Funding; Baxter: Other: Travel Grants, Research Funding; Sobi: Research Funding; CSL Behring: Other: Travel Grants, Research Funding; Nordic Pharma: Research Funding; Novo Nordisk: Research Funding; Pfizer: Other: Travel Grants, Research Funding; Bayer: Other: Travel Grants, Research Funding.

Materialart: Online-Ressource

ISSN: 0006-4971 , 1528-0020

URL: Article

DOI: 10.1182/blood-2019-129888

Sprache: Englisch

Verlag: American Society of Hematology

Publikationsdatum: 2019

ZDB Id: 1468538-3

ZDB Id: 80069-7

In: International Journal of Laboratory Hematology, Wiley

Materialart: Online-Ressource

ISSN: 1751-5521 , 1751-553X

URL: Article

DOI: 10.1111/ijlh.14208

Sprache: Englisch

Verlag: Wiley

Publikationsdatum: 2023

ZDB Id: 2268600-9

In: Pediatric Blood & Cancer, Wiley, Vol. 67, No. 9 ( 2020-09)

Kurzfassung: While multiple studies have examined the cost of health care for one aspect of sickle cell disease care, few have focussed on the overall cost of comprehensive care for sickle cell disease. Methods We conducted a retrospective cohort study of children with sickle cell disease treated in a comprehensive care centre from 1 January 2015 to 31 December 2016. Health care utilisation of included patients was based upon data from two main sources. The clinical practice guideline was used to determine the expected resource use of routine comprehensive care (planned elective care), and the financial claims database was used to estimate real‐world resource use associated with acute and inpatient care (additional care). Results A total of 125 children with sickle cell disease were analysed. Expenditures for these patients averaged €5049 [standard deviation (SD) €1634] per child per year. Total yearly costs per patient varied considerably, ranging from €669 to €84 010, and less than 15% of patients were responsible for 50% of the health care costs. The majority (37%) of costs was associated with inpatient hospital care, which increased by age group, 27% with diagnostics, 19% with treatment, 11% with outpatients’ visits and 6% with emergency care. Conclusion We have described real‐world resource use and expenditures for children with sickle cell disease in a European comprehensive care centre. It seems that costs of a comprehensive approach with effective management in the outpatient setting is favourable when compared to episodic health care.

Materialart: Online-Ressource

ISSN: 1545-5009 , 1545-5017

URL: Issue

URL: Article

DOI: 10.1002/pbc.v67.9

DOI: 10.1002/pbc.28588

Sprache: Englisch

Verlag: Wiley

Publikationsdatum: 2020

ZDB Id: 2130978-4

In: Blood, American Society of Hematology, Vol. 136, No. Supplement 1 ( 2020-11-5), p. 20-21

Kurzfassung: Background: In sickle cell disease (SCD), hemoglobin S (HbS) polymerizes upon deoxygenation, reducing red blood cell (RBC) deformability. RBC deformability can be measured over a gradient of oxygen tensions (pO2) with the Laser Optical Rotational Red Cell Analyzer (Lorrca) ektacytometer (RR Mechatronics). Oxygen gradient ektacytometry generates 3 key parameters: 1) EImax, RBC deformability at normoxia; 2) EImin, minimum RBC deformability upon deoxygenation; and 3) the point of sickling (PoS): the oxygen tension at which a 5% decrease in deformability is observed during deoxygenation, reflecting the patient-specific pO2 at which sickling begins (Figure 1A). Previously we showed that oxygen gradient ektacytometry-derived biomarkers correlate with measures of SCD disease severity and hemolytic rate (Rab et al. Blood 2018), and is associated with vaso-occlusive crisis (VOC) frequency (Rab et al, Blood 2019). In this study, we confirm these observations in 2 independent cohorts and extend it to occurrence of acute chest syndrome (ACS), stroke including silent cerebral infarction (SCI), and transcranial Doppler (TCD) outcome. Methods: We analyzed 2 cohorts of SCD patients; an adult patient cohort of 53 SCD patients, enrolled at either University Medical Center Utrecht, The Netherlands (UMCU, n=25) or Hospital Lyon France (LIBM, n=28), and a pediatric patient cohort of 190 SCD patients enrolled at Texas Children's Hospital, USA (TCH). Subjects were HbSS or HbS/β-thalassemia, with a substantial number of subjects on hydroxyurea (HU) therapy (adult cohort 66% and pediatric cohort 86%), and not on chronic transfusion therapy. Correlations between oxygen gradient ektacytometry-derived biomarkers and the clinical complications of stroke or silent infarcts (SCI), ACS, VOC were assessed in both pediatric and adult patients. Patient groups generally did not significantly differ significantly by age, gender or HU treatment in the adult cohort except for age, which was lower in the ACS+ group (25.3years (y) compared to 32.0y) and also lower in the VOC+ group (27.1y compared to 35.8y). In the pediatric cohort, patient groups differed significantly in the ACS+ group compared to the ACS- group by age (ACS- group 8.37, ACS+ group 10.9y) and HU treatment (ACS- group 76%, ACS+ group 93%). Similarly, age was significantly higher in the Stroke+ group compared to the Stroke- group (14.0y compared to 9.3y), which was also found when studying VOC (VOC+ group 11.6y, VOC- group 8.2y). Results: In the pediatric cohort, PoS was significantly higher in patients with ACS (mean 40.3 compared to 34.9 mmHg, p=0.0001, Figure 1B). In the adult cohort, PoS was also higher in those with ACS although this did not reach significance (p=0.053, Figure 1C). In the pediatric cohort, PoS was higher in patients with stroke or SI (mean 43.0 mmHg compared to mean 37.3 mmHg, p & lt;0.05), Figure 1D). This finding was replicated in the adult cohort (p & lt;0.05, Figure 1E). EImin and EImax in both cohorts were significantly lower in patients who experienced stroke or SCI (all p & lt;0.05). PoS was higher and EImin and EImax lower in subjects with elevated TCD measurements (all p & lt;0.01, Figure 1F and G). In the adult and pediatric patient cohort, PoS was higher in patients with recent VOC (both p & lt;0.05, Figure 1H and I). Differences in mean PoS between pediatric and adult patient cohorts could be due to differences in treatment, age, genetic background or technical differences between Lorrca devices. Lower significance levels found in the adult patient cohort could be due to smaller sample size. Conclusion: We show that oxygen gradient ektacytometry provides functional, clinically relevant next generation biomarkers that are associated with ACS, stroke and VOC. This study therefore further validates the clinical usefulness of these biomarkers, in particular in relation to cerebral vasculopathy. Since our results merely describe an association and not causality further validation is warranted to establish how well oxygen gradient ektacytometry can assess disease severity. However, its parameters could already provide the clinician with information about patient RBC characteristics and sickling propensity that could aid in clinical decision making. Our results provide a rationale for further development of these biomarkers in the evaluation of novel therapies as part of clinical care, or clinical trial endpoints. Disclosures Rab: RR Mechatronics: Research Funding. Bos:RR Mechatronics: Research Funding. Cnossen:Takeda: Research Funding; Shire: Research Funding; Baxter: Research Funding; Bayer: Research Funding; Sobi: Research Funding; CSL behring: Research Funding; Nordic Pharma: Research Funding; Novo Nordisk: Research Funding; Pfizer: Research Funding. Wijk:Agios Pharmaceuticals Inc.: Research Funding; RR mechatronics: Research Funding. Sheehan:Emmaus: Research Funding; Global Blood Therapeutics: Research Funding; Novartis: Research Funding. van Beers:Pfizer: Research Funding; RR mechatronics: Research Funding; Agios: Membership on an entity's Board of Directors or advisory committees, Research Funding; Novartis: Research Funding.

Materialart: Online-Ressource

ISSN: 0006-4971 , 1528-0020

URL: Article

DOI: 10.1182/blood-2020-135934

Sprache: Englisch

Verlag: American Society of Hematology

Publikationsdatum: 2020

ZDB Id: 1468538-3

ZDB Id: 80069-7

In: Blood, American Society of Hematology, Vol. 134, No. Supplement_1 ( 2019-11-13), p. 2275-2275

Kurzfassung: Background: In sickle cell disease (SCD), hemoglobin S (HbS) polymerizes upon deoxygenation, reducing red blood cell (RBC) deformability. RBC deformability can be measured over a range of oxygen concentrations using a Laser Optical Rotational Red Cell Analyzer (Lorrca) ektacytometer (RR Mechatronics, Zwaag, the Netherlands) with Oxygenscan module. The Oxygenscan measures 3 key parameters: 1) EImax, RBC deformability at normoxia; 2) EImin, RBC deformability upon deoxygenation; and 3) the point of sickling (PoS): the oxygen tension at which a 5% decrease in normoxic EI is observed during deoxygenation, reflecting the patient-specific pO2 at which sickling begins (Figure 1A). Previously we showed that Oxygenscan parameters correlate with measures of SCD disease severity and hemolysis (absolute reticulocyte count, %fetal hemoglobin, %HbS, total Hb, %dense RBC, (Rab et al. Blood 2018). In this study, we investigated the relationship between oxygenscan parameters and incidence of vaso-occlusive crisis (VOC), and we tested the ability of the Oxygenscan parameters to assess response to hydroxyurea (HU) and chronic transfusion (CTF) in patients with SCD. Methods: We analyzed 2 cohorts: a European cohort (EUC) of 62 SCD patients (all HbSS or HbS/β-thalassemia), enrolled at either University Medical Center Utrecht (UMCU, n= 42) or Hospital Lyon (LIBM, n=20), and a US cohort (USC) of 97 SCD patients enrolled at Texas Children's Hospital (TCH). Differences in Oxygenscan parameters in SCD patients without/with VOC (requiring a doctor's evaluation) in the past 2 years were measured in 46 adult EUC SCD patients and 80 pediatric USC SCD patients. EUC patients without VOC (n=18, median age 40.6 years (y); 11 female (F), 44% on HU, 6% on CTF, 28% on both treatments), were compared to patients with a positive history of VOC (n=28, median age 23.9y, 13F, 39% on HU, 11% on CTF and 11% on both). Patients without VOC in the USC (n= 34, median age 8.0y, 15F, 53% on HU, 15% on CTF and 21% on both treatments) were compared to patients with VOC (n=46, median age 11.8y, 20F, 74% on HU, 13% on CTF and 11% on both treatments). To establish treatment related Oxygenscan parameter changes, we analyzed RBCs from 9 SCD patients from UMCU (median age 19y, 5F), before and during HU treatment (measurements performed at baseline, and 1, 3 and 6 months after starting HU), 7 SCD patients from UMCU (median age 26.7y; 6F) before and after transfusion and 17 SCD patients from TCH (median age 10.8y; 6F) on HU before and after transfusion. Results: In the EUC, PoS differed significantly between patients without VOC in the last 2 years (median 41.6mmHg) and patients with VOC in the last 2 years (median 53.7 mmHg, p 〈 0.001, Figure 1B). In the USC, PoS was also lower in patients without VOC compared to those with a VOC in past 2 years (p 〈 0.05, Figure 1C). EImin in both cohorts was significantly lower in patients who experienced VOC (p 〈 0.05). EImax did not show a significant difference in both cohorts. Correlation of PoS with VOC episodes was significant in the EUC (r=0.447, p 〈 0.01, Figure 1D) and USC (r=0.228, p 〈 0.05), indicating that RBCs start to sickle at a higher pO2 in patients where VOC occur more often. Differences found in median PoS between EUC and USC could be due to differences in treatment (EUC 30% no treatment, USC 5% no treatment), difference in age (EUC median age 28.5y, USC median age 11.6y), genetic background or technical differences between Oxygenscan devices. Transfusion improved EImax, EImin, and PoS (USC: p 〈 0.001, p 〈 0.0001, and p 〈 0.01, EUC: all parameters p 〈 0.05 Figure 1E). HU treatment improved all parameters after 3 and 6 months compared to baseline (p 〈 0.05 and p 〈 0.0001 Figure 1F). Conclusion: Our results show that RBC from SCD patients without VOC in the last two years were able to tolerate lower oxygen concentrations before sickling (PoS). RBCs from patients without VOC were also more deformable when deoxygenated (EImin) compared to patients who had experienced one or more VOCs in the last two years. In contrast, RBC deformability, when oxygenated (EImax) was not different in patients with or without VOC in the last two years. All 3 Oxygenscan parameters significantly improved with standard of care SCD treatments, namely CTF and/or HU. We therefore conclude that the PoS, EImax and EImin are useful biomarkers of clinical severity and treatment response, and may be essential in monitoring novel SCD treatments as part of a clinical trial as a surrogate endpoint. Disclosures Rab: RR Mechatronics: Research Funding. van Wijk:Agios Pharmaceuticals: Consultancy, Research Funding; RR Mechatronics: Research Funding. Bos:RR Mechatronics: Research Funding. Nur:Novartis Pharmaceuticals: Consultancy. Cnossen:NWO: Other: Governmental grants , ZonMW, Innovation fund and Nationale Wetenschapsagenda 2018; Roche: Other: Travel Grants; Takeda: Other: Travel Grants, Research Funding; Shire: Other: Travel Grants, Research Funding; Baxter: Other: Travel Grants, Research Funding; Sobi: Research Funding; CSL Behring: Other: Travel Grants, Research Funding; Nordic Pharma: Research Funding; Novo Nordisk: Research Funding; Bayer: Other: Travel Grants, Research Funding; Pfizer: Other: Travel Grants, Research Funding. van Beers:Agios Pharmaceuticals, Inc.: Membership on an entity's Board of Directors or advisory committees, Research Funding; Novartis: Consultancy, Research Funding; Pfizer: Research Funding; RR Mechatronics: Research Funding.

Materialart: Online-Ressource

ISSN: 0006-4971 , 1528-0020

URL: Article

DOI: 10.1182/blood-2019-128870

Sprache: Englisch

Verlag: American Society of Hematology

Publikationsdatum: 2019

ZDB Id: 1468538-3

ZDB Id: 80069-7