Cnossen Marjon H, van Moort Iris, Reitsma Simone H, de Maat Moniek P M, Schutgens Roger E G, Urbanus Rolf T, Lingsma Hester F, Mathot Ron A A, Gouw Samantha C, Meijer Karina, Bredenoord Annelien L, van der Graaf Rieke, Fijnvandraat Karin, Meijer Alexander B, van den Akker Emile, Bierings Ruben, Eikenboom Jeroen C J, van den Biggelaar Maartje, de Haas Masja, Voorberg Jan, Leebeek Frank W G
Department of Pediatric Hematology and Oncology, Erasmus University Medical Center, Rotterdam, The Netherlands.
Department of Hematology, Erasmus University Medical Center, Rotterdam, The Netherlands.
J Thromb Haemost. 2022 Jun 2;20(9):2001-11. doi: 10.1111/jth.15778.
Treatment choices for individual patients with an inborn bleeding disorder are increasingly challenging due to increasing options and rising costs for society. We have initiated an integrated interdisciplinary national research programme.
The SYMPHONY consortium strives to orchestrate personalized treatment in patients with an inborn bleeding disorder, by unravelling the mechanisms behind inter-individual variations of bleeding phenotype.
The SYMPHONY consortium will investigate patients with an inborn bleeding disorder, both diagnosed and not yet diagnosed.
Research questions are categorized under the themes: 1) Diagnosis; 2) Treatment; and 3) Fundamental research and consist of workpackages addressing specific domains. Importantly, collaborations between patients and talented researchers from different areas of expertise promise to augment the impact of the SYMPHONY consortium, leading to unique interactions and intellectual property.
SYMPHONY will perform research on all aspects of care, treatment individualization in patients with inborn bleeding disorders as well as diagnostic innovations and results of molecular genetics and cellular model technology with regard to the hemostatic process. We believe that these research investments will lead to health care innovations with long-term clinical and societal impact. This consortium has been made possible by a governmental, competitive grant from the Netherlands Organization for Scientific Research (NWO) within the framework of the NWA-ORC Call grant agreement NWA.1160.18.038.
由于治疗选择增多以及社会成本上升,为患有先天性出血性疾病的个体患者选择治疗方案变得越来越具有挑战性。我们启动了一项综合性跨学科国家研究计划。
SYMPHONY联盟致力于通过揭示出血表型个体间差异背后的机制,为患有先天性出血性疾病的患者精心制定个性化治疗方案。
SYMPHONY联盟将对患有先天性出血性疾病的患者进行研究,包括已确诊和尚未确诊的患者。
研究问题分为以下主题:1)诊断;2)治疗;3)基础研究,由涉及特定领域的工作包组成。重要的是,患者与来自不同专业领域的优秀研究人员之间的合作有望增强SYMPHONY联盟的影响力,从而产生独特的互动和知识产权。
SYMPHONY将对护理的各个方面、先天性出血性疾病患者的治疗个体化以及止血过程的分子遗传学和细胞模型技术的诊断创新及结果进行研究。我们相信,这些研究投入将带来具有长期临床和社会影响的医疗保健创新。该联盟是在荷兰科学研究组织(NWO)的政府竞争性拨款支持下,在NWA-ORC呼吁拨款协议NWA.1160.18.038的框架内成立的。