Nikola Hagedorn

Charité - Universitätsmedizin Berlin

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Cornelia Eckert

Charité - Universitätsmedizin Berlin

Susanne Holzhauer

Charité - Universitätsmedizin Berlin

Arend von Stackelberg

Charité - Universitätsmedizin Berlin

André Schrauder

Kiel University

Gunnar Cario

University Hospital Schleswig-Holstein

Madlen Reschke

Charité - Universitätsmedizin Berlin

Gabriele Escherich

Universität Hamburg

Caspar D. Kühnöl

University Hospital in Halle

Christina Peters

St Anna Children's Hospital

Franziska Cuntz

Charité - Universitätsmedizin Berlin

Cooperative Institutions

Charité - Universitätsmedizin Berlin

Heidelberg University

University of Lübeck

Klinik und Poliklinik für Kinder- und Jugendmedizin

University Hospital Schleswig-Holstein

Humboldt-Universität zu Berlin

Universität Hamburg

Freie Universität Berlin

Goethe University Frankfurt

University Medical Center Hamburg-Eppendorf

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Monitoring minimal residual disease in children with high-risk relapses of acute lymphoblastic leukemia: prognostic relevance of early and late assessment

Leukemia (2015)

Cornelia Eckert Nikola Hagedorn Lucie Šrámková Georg Mann R Panzer-Grümayer

Minimal Residual Disease

Induction chemotherapy

Acute lymphocytic leukemia

10.1038/leu.2015.59

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Citations (63)

Single center experience with emicizumab for primary bleeding prophylaxis in children < 2 years of age with severe hemophilia A

Hämostaseologie (2023)

Madlen Reschke Franziska Cuntz J. Ruthenberg Susanne Holzhauer Nikola Hagedorn

Introduction Primary bleeding prophylaxis with FVIII is still standard of care for patients with severe hemophilia A. Prophylaxis usually starts at the end of the first year of life. Major challenges of prophylaxis are venous access and development of neutralizing antibodies against factor VIII. With emicizumab an effective alternative drug for primary prophylaxis has been introduced. We report the management of primary prophylaxis with emicizumab in children <2 years of age with hemophilia.

Secondary prophylaxis

Single Center

Venous access

10.1055/s-0042-1760531

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Current Diagnostic and Therapeutic Approaches in May–Thurner Syndrome in Children, Adolescents, and Young Adults: A Survey among Thrombosis Experts of the German Society of Thrombosis and Haemostasis

Hämostaseologie (2024)

Franziska Cuntz Bernhard Gebauer Andreas Greiner Nikola Hagedorn Madlen Reschke

Abstract May–Thurner syndrome (MTS) is a pelvic venous disorder involving compression of the left common iliac vein by the right common iliac artery, which results in predisposition for deep vein thrombosis. Although MTS is increasingly recognized in young patients, specific guidelines on diagnosis and management for children, adolescents, and young adults do not exist so far. The aim of this study was to assess current diagnostic and therapeutic practice in Germany, Austria, and Switzerland in children and young adults with thrombosis and MTS. We designed an online survey with 11 questions, which we sent via a mailing list to all members of the German, Austrian, and Swiss Society of Thrombosis and Haemostasis Research. Between July and October 2022, 33 specialists answered the questionnaire. Most participating specialists worked at pediatric hospitals (61%). Numbers of annually treated thromboses ranged from <5 (26%) to >30 (13%). Most specialists used venous ultrasound to diagnose deep vein thrombosis, 53% magnetic resonance imaging. Only 25% of specialists systematically screened for MTS in deep vein thrombosis. MTS was managed with anticoagulation (65%), iliac vein stent placement (32%), or balloon angioplasty (13%). In total, 31% of specialists reported to use more than one therapeutic method. Diagnostic and therapeutic approaches for MTS differed between specialists. Lack of standardization resulted in individualized and highly diverse management. Prospective observational clinical studies investigating the outcome of different management strategies including long-term follow-up on outcome and incidence of postthrombotic syndrome will help in defining patient groups who benefit most from revascularizing interventional strategies and developing standardized guidelines.

May–Thurner syndrome

Post-thrombotic syndrome

10.1055/a-2282-4565

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Changes in Hemophilia Treatment in the Eastern Part of Germany between 2015 and 2021—Data from the Kompetenznetz Hämorrhagische Diathese Ost (KHDO)

Hämostaseologie (2025)

Christian Pfrepper Annika Siecke Robert Klamroth Caspar D. Kühnöl Karim Kentouche

Abstract Introduction Treatment options for patients with hemophilia (PWH) have changed substantially in the last years. This study aimed to compare hemophilia treatment in the eastern part of Germany in 2021 with data from 2015. Methods Substitution diaries and patient records of PWH from 2021 were collected in 13 hemophilia centers from the “Kompetenznetz Hämorrhagische Diathese Ost” (KHDO) and compared with 2015. Results A total of 130 children and 357 adults, 411 hemophilia A (HA) and 76 hemophilia B (HB), were included in 2021, and 359 were already analyzed in 2015. In 2021, 97.8% of children and 95.7% of adults with severe hemophilia had prophylaxis compared with 98.8 and 80.2% in 2015. Plasma-derived concentrates were used in 25.6%, recombinant in 30.2%, extended half-life (EHL) factor concentrates in 24.4%, and emicizumab in 19.8% of the children with severe HA (sHA). In adults with sHA, plasma-derived, recombinant concentrates, EHL, and emicizumab were used in 21.0, 33.2, 31.2, and 14.2%, respectively. All children and 93.3% of the adults with severe HB (sHB) were on EHL. Median annual factor consumption per body weight increased in adults with sHA, remained stable in children with sHA and adults with sHB, and decreased in children with sHB between 2015 and 2021. Annualized bleeding rate (ABR) decreased in children with sHB and sHA. Conclusion The use of EHL and emicizumab has changed hemophilia treatment. About 50% of the sHA patients switched to EHL or emicizumab and almost all sHB patients to EHL. More adults with sHA received prophylaxis and ABR decreased in children.

10.1055/a-2499-2912

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Changes in hemophilia treatment in the eastern part of Germany between 2015 and 2021 – data from the Kompetenznetz Hämorrhagische Diathese Ost (KHDO)

Hämostaseologie (2024)

Christian Pfrepper Annika Siecke Robert Klamroth Caspar D. Kühnöl Karim Kentouche

Introduction Treatment options for patients with hemophilia (PWH) have changed substantially in the last years. Since 2015, extended half-life factor concentrates (EHL) and the bispecific antibody emicizumab have been approved. The aim of this epidemiologic study was to compare hemophilia treatment in the eastern part of Germany in 2021 with data from 2015 prior to the approval of EHL and emicizumab.

Bispecific antibody

10.1055/s-0044-1779142

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Quantitativer Ultraschall bei Kindern und Jugendlichen mit Hämophilie

RöFo - Fortschritte auf dem Gebiet der Röntgenstrahlen und der bildgebenden Verfahren (2023)

L. Kanthack Karim Kentouche Nikola Hagedorn Ralf Knöfler S Kim

Hintergrund Hämophilie kann den Knochenstoffwechsel bei Kindern und Jugendlichen negativ beeinflussen. Wiederholte Blutungen schädigen die Gelenke und durch Immobilisation kann die funktionelle Muskel-Knochen-Einheit beeinträchtigt sein. Der Quantitative Ultraschall (QUS) am Kalkaneus (Quertransmission) ist geeignet, um den Knochenstatus bei Kindern und Jugendlichen ohne ionisierende Strahlung zu untersuchen. Breitbandultraschallabschwächung (BUA) [dB/MHz], Schallleitungsgeschwindigkeit (SOS) [m/s] und Steifigkeitsindex (SI) liefern Informationen zur Dichte, Struktur und Elastizität des Kalkaneus.

10.1055/s-0043-1771429

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Noonon syndrome as unlikely cause of increased bruising in a case of suspected battered child syndrome

Hämostaseologie (2021)

Nikola Hagedorn C Mischung Madlen Reschke

Objective On a regular basis, children are referred to our hemostaseology center because of increased bruising in order to rule out bleeding disorders. Usually diseases like hemophilia or Willebrand syndrome, as well as thrombozytopathy or thrombozytopenia, or other selective factor deficiencies are examined.

Shaken Baby Syndrome

Battered Child Syndrome

10.1055/s-0041-1728164

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Very Early/Early Relapses of ALL Show Unexpected Changes of Clonal Markers and High Heterogeneity in Initial and Relapse Treatment Response: ALL-BFM 2000 and ALL-REZ BFM 96/2002.

Blood (2009)

Cornelia Eckert Rolf Koehler Thomas Flohr Nikola Hagedorn Anja Moericke

Minimal Residual Disease

Gene rearrangement

10.1182/blood.v114.22.2612.2612

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Citations (1)

Ambulante Nachsorge nach stationärer Adipositasrehabilitation: Machbarkeit des KgAS-Nachsorgekonzeptes

Die Rehabilitation (2019)

Miriam Pankatz Ines Gellhaus Nikola Hagedorn Petra Hampel Uwe Tiedjen

Zusammenfassung Rehabilitationsmaßnahmen für Kinder und Jugendliche mit Adipositas sind kurzfristig sehr erfolgreich, die Rückfallrate ist jedoch hoch. Nachsorgeprogramme hatten in Wirksamkeitsstudien keinen nachweisbaren Erfolg oder wurden eingestellt. Die Konsensusgruppe Adipositasschulung für Kinder und Jugendliche (KgAS) hat daher ein neues Programm entwickelt, das in einer Machbarkeitsstudie evaluiert wurde. In Kooperation der Fachklinik Sylt für Kinder und Jugendliche der DRV Nord mit ambulanten Partnern wurde 25 Familien die Teilnahme ermöglicht. Während des stationären Aufenthaltes des Kindes wurden sie in eine anschließende ambulante Nachsorge am Heimatort vermittelt. Die Teilnahme wurde durch eine Fragebogenerhebung (Eltern, Kinder/Jugendliche, Nachsorgekräfte) begleitet, außerdem wurden Einzelgespräche und ein Fokusgruppeninterview mit den Nachsorgekräften ausgewertet. Die Auswertung zeigte eine gute Machbarkeit und hohe Zufriedenheitswerte bei allen beteiligten Gruppen (Kinder/Jugendliche, Eltern, Nachsorgekräfte, Rehabilitationsklinik). Somit scheint der Nachsorgeansatz der KgAS geeignet, in das Regelangebot der Behandlung überführt zu werden. Zukünftig muss jedoch eine randomisiert-kontrollierte Studie die Wirksamkeit überprüfen.

10.1055/a-0808-1076

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Citations (8)

Improving Stratification for Children With Late Bone Marrow B-Cell Acute Lymphoblastic Leukemia Relapses With Refined Response Classification and Integration of Genetics

Journal of Clinical Oncology (2019)

Cornelia Eckert Stefanie Groeneveld‐Krentz Renate Kirschner‐Schwabe Nikola Hagedorn Christiane Chen‐Santel

Minimal residual disease (MRD) helps to accurately assess when children with late bone marrow relapses of B-cell precursor (BCP) acute lymphoblastic leukemia (ALL) will benefit from allogeneic hematopoietic stem-cell transplantation (allo-HSCT). More detailed dissection of MRD response heterogeneity and the specific genetic aberrations could improve current practice.MRD was assessed after induction treatment and at different times during relapse treatment until allo-HSCT (indicated in poor responders to induction; MRD ≥ 10-3) for patients being treated for late BCP-ALL bone marrow relapses (n = 413; median follow-up, 9.4 years) in the ALL-REZ BFM 2002 trial/registry (ClinicalTrials.gov identifier: NCT00114348).Patients with both good (MRD < 10-3) and poor responses to induction treatment reached excellent event-free survival (EFS; 72% v 65%) and overall survival (OS; 82% v 74%). Patients with MRD of 10-2 or greater after induction had reduced EFS (56%), and their MRD persisted until allo-HSCT more frequently than it did in patients with MRD of 10-3 or greater to less than 10-2 (P = .037). Patients with 25% or more leukemic blasts after induction (early nonresponders) had the poorest prognosis (EFS, 22%). Interestingly, patients with MRD of 10-3 or greater before allo-HSCT (late nonresponders) still had an EFS of 50% and OS of 63%, which in principle justifies allo-HSCT in these patients. From a panel of selected candidate genes, TP53 alterations (frequency, 8%) were the only genetic alteration with independent prognostic value in any MRD-based response subgroup.After induction treatment, MRD-based treatment stratification resulted in excellent survival in patients with late relapsed BCP-ALL. Prognosis could be further improved in very poor responders by intensifying treatment directly after induction. TP53 alterations can be defined as a novel genetic high-risk marker in all MRD response groups in late relapsed BCP-ALL. Here we identified early and late nonresponders to be considered as events in future trials.

Minimal Residual Disease

10.1200/jco.19.01694

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Citations (27)