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Набор скоро начнётся NCT07605884

Denys-Drash Syndrome and Risk of Post-transplant Lymphoproliferative Disorder

— Наблюдательное Denys-Drash Syndrome

Ориентир для пациента и семьи

Простыми словами

Автоматическая сводка по структурированным данным реестра. Она помогает сориентироваться, но не заменяет официальный протокол или оценку врача.

Что изучают
В протоколе указаны: Collection of data from the patient's medical file.
Кому может быть актуально
Состояния в реестре: Denys-Drash Syndrome. Базовые параметры: Без ограничений · Все.
Что важно проверить
Возраст, диагноз и пол — только базовые ориентиры. Предыдущее лечение, анализы и другие обязательные условия указаны ниже в критериях участия.
Где проводится
Франция
Следующий шаг
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Официальное название

Post-transplant Lymphoproliferative Disorder Following Kidney Transplantation in Denys-Drash Syndrome: a Case-control Study

Обзор

Denys-Drash syndrome is a rare genetic disorder of childhood characterized by nephrotic syndrome, nephroblastomas, and genital developmental abnormalities. These children present with rapidly progressive renal failure, leading to kidney transplantation at a median age of 3.6 years. In a study of the French cohort of patients with Denys-Drash syndrome, a high risk of lymphoproliferative syndrome was observed (20%). This frequency is significantly higher than in the general transplant population (4%). The aim of the study is to evaluate the risk of post-transplant lymphoproliferative disorder following kidney transplantation in patients with Denys-Drash syndrome compared to patients with kidney transplant patients without Denys-Drash syndrome.

Подробное описание

Denys-Drash syndrome is a rare genetic disorder of childhood characterized by nephrotic syndrome, nephroblastomas, and genital developmental abnormalities. These children present with rapidly progressive renal failure, leading to kidney transplantation at a median age of 3.6 years. In a study of the French cohort of patients with Denys-Drash syndrome, a high risk of lymphoproliferative syndrome was observed (20%). This frequency is significantly higher than in the general transplant population (4%).

The aim of the study is to evaluate the risk of post-transplant lymphoproliferative disorder following kidney transplantation in patients with Denys-Drash syndrome compared to patients with kidney transplant patients without Denys-Drash syndrome.

The study hypothesizes that there is an increased risk of lymphoproliferative disorder in children with Denys-Drash syndrome. If this hypothesis is confirmed, it would allow for the development of active treatment methods to combat post-transplant lymphoproliferative disorder in this population.

Вмешательства

  • Другое Collection of data from the patient's medical file
    Collection of data from the patient's medical file. The data will be collected until 2024.

Первичные конечные точки

  • Occurrence of lymphoproliferative disorder [Срок оценки: Up to 24 years]
Вторичные конечные точки (2)
  • Description of risk factors of post-transplant lymphoproliferative disorder following kidney transplantation [Срок оценки: Up to 24 years]
  • Comparison of the time to onset of post-transplant lymphoproliferative disorder following kidney transplantation [Срок оценки: Up to 24 years]

Критерии участия

Критерии включения

  • Patient must be a minor at the time of kidney transplantation
  • Diagnosis of Denys-Drash syndrome (WT1 pathogenic variants in exons 8 or 9) for cases
  • Controls: minor patient, kidney transplanted at the same center as the case, immediately before and immediately after the case
  • Kidney transplant recipient on immunosuppressants
  • Hospital follow-up in a participating center in France
  • Regardless of their Epstein-Barr virus status before transplantation
  • Between 2000 and 2022
  • Holders of parental authority or adult patients informed of the study and not objecting to the processing of medical data for the study

Критерии исключения

  • History of lymphoproliferative disorder prior to transplantation
  • Other hematopoietic cancer
  • Other genetic disease with a proven increased risk of lymphoproliferative disorder

Критерии приведены из реестра в оригинале (на английском). Окончательную оценку соответствия проводит исследовательский центр.

Здоровые добровольцы: Нет

Дизайн исследования

Модель наблюдения
Случай-контроль

Центры проведения

Франция · 7 центров
  • Hôpital Femme Mère Enfant, Hospices Civils de Lyon — Bron
  • CHU de Lille — Lille
  • CHU Montpellier — Montpellier
  • CHU de Nancy - Hôpitaux de Brabois — Nancy
  • Hôpital enfants et adolescents, CHU Nantes — Nantes
  • Hôpital Necker-Enfants Malades — Paris
  • Hôpital Robert Debré — Paris

Публикации

  • Glenisson M, Grapin M, Blanc T, Preka E, Hogan J, Aurelle M, Roussey G, Mouche A, Rousset-Rouviere C, Novo R, Faudeux C, Fila M, Vrillon I, Cloarec S, Simon T, Harambat J, Casado EM, Rod J, Lecoindre MC, Heidet L, Boyer O, Garcelon N, Kachmar J, Dorval G, Sarnacki S. Genotype-Phenotype Correlations in Denys-Drash Syndrome in Children. Kidney Int Rep. 2025 Jan 16;10(4):1205-1212. doi: 10.1016/j.eki PMID 40303223
  • Lopez-Gonzalez M, Ariceta G. WT1-related disorders: more than Denys-Drash syndrome. Pediatr Nephrol. 2024 Sep;39(9):2601-2609. doi: 10.1007/s00467-024-06302-y. Epub 2024 Feb 7. PMID 38326647
  • Fulchiero R, Amaral S. Post-transplant lymphoproliferative disease after pediatric kidney transplant. Front Pediatr. 2022 Dec 7;10:1087864. doi: 10.3389/fped.2022.1087864. eCollection 2022. PMID 36568415
  • Mynarek M, Hussein K, Kreipe HH, Maecker-Kolhoff B. Malignancies after pediatric kidney transplantation: more than PTLD? Pediatr Nephrol. 2014 Sep;29(9):1517-28. doi: 10.1007/s00467-013-2622-5. Epub 2013 Sep 24. PMID 24061645

Идентификаторы

NCT: NCT07605884 · APHP251805

Первоисточники (государственные реестры)

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