The Oklahoma thrombotic thrombocytopenic purpura-haemolytic uraemic syndrome Registry

Summary

The Oklahoma Thrombotic Thrombocytopenic Purpura-Haemolytic Uraemic Syndrome (TTPHUS) Registry has a 24 year record of success for collaborative clinical research, education, and patient care. This article tells the story of how the Registry began and it describes the Registry’s structure and function. The Registry provides a model for using a cohort of consecutive patients to investigate a rare disorder. Collaboration between Oklahoma, United States and Bern, Switzerland has been the basis for successful interpretation of Registry data.

Registry data have provided new insights into the evaluation and management of TTP. Because recovery from acute episodes of TTP has been assumed to be complete, the increased prevalence of hypertension, diabetes, depression, and death documented by long-term follow-up was unexpected. Registry data have provided opportunities for projects for students and trainees, education of physicians and nurses, and also for patients themselves. During our follow-up, patients have also educated Registry investigators about problems that persist after recovery from an acute episode of TTP. Most important, Registry data have resulted in important improvements for patient care.

Zusammenfassung

Das Oklahoma-Register für thrombotischthrombozytopenische Purpura / hämolytischurämisches Syndrom (TTP-HUS) steht für eine 24-jährige erfolgreiche Zusammenarbeit zwischen klinischer Forschung, Ausbildung und Patientenversorgung. In diesem Artikel erzählen wir die Geschichte über die Anfänge des Registers und beschreiben seine Struktur und Funktion. Das Register liefert ein Modell dafür, wie man eine Kohorte aufeinander folgender Patienten für die Erforschung seltener Krankheiten nutzen kann. Die Zusammen - arbeit zwischen Oklahoma, den Vereinigten Staaten und Bern in der Schweiz war die Grundlage für eine erfolgreiche Interpretation der Daten.

Die Registerdaten ermöglichen neue Ein - blicke in die Diagnose und Behandlung der TTP. Da man davon ausging, dass man sich von akuten TTP-Episoden wieder vollständig erholt, war die bei der Langzeitnachbeobachtung dokumentierte erhöhte Prävalenz von Hypertonie, Diabetes, Depression und Tod unerwartet. Die Registerdaten eröffneten Möglichkeiten für Projekte für Studenten und Praktikanten, Fortbildung von Ärzten und Pflegekräften und auch für die Patienten selbst. Während unseres Follow-up haben die Patienten die Ärzte des Registers auch fortlaufend über die Probleme informiert, die nach der Erholung von einem akuten TTPAnfall fortbestehen. Das bedeutendste Ergebnis ist, dass die Registerdaten zu einer Verbesserung der Patientenversorgung geführt haben.

Keywords

TTP-HUS - thrombotic thrombocytopenic purpura - cohort studies - rare disorders

Schlüsselwörter

TTP-HUS - thrombotisch-thrombozytopenische Purpura - Kohortenstudien - seltene Erkrankung

• References

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