Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
This study is a collection of data to evaluate safety and characterize progression of Paroxysmal Nocturnal Hemoglobinuria (PNH).
Collection of data to evaluate safety and characterize progression of Paroxysmal Nocturnal Hemoglobinuria (PNH).
Not provided
Not provided
Not provided
Not provided
Not provided
| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Receiving Soliris or Ultomiris | PNH patients of any age, including minors, that are receiving Soliris or Ultomiris | ||
| Not receiving Soliris or Ultomiris | PNH patients of any age, including minors, that are not receiving Soliris or Ultomiris |
Not provided
| Measure | Description | Time Frame |
|---|---|---|
| Evaluate safety data specific to the use of Soliris and Ultomiris | Primary analyses will assess safety endpoints, including occurrence and time to first event for the following: meningococcal infections, infections with serious outcomes, formation of human anti-drug antibodies (ADA) to Soliris and Ultomiris, malignancy, thrombotic events, pulmonary hypertension, impaired renal function, impaired hepatic function, hemolysis, pregnancies, lactation, infusion reactions, bone marrow transplant, serious adverse events, and mortality. | Ongoing (up to 13 years) |
| Measure | Description | Time Frame |
|---|---|---|
| Collect data to characterize the progression of PNH as well as clinical outcomes, mortality and morbidity in Soliris or Ultomiris and non-Soliris or non- Ultomiris treated patients | Secondary analyses will include descriptions of patient populations, PNH specific treatments, concomitant medications, progression of disease, PNH clone sites, clinical symptoms, and clinical outcomes. | Ongoing (up to 13 years) |
Not provided
Inclusion Criteria:
Exclusion Criteria:
Not provided
Not provided
Not provided
Not provided
PNH Patients
Not provided
| Name | Affiliation | Role |
|---|---|---|
| Phillipe Gustovic | Alexion Pharmaceuticals, Inc. | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Contact the PNH Registry at Alexion Pharmaceuticals, Inc. for worldwide locations. | Boston | Massachusetts | 02210 | United States |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 41795103 | Derived | Roth A, Patriquin CJ, Szer J, Terriou L, Patel AS, Metzger J, Gustovic P, Nishimura JI, Brodsky RA. Real-World Effectiveness and Safety of Ravulizumab in Patients With Paroxysmal Nocturnal Hemoglobinuria: Evidence From the International PNH Registry. Am J Hematol. 2026 May;101(5):939-948. doi: 10.1002/ajh.70268. Epub 2026 Mar 7. | |
| 37199789 |
| Label | URL |
|---|---|
| Registry Summary Overview | View source |
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
| ID | Term |
|---|---|
| D006457 | Hemoglobinuria, Paroxysmal |
| ID | Term |
|---|---|
| D000743 | Anemia, Hemolytic |
| D000740 | Anemia |
| D006402 | Hematologic Diseases |
| D006425 | Hemic and Lymphatic Diseases |
Not provided
Not provided
Not provided
Not provided
Not provided
| Dingli D, Maciejewski JP, Larratt L, Go RS, Hochsmann B, Zu K, Gustovic P, Kulagin AD. Relationship of paroxysmal nocturnal hemoglobinuria (PNH) granulocyte clone size to disease burden and risk of major vascular events in untreated patients: results from the International PNH Registry. Ann Hematol. 2023 Jul;102(7):1637-1644. doi: 10.1007/s00277-023-05269-4. Epub 2023 May 18. |
| 36108594 | Derived | Hill A, de Latour RP, Kulasekararaj AG, Griffin M, Brodsky RA, Maciejewski JP, Marantz JL, Gustovic P, Schrezenmeier H. Concomitant Immunosuppressive Therapy and Eculizumab Use in Patients with Paroxysmal Nocturnal Hemoglobinuria: An International PNH Registry Analysis. Acta Haematol. 2023;146(1):1-13. doi: 10.1159/000526979. Epub 2022 Sep 15. |
| 27766064 | Derived | Chou WC, Huang WH, Wang MC, Chang CS, Yeh SP, Chiou TJ, Chen YC, Lin TH, Shen MC; Taiwan PNH study group. Characteristics of Taiwanese patients of PNH in the international PNH registry. Thromb J. 2016 Oct 4;14(Suppl 1):39. doi: 10.1186/s12959-016-0094-0. eCollection 2016. |
| 26352814 | Derived | Kelly RJ, Hochsmann B, Szer J, Kulasekararaj A, de Guibert S, Roth A, Weitz IC, Armstrong E, Risitano AM, Patriquin CJ, Terriou L, Muus P, Hill A, Turner MP, Schrezenmeier H, Peffault de Latour R. Eculizumab in Pregnant Patients with Paroxysmal Nocturnal Hemoglobinuria. N Engl J Med. 2015 Sep 10;373(11):1032-9. doi: 10.1056/NEJMoa1502950. |
| D009190 |
| Myelodysplastic Syndromes |
| D001855 | Bone Marrow Diseases |