Spontaneous Remission in Paroxysmal Nocturnal Hemoglobinuria—Return to Health or Transition Into Malignancy?
Paroxysmal nocturnal hemoglobinuria (PNH) is an acquired syndrome characterized by intravascular hemolysis, thrombosis, and bone marrow failure. The disease is caused by a mutation in the PIG-A gene that leads to the lack of glycosylphosphatidylinositol-anchored complement regulatory molecules CD55...
| Main Authors: | , , , |
|---|---|
| Format: | Article |
| Language: | English |
| Published: |
Frontiers Media S.A.
2018-08-01
|
| Series: | Frontiers in Immunology |
| Subjects: | |
| Online Access: | https://www.frontiersin.org/article/10.3389/fimmu.2018.01749/full |
| _version_ | 1828279839275089920 |
|---|---|
| author | Eva-Stina Korkama Anna-Elina Armstrong Hanna Jarva Hanna Jarva Seppo Meri Seppo Meri |
| author_facet | Eva-Stina Korkama Anna-Elina Armstrong Hanna Jarva Hanna Jarva Seppo Meri Seppo Meri |
| author_sort | Eva-Stina Korkama |
| collection | DOAJ |
| description | Paroxysmal nocturnal hemoglobinuria (PNH) is an acquired syndrome characterized by intravascular hemolysis, thrombosis, and bone marrow failure. The disease is caused by a mutation in the PIG-A gene that leads to the lack of glycosylphosphatidylinositol-anchored complement regulatory molecules CD55 and CD59 on affected blood cell surfaces. In previous studies, spontaneous clinical remissions have been described. The disease manifestations are very heterogeneous, and we wanted to examine if true remissions and disappearance of the clone occur. In a follow-up of a nation-wide cohort of 106 Finnish patients with a PNH clone, we found six cases, where the clone disappeared or was clearly diminished. Two of the patients subsequently developed leukemia, while the other four are healthy and in clinical remission. According to our data, spontaneous remissions are not as frequent as described earlier. Since the disappearance of the PNH cell clone may indicate either a favorable or a poor outcome—remission or malignancy—careful clinical monitoring in PNH is mandatory. Nevertheless, true remissions occur, and further studies are needed to understand the immunological background of this phenomenon and to obtain a better understanding of the natural history of the disease. |
| first_indexed | 2024-04-13T07:49:32Z |
| format | Article |
| id | doaj.art-d4f55107b86d437091181a56adbcdcfa |
| institution | Directory Open Access Journal |
| issn | 1664-3224 |
| language | English |
| last_indexed | 2024-04-13T07:49:32Z |
| publishDate | 2018-08-01 |
| publisher | Frontiers Media S.A. |
| record_format | Article |
| series | Frontiers in Immunology |
| spelling | doaj.art-d4f55107b86d437091181a56adbcdcfa2022-12-22T02:55:34ZengFrontiers Media S.A.Frontiers in Immunology1664-32242018-08-01910.3389/fimmu.2018.01749401965Spontaneous Remission in Paroxysmal Nocturnal Hemoglobinuria—Return to Health or Transition Into Malignancy?Eva-Stina Korkama0Anna-Elina Armstrong1Hanna Jarva2Hanna Jarva3Seppo Meri4Seppo Meri5Immunobiology Research Program, Department of Bacteriology and Immunology, University of Helsinki, Helsinki, FinlandCoagulation Disorder Unit, Helsinki University Hospital Comprehensive Cancer Center, Helsinki, FinlandImmunobiology Research Program, Department of Bacteriology and Immunology, University of Helsinki, Helsinki, FinlandHelsinki University Hospital Laboratory (HUSLAB), Helsinki, FinlandImmunobiology Research Program, Department of Bacteriology and Immunology, University of Helsinki, Helsinki, FinlandHelsinki University Hospital Laboratory (HUSLAB), Helsinki, FinlandParoxysmal nocturnal hemoglobinuria (PNH) is an acquired syndrome characterized by intravascular hemolysis, thrombosis, and bone marrow failure. The disease is caused by a mutation in the PIG-A gene that leads to the lack of glycosylphosphatidylinositol-anchored complement regulatory molecules CD55 and CD59 on affected blood cell surfaces. In previous studies, spontaneous clinical remissions have been described. The disease manifestations are very heterogeneous, and we wanted to examine if true remissions and disappearance of the clone occur. In a follow-up of a nation-wide cohort of 106 Finnish patients with a PNH clone, we found six cases, where the clone disappeared or was clearly diminished. Two of the patients subsequently developed leukemia, while the other four are healthy and in clinical remission. According to our data, spontaneous remissions are not as frequent as described earlier. Since the disappearance of the PNH cell clone may indicate either a favorable or a poor outcome—remission or malignancy—careful clinical monitoring in PNH is mandatory. Nevertheless, true remissions occur, and further studies are needed to understand the immunological background of this phenomenon and to obtain a better understanding of the natural history of the disease.https://www.frontiersin.org/article/10.3389/fimmu.2018.01749/fullparoxysmal nocturnal hemoglobinuriaaplastic anemiaspontaneous remissionleukemia |
| spellingShingle | Eva-Stina Korkama Anna-Elina Armstrong Hanna Jarva Hanna Jarva Seppo Meri Seppo Meri Spontaneous Remission in Paroxysmal Nocturnal Hemoglobinuria—Return to Health or Transition Into Malignancy? Frontiers in Immunology paroxysmal nocturnal hemoglobinuria aplastic anemia spontaneous remission leukemia |
| title | Spontaneous Remission in Paroxysmal Nocturnal Hemoglobinuria—Return to Health or Transition Into Malignancy? |
| title_full | Spontaneous Remission in Paroxysmal Nocturnal Hemoglobinuria—Return to Health or Transition Into Malignancy? |
| title_fullStr | Spontaneous Remission in Paroxysmal Nocturnal Hemoglobinuria—Return to Health or Transition Into Malignancy? |
| title_full_unstemmed | Spontaneous Remission in Paroxysmal Nocturnal Hemoglobinuria—Return to Health or Transition Into Malignancy? |
| title_short | Spontaneous Remission in Paroxysmal Nocturnal Hemoglobinuria—Return to Health or Transition Into Malignancy? |
| title_sort | spontaneous remission in paroxysmal nocturnal hemoglobinuria return to health or transition into malignancy |
| topic | paroxysmal nocturnal hemoglobinuria aplastic anemia spontaneous remission leukemia |
| url | https://www.frontiersin.org/article/10.3389/fimmu.2018.01749/full |
| work_keys_str_mv | AT evastinakorkama spontaneousremissioninparoxysmalnocturnalhemoglobinuriareturntohealthortransitionintomalignancy AT annaelinaarmstrong spontaneousremissioninparoxysmalnocturnalhemoglobinuriareturntohealthortransitionintomalignancy AT hannajarva spontaneousremissioninparoxysmalnocturnalhemoglobinuriareturntohealthortransitionintomalignancy AT hannajarva spontaneousremissioninparoxysmalnocturnalhemoglobinuriareturntohealthortransitionintomalignancy AT seppomeri spontaneousremissioninparoxysmalnocturnalhemoglobinuriareturntohealthortransitionintomalignancy AT seppomeri spontaneousremissioninparoxysmalnocturnalhemoglobinuriareturntohealthortransitionintomalignancy |