Atypical Hemolytic Uremic Syndrome: Differential Diagnosis from TTP/HUS and Management
Atypical hemolytic uremic syndrome (aHUS) is a rare form of thrombotic microangiopathy (TMA). It has an unfavorable outcome with death rates as high as 25% during the acute phase and up to 50% of cases progressing to end-stage renal failure. Uncontrolled complement activation through the alternative...
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| Format: | Article |
| Language: | English |
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Galenos Publishing House
2014-08-01
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| Series: | Turkish Journal of Hematology |
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| Online Access: | https://jag.journalagent.com/z4/download_fulltext.asp?pdir=tjh&un=TJH-66642 |
| _version_ | 1797921046986227712 |
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| author | Mustafa N. Yenerel |
| author_facet | Mustafa N. Yenerel |
| author_sort | Mustafa N. Yenerel |
| collection | DOAJ |
| description | Atypical hemolytic uremic syndrome (aHUS) is a rare form of thrombotic microangiopathy (TMA). It has an unfavorable outcome with death rates as high as 25% during the acute phase and up to 50% of cases progressing to end-stage renal failure. Uncontrolled complement activation through the alternative pathway is thought to be the main underlying pathopysiology of aHUS and corresponds to all the deleterious findings of the disease. Thrombotic thrombocytopenic purpura (TTP) and Shiga toxin-associated HUS are the 2 other important TMA diseases. Although differentiating HUS from TTP is relatively easy in children with a preceding diarrheal illness or invasive S. pneumoniae, differentiating aHUS from TTP or other microangiopathic disorders can present a major diagnostic challenge in adults. ADAMTS13 analysis is currently the most informative diagnostic test for differentiating TTP, congenital TTP, and aHUS. Today empiric plasma therapy still is recommended by expert opinion to be used as early as possible in any patient with symptoms of aHUS. The overall treatment goal remains restoration of a
physiological balance between activation and control of the alternative complement pathway. So it is a reasonable approach to block the terminal complement complex with eculizumab in order to prevent further organ injury and increase the likelihood organ recovery. Persistence of hemolysis or lack of improvement of renal function after 3-5 daily plasmaphereses have to be regarded as the major criteria for uncontrolled TMA even if platelet count has normalized and as an indication to switch the treatment to eculizumab. Eculizumab has changed the future perspectives of patients with aHUS and both the FDA and the EMA have approved it as life-long treatment. However, there are still some unresolved issues about the follow-up such as the optimal duration of eculizumab treatment and whether it can be stopped or how to stop the therapy. |
| first_indexed | 2024-04-10T14:10:25Z |
| format | Article |
| id | doaj.art-da28e45ae2764b4eaeb59c58ba2b20f3 |
| institution | Directory Open Access Journal |
| issn | 1308-5263 |
| language | English |
| last_indexed | 2024-04-10T14:10:25Z |
| publishDate | 2014-08-01 |
| publisher | Galenos Publishing House |
| record_format | Article |
| series | Turkish Journal of Hematology |
| spelling | doaj.art-da28e45ae2764b4eaeb59c58ba2b20f32023-02-15T16:09:47ZengGalenos Publishing HouseTurkish Journal of Hematology1308-52632014-08-0131321622510.4274/tjh.2013.0374TJH-66642Atypical Hemolytic Uremic Syndrome: Differential Diagnosis from TTP/HUS and ManagementMustafa N. Yenerel0İstanbul University İstanbul Faculty Of Medicine, Department Of Internal Medicine, Division Of Hematology, İstanbul, TurkeyAtypical hemolytic uremic syndrome (aHUS) is a rare form of thrombotic microangiopathy (TMA). It has an unfavorable outcome with death rates as high as 25% during the acute phase and up to 50% of cases progressing to end-stage renal failure. Uncontrolled complement activation through the alternative pathway is thought to be the main underlying pathopysiology of aHUS and corresponds to all the deleterious findings of the disease. Thrombotic thrombocytopenic purpura (TTP) and Shiga toxin-associated HUS are the 2 other important TMA diseases. Although differentiating HUS from TTP is relatively easy in children with a preceding diarrheal illness or invasive S. pneumoniae, differentiating aHUS from TTP or other microangiopathic disorders can present a major diagnostic challenge in adults. ADAMTS13 analysis is currently the most informative diagnostic test for differentiating TTP, congenital TTP, and aHUS. Today empiric plasma therapy still is recommended by expert opinion to be used as early as possible in any patient with symptoms of aHUS. The overall treatment goal remains restoration of a physiological balance between activation and control of the alternative complement pathway. So it is a reasonable approach to block the terminal complement complex with eculizumab in order to prevent further organ injury and increase the likelihood organ recovery. Persistence of hemolysis or lack of improvement of renal function after 3-5 daily plasmaphereses have to be regarded as the major criteria for uncontrolled TMA even if platelet count has normalized and as an indication to switch the treatment to eculizumab. Eculizumab has changed the future perspectives of patients with aHUS and both the FDA and the EMA have approved it as life-long treatment. However, there are still some unresolved issues about the follow-up such as the optimal duration of eculizumab treatment and whether it can be stopped or how to stop the therapy.https://jag.journalagent.com/z4/download_fulltext.asp?pdir=tjh&un=TJH-66642atypical hemolytic uremic syndrome (ahus)thrombotic thrombocytopenic purpura (ttp)eculizumabttp/ husthrombotic microangiopathy (tma)adamts13 |
| spellingShingle | Mustafa N. Yenerel Atypical Hemolytic Uremic Syndrome: Differential Diagnosis from TTP/HUS and Management Turkish Journal of Hematology atypical hemolytic uremic syndrome (ahus) thrombotic thrombocytopenic purpura (ttp) eculizumab ttp/ hus thrombotic microangiopathy (tma) adamts13 |
| title | Atypical Hemolytic Uremic Syndrome: Differential Diagnosis from TTP/HUS and Management |
| title_full | Atypical Hemolytic Uremic Syndrome: Differential Diagnosis from TTP/HUS and Management |
| title_fullStr | Atypical Hemolytic Uremic Syndrome: Differential Diagnosis from TTP/HUS and Management |
| title_full_unstemmed | Atypical Hemolytic Uremic Syndrome: Differential Diagnosis from TTP/HUS and Management |
| title_short | Atypical Hemolytic Uremic Syndrome: Differential Diagnosis from TTP/HUS and Management |
| title_sort | atypical hemolytic uremic syndrome differential diagnosis from ttp hus and management |
| topic | atypical hemolytic uremic syndrome (ahus) thrombotic thrombocytopenic purpura (ttp) eculizumab ttp/ hus thrombotic microangiopathy (tma) adamts13 |
| url | https://jag.journalagent.com/z4/download_fulltext.asp?pdir=tjh&un=TJH-66642 |
| work_keys_str_mv | AT mustafanyenerel atypicalhemolyticuremicsyndromedifferentialdiagnosisfromttphusandmanagement |