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Autoimmunity Reviews Apr 2022To describe the real-world experience of eculizumab use in patients with catastrophic antiphospholipid syndrome (CAPS) according to the information provided by the "CAPS... (Review)
Review
OBJECTIVES
To describe the real-world experience of eculizumab use in patients with catastrophic antiphospholipid syndrome (CAPS) according to the information provided by the "CAPS Registry".
METHODS
We analyzed the demographic, clinical and immunological data from all the patients included in the "CAPS Registry" treated with eculizumab and described the indications for eculizumab administration, dose, outcome, use of prophylactic vaccines and adverse effects.
RESULTS
The "CAPS Registry" currently includes 584 patients from whom 39 (6.7%) were treated with eculizumab (it was used as a rescue therapy in 30 cases while in 6 cases it was used as first line therapy). Mean age of eculizumab treated patients was 39 years (SD = 14.6), 72% were female, 77% had a primary APS and 79% had a precipitating factor before the CAPS event. Thrombocytopenia was present in 28 (72%) cases and features of microangiopathic hemolytic anemia were present in 15 (38.5%). Twenty-nine (74.4%) patients recovered from the episode of CAPS (four showed only partial remission). Symptoms worsened in 9 patients, from whom 5 finally died despite the treatment. There was only one relapse after a median follow up of 10.7 months. The most common treatment regimen was 900 mg weekly for four weeks and 1200 mg fortnightly.
CONCLUSION
According to the real-world experience provided by the "CAPS Registry", eculizumab can be considered in some patients with CAPS refractory to previous therapies, especially if they present with features of complement-mediated thrombotic microangiopathy.
Topics: Adult; Anemia, Hemolytic; Antibodies, Monoclonal, Humanized; Antiphospholipid Syndrome; Catastrophic Illness; Female; Humans; Male; Middle Aged; Registries; Thrombocytopenia
PubMed: 35085802
DOI: 10.1016/j.autrev.2022.103055 -
Lupus Oct 2018
Review
Topics: Antibodies, Antiphospholipid; Antiphospholipid Syndrome; Humans; Nervous System
PubMed: 30452328
DOI: 10.1177/0961203318801671 -
Pediatric Rheumatology Online Journal Feb 2022Describe the frequency of thrombotic and non-thrombotic clinical manifestations, laboratory, treatment and prognosis in patients with pediatric primary antiphospholipid... (Review)
Review
OBJECTIVE
Describe the frequency of thrombotic and non-thrombotic clinical manifestations, laboratory, treatment and prognosis in patients with pediatric primary antiphospholipid syndrome.
MATERIAL AND METHODS
A retrospective study was carried out in patients with a diagnosis of primary antiphospholipid antibody syndrome, under 16 years of age, under follow-up by the pediatric rheumatology service of the General Hospital, National Medical Center, La Raza, from January 2013 to December 2020. The antiphospholipid syndrome was defined when it met the laboratory criteria of the Sidney criteria and the presence of thrombosis or non-criteria manifestations of the disease (hematological, neurological, cutaneous, renal, cardiac or pulmonary). Demographic, clinical, laboratory, treatment, and prognosis data were collected.
RESULTS
We report 32 patients, 21 female (65%) and 11 male (35%), mean age 11.75 years, evolution time 16 weeks. Thrombosis 9 patients (28%), 1 arterial and 8 venous. Non-thrombotic manifestations; Hematologic: thrombocytopenia 22 patients (69%), autoimmune hemolytic anemia 13 (40%), Fisher-Evans syndrome 6 (19%), lupus anticoagulant with hypoprothrombinemia syndrome 2 (6%). Dermatological: livedo reticularis 20 (62%), skin ulcers 2 (6%), Raynaud's phenomenon 8 (25%). Neurological: epilepsy 1 (3%), migraine 3 (9%), chorea 1 (3%) and cognitive impairment 3 (9%). Renal in 4 (13%). Laboratory: prolonged aPTT 30 (93%), lupus anticoagulant 32 (100%), positive IgG anticardiolipin 20 (62%), positive IgM anticardiolipin 19 (60%). AntiB2GPI was performed in only 3 patients, being positive in all.
TREATMENT
anticoagulation in patients with thrombosis, antiplatelet in 23 (72%), steroid 30 (94%), immunosuppressant 30 (94%) and rituximab 4 (12.5%). No deaths were reported.
CONCLUSIONS
The clinical characteristics of patients with pediatric primary antiphospholipid syndrome differ from those presented in adults, since non-thrombotic manifestations are more frequent in children, for which classification criteria that include these manifestations are necessary for a better characterization of the disease in pediatric population.
Topics: Adolescent; Antiphospholipid Syndrome; Child; Child, Preschool; Female; Humans; Infant; Male; Retrospective Studies
PubMed: 35164787
DOI: 10.1186/s12969-022-00673-y -
Pathophysiology of Haemostasis and... 2006The catastrophic antiphospholipid syndrome is a potentially life-threatening condition with a high mortality, which requires a high degree of clinical awareness on the... (Review)
Review
The catastrophic antiphospholipid syndrome is a potentially life-threatening condition with a high mortality, which requires a high degree of clinical awareness on the part of attending physicians. Patients with this syndrome have in common: a) clinical evidence of multiple organ involvement developed over a very short time period; b) histopathological evidence of multiple small vessel occlusions, and c) laboratory confirmation of the presence of antiphospholipid antibodies, usually in high titre. The combination of high doses of intravenous (iv) heparin, iv steroids, iv gamma globulins and/or repeated plasma exchanges is the basic treatment of choice for all patients with this severe condition.
Topics: Algorithms; Antiphospholipid Syndrome; Catastrophic Illness; Humans; Treatment Outcome
PubMed: 16855368
DOI: 10.1159/000093565 -
Clinical and Experimental Medicine Nov 2003The antiphospholipid syndrome (APS) was reported in the early 1980s as the association of thrombosis, recurrent pregnancy loss in the presence of anticardiolipin... (Review)
Review
The antiphospholipid syndrome (APS) was reported in the early 1980s as the association of thrombosis, recurrent pregnancy loss in the presence of anticardiolipin antibodies (aCL) and/or lupus anticoagulant (LA). Since then, many other clinical manifestations have been associated with aPL. Almost any organ and tissue may be involved in the disease, including the brain, the heart, the kidneys, the placenta and many more. aPL are a heterogeneous group of autoantibodies that are detected by immunoassays and functional coagulation tests. The antigenic targets are negatively charged phospholipids and serum phospholipid-binding proteins. Despite the strong association between aPL and thrombosis, the pathogenic role of aPL in the development of thrombosis has not been fully elucidated. Proposed mechanisms include antibody-mediated interference with coagulation homeostasis, activation of platelets and endothelial cells and a T-cell immune response to serum phospholipid-binding proteins. The mainstay of therapy is anticoagulation, whereas immunosuppression seems to be ineffective. Recommendations for the management of thrombosis in the antiphospholipid antibody syndrome have been based largely on retrospective case series. Several prospective clinical trials are currently underway and their results will probably lead to a more precise therapeutic approach of this problem.
Topics: Abortion, Habitual; Antiphospholipid Syndrome; Female; Fibrinolytic Agents; Humans; Pregnancy; Pregnancy Complications; Thrombosis
PubMed: 14648227
DOI: 10.1007/s10238-003-0016-x -
Reumatologia Clinica 2017Antiphospholipid antibody syndrome is a non-inflammatory autoimmune disease characterized by recurrent thrombotic events and/or obstetric complications associated with... (Review)
Review
Antiphospholipid antibody syndrome is a non-inflammatory autoimmune disease characterized by recurrent thrombotic events and/or obstetric complications associated with the presence of circulating antiphospholipid antibodies (anticardiolipin antibodies, anti-β glycoprotein-i antibodies, and/or lupus anticoagulant. Antiphospholipid antibodies are a heterogeneous group of autoantibodies associated with recurrent miscarriage, stillbirth, fetal growth restriction and premature birth. The diversity of the features of the proposed placental antiphospholipid antibodies fingerprint suggests that several disease processes may occur in the placentae of women with antiphospholipid antibody syndrome in the form of immune responses: inflammatory events, complement activation, angiogenic imbalance and, less commonly, thrombosis and infarction. Because of the disparity between clinical and laboratory criteria, and the impact on perinatal outcome in patients starting treatment, we reviewed the aspects of antiphospholipid antibody syndrome related to obstetric complications and seronegative antiphospholipid antibody syndrome, and their treatment in obstetrics.
Topics: Antiphospholipid Syndrome; Female; Humans; Pregnancy; Pregnancy Complications
PubMed: 27291869
DOI: 10.1016/j.reuma.2016.04.011 -
International Archives of Allergy and... Sep 2000Antiphospholipid syndrome (APS) is a disease characterized by venous and arterial thromboses or spontaneous abortions and the repeated detection of antiphospholipid... (Review)
Review
Antiphospholipid syndrome (APS) is a disease characterized by venous and arterial thromboses or spontaneous abortions and the repeated detection of antiphospholipid antibodies (aPL). APS may be associated with another autoimmune disease (secondary APS), particularly systemic lupus erythematosus (SLE), or unrelated to an underlying disease (primary APS). APS affects almost all organs. In addition to the clinical criteria, lupus anticoagulant testing and immunological aPL determinations are required to establish the diagnosis of APS.
Topics: Antiphospholipid Syndrome; Diagnosis, Differential; Female; Humans; Male; Pregnancy; Pregnancy Complications, Cardiovascular
PubMed: 11014973
DOI: 10.1159/000024425 -
Current Opinion in Rheumatology Sep 2017To review the difficult syndrome of catastrophic antiphospholipid syndrome, emphasizing new developments in the diagnosis, pathogenesis and treatment. (Review)
Review
PURPOSE OF REVIEW
To review the difficult syndrome of catastrophic antiphospholipid syndrome, emphasizing new developments in the diagnosis, pathogenesis and treatment.
RECENT FINDINGS
Few recent publications directly address pediatric catastrophic antiphospholipid syndrome (CAPS). Most articles are case reports or are data from adult and pediatric registries. The major factors contributing to most pediatric catastrophic antiphospholipid syndrome include infection and the presence of antiphospholipid antibodies, but complement activation also is important in creating diffuse thrombosis in the microcirculation. Treatment of the acute emergency requires anticoagulation, suppression of the hyperinflammatory state and elimination of the triggering infection. Inhibition of complement activation appears to improve outcome in limited studies, and suppression of antiphospholipid antibody formation may be important in long-term management.
SUMMARY
CAPS, an antibody-mediated diffuse thrombotic disease of microvasculature, is rare in childhood but has high mortality (33-50%). It requires prompt recognition and aggressive multimodality treatment, including anticoagulation, anti-inflammatory therapy and elimination of inciting infection and pathogenic autoantibodies.
Topics: Antibodies, Antiphospholipid; Antiphospholipid Syndrome; Autoimmunity; Catastrophic Illness; Child; Disease Management; Humans
PubMed: 28632503
DOI: 10.1097/BOR.0000000000000426 -
Autoimmunity Reviews Oct 2018The optimal treatment of women with primary antiphospholipid syndrome (APS) is still debated. About 20-30% of women with APS remain unable to give birth to healthy... (Review)
Review
BACKGROUND
The optimal treatment of women with primary antiphospholipid syndrome (APS) is still debated. About 20-30% of women with APS remain unable to give birth to healthy neonates despite conventional treatment, consisting of prophylactic-dose heparin and low-dose aspirin. These cases are defined "refractory obstetric APS". The early identification of risk factors associated with poor pregnancy outcome could be the optimal strategy to establish criteria for additional therapies, such as hydroxychloroquine, steroids, intravenous immunoglobulin, and plasma exchange.
PURPOSE
The aim of the present study was to review current literature about risk factors for poor pregnancy outcome.
SEARCH METHODS
The PubMed database was used to search for peer-reviewed original and review articles concerning risk factors for pregnancy outcome in APS from 1st January 1990 to 15th January 2018.
OUTCOMES
History of pregnancy morbidity and/or thrombosis, the association with SLE and/or other autoimmune diseases are well known history-based predictive factors for obstetrical complications, such as miscarriage, maternal venous thromboembolism, intrauterine foetal demise, preeclampsia, and neonatal death. Moreover, laboratory findings associated with poor pregnancy outcome are:triple antiphospholipid antibodies aPL positivity, double aPL positivity, single aPL positivity, false-positive IgM for CMV, and hypocomplementemia. Triple positivity is confirmed as the most significant risk factor by a large body of evidence. Furthermore, the abnormal uterine arteries Doppler velocimetry results are confirmed to be strongly associated with poor pregnancy outcomes in APS. The good performance of the uterine arteries velocimetry, as a negative predictive factor, was reported by different studies. On the contrary, in case of abnormal uterine arteries results, the relevance of a careful surveillance is highlighted for the high risk of maternal-foetal complications. Nevertheless, this tool is a late indicator to suggest any additional treatments.
CONCLUSIONS
In order to prevent obstetrical complications and establish the optimal combination therapy, the knowledge at preconception or at the beginning of pregnancy of risk factors associated with poor pregnancy outcome could be a crucial step for management and treatment of APS. In addition, in the preconception assessment a regimen with low-dose aspirin, folic acid, and vitamin D supplementation should be offered, and a treatment strategy has to be established (conventional vs additional therapy). In fact, additional treatment has to be tailored for each patient.
Topics: Antibodies, Antiphospholipid; Antiphospholipid Syndrome; Female; Humans; Pregnancy; Pregnancy Complications; Pregnancy Outcome; Risk Factors
PubMed: 30118899
DOI: 10.1016/j.autrev.2018.03.018 -
Seminars in Nephrology Jan 2007The antiphospholipid syndrome (APS) is an autoimmune disorder characterized by the clinical association of antiphospholipid autoantibodies (aPL) with a syndrome of... (Review)
Review
The antiphospholipid syndrome (APS) is an autoimmune disorder characterized by the clinical association of antiphospholipid autoantibodies (aPL) with a syndrome of hypercoagulability that can affect any blood vessel, irrespective of type or size. Involvement of larger vessels, such as arteries or veins, manifests in the form of thrombosis or embolism, whereas involvement of smaller vessels, including capillaries, arterioles, and venules, manifests as thrombotic microangiopathy. Virtually any organ in the body, including the kidney, can be affected. Here, we review the basic principles and recent advances in our understanding of APS, and discuss the broad spectrum of renal diseases that have been observed in association with this syndrome. We also discuss the impact that APS may have on pre-existing renal disease as well as current recommendations for treatment of APS.
Topics: Antiphospholipid Syndrome; Diagnosis, Differential; Humans; Lupus Nephritis; Terminology as Topic; Thrombosis
PubMed: 17336687
DOI: 10.1016/j.semnephrol.2006.09.006