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1.
N Engl J Med ; 384(14): 1323-1334, 2021 04 08.
Artículo en Inglés | MEDLINE | ID: mdl-33826820

RESUMEN

BACKGROUND: Cold agglutinin disease is a rare autoimmune hemolytic anemia characterized by hemolysis that is caused by activation of the classic complement pathway. Sutimlimab, a humanized monoclonal antibody, selectively targets the C1s protein, a C1 complex serine protease responsible for activating this pathway. METHODS: We conducted a 26-week multicenter, open-label, single-group study to assess the efficacy and safety of intravenous sutimlimab in patients with cold agglutinin disease and a recent history of transfusion. The composite primary end point was a normalization of the hemoglobin level to 12 g or more per deciliter or an increase in the hemoglobin level of 2 g or more per deciliter from baseline, without red-cell transfusion or medications prohibited by the protocol. RESULTS: A total of 24 patients were enrolled and received at least one dose of sutimlimab; 13 patients (54%) met the criteria for the composite primary end point. The least-squares mean increase in hemoglobin level was 2.6 g per deciliter at the time of treatment assessment (weeks 23, 25, and 26). A mean hemoglobin level of more than 11 g per deciliter was maintained in patients from week 3 through the end of the study period. The mean bilirubin levels normalized by week 3. A total of 17 patients (71%) did not receive a transfusion from week 5 through week 26. Clinically meaningful reductions in fatigue were observed by week 1 and were maintained throughout the study. Activity in the classic complement pathway was rapidly inhibited, as assessed by a functional assay. Increased hemoglobin levels, reduced bilirubin levels, and reduced fatigue coincided with inhibition of the classic complement pathway. At least one adverse event occurred during the treatment period in 22 patients (92%). Seven patients (29%) had at least one serious adverse event, none of which were determined by the investigators to be related to sutimlimab. No meningococcal infections occurred. CONCLUSIONS: In patients with cold agglutinin disease who received sutimlimab, selective upstream inhibition of activity in the classic complement pathway rapidly halted hemolysis, increased hemoglobin levels, and reduced fatigue. (Funded by Sanofi; CARDINAL ClinicalTrials.gov number, NCT03347396.).


Asunto(s)
Anemia Hemolítica Autoinmune/tratamiento farmacológico , Anticuerpos Monoclonales Humanizados/uso terapéutico , Complemento C1s/antagonistas & inhibidores , Anciano , Anciano de 80 o más Años , Anemia Hemolítica Autoinmune/sangre , Anemia Hemolítica Autoinmune/complicaciones , Anemia Hemolítica Autoinmune/terapia , Anticuerpos Monoclonales Humanizados/efectos adversos , Anticuerpos Monoclonales Humanizados/farmacología , Transfusión Sanguínea , Fatiga/tratamiento farmacológico , Fatiga/etiología , Femenino , Hemoglobinas/análisis , Hemólisis/efectos de los fármacos , Humanos , Masculino , Persona de Mediana Edad , Calidad de Vida
2.
Cochrane Database Syst Rev ; 3: CD012493, 2021 03 26.
Artículo en Inglés | MEDLINE | ID: mdl-33786812

RESUMEN

BACKGROUND: Primary autoimmune haemolytic anaemia (AIHA) is an autoantibody mediated condition characterised by a variable disease course. A myriad of immunomodulatory agents have been employed but there is a paucity of evidence to support their use or compare their effectiveness. OBJECTIVES: To determine the effects of various disease-modifying treatment modalities in people with AHIHA. SEARCH METHODS: We searched MEDLINE (Ovid) (1946 to 2021), Embase (Ovid) (1974 to 2021), Latin American and Caribbean Health Sciences Literature (LILACS) (1982 to 2021), and the Cochrane Library (CENTRAL). Clinical trial registries and relevant conference proceedings were also reviewed. Records were included as of 7 March 2021. We did not impose any language restrictions. SELECTION CRITERIA: Randomised controlled trials (RCTs) comparing immunosuppressive or immunomodulatory treatments against no treatment, placebo, or another immunosuppressive or immunomodulatory treatment, for people of all age with idiopathic AIHA. DATA COLLECTION AND ANALYSIS: We used standard methodological procedures expected by Cochrane. The prioritised pre-defined outcomes included complete haematological response at 12 months, frequency of adverse events at two, six and 12 months, partial haematological response at 12 months, overall survival at six and 12 months, relapse-free survival (RFS) at six and 12 months, red blood cel (RBC) transfusion requirement after treatment at 12 months, and quality of life (QOL) as measured by validated instruments at 12 months. Based on data availability, we were only able to perform meta-analysis on frequency of complete haematological response. MAIN RESULTS: Two trials were included, enrolling a total of 104 adult participants (96 randomised) with warm AIHA in the setting of tertiary referral centres, both comparing the effectiveness between rituximab (375 mg/m2 weekly for four weeks, or 1000 mg for two doses two weeks apart) plus glucocorticoid (prednisolone 1.5 or 1mg/kg/day with taper) and glucocorticoid monotherapy. The average age of participants in the two trials were 67 and 71, respectively. One of the included studies had good methodological quality with low risk of bias, whereas the other study had high risk of performance and detection bias due to lack of blinding. Compared with glucocorticoid alone, adding rituximab may result in a large increase of complete response at 12 months (n = 96, risk ratio (RR) 2.13, 95% confidence interval (CI) 1.34 to 3.40, GRADE: low-certainty evidence). Rates of adverse effects at prespecified time-points were not reported. Limited data on partial haematological response were reported. The evidence is very uncertain about the effect of adding rituximab to glucocorticoids on partial haematological response at 12 months (n = 32; study = 1; RR 3.00, 95% CI 0.13 to 68.57; GRADE very low-certainty evidence). RBC transfusion need at 12 months was reported in one study, with four participants (mean number of packed red cell units 4.0 ± 2.82) from the rituximab group and five participants from the placebo (corticosteroid only) (mean number of packed red cell units 5.6 ± 4.15) group requiring transfusion, indicating very uncertain evidence about the effect of adding rituximab to glucocorticoids (n = 32, RR 0.80, 95% CI 0.26 to 2.45, GRADE very low-certainty evidence). The other study did not report transfusion requirement at prespecified time points but reported no difference in transfusion requirement between the two groups when comparing responders from enrolment to end of response or to the end of study follow-up (34 units versus 30 units, median [range]: 0 [1 to 6] versus 0 [1 to 5], P = 0·81). Overall survival and RFS rates at prespecified time-points were not explicitly reported in either study. Data on QOL were not available. AUTHORS' CONCLUSIONS: Available literature on the effectiveness of immunomodulatory therapy for primary AIHA is restricted to comparison between rituximab plus glucocorticoid and glucocorticoid alone, in patients with newly diagnosed warm AIHA, calling for need for additional studies. The current result suggests that combinatory therapy with rituximab and glucocorticoid may increase the rate of complete haematological response over glucocorticoid monotherapy.


Asunto(s)
Anemia Hemolítica Autoinmune/tratamiento farmacológico , Glucocorticoides/uso terapéutico , Factores Inmunológicos/uso terapéutico , Prednisolona/uso terapéutico , Rituximab/uso terapéutico , Anciano , Sesgo , Intervalos de Confianza , Quimioterapia Combinada/métodos , Transfusión de Eritrocitos , Femenino , Glucocorticoides/administración & dosificación , Humanos , Factores Inmunológicos/administración & dosificación , Masculino , Prednisolona/administración & dosificación , Ensayos Clínicos Controlados Aleatorios como Asunto
3.
J Med Case Rep ; 14(1): 246, 2020 Dec 18.
Artículo en Inglés | MEDLINE | ID: mdl-33339534

RESUMEN

BACKGROUND: In December 2019, a new coronavirus (named severe acute respiratory syndrome coronavirus 2, SARS-CoV-2) spread from China, causing a pandemic in a very short time. The main clinical presentation of SARS-CoV-2 infection (COVID-19, coronavirus disease-2019) is pneumonia, but several cardiovascular complications may also occur (e.g., acute coronary syndromes, pulmonary embolism, stroke, arrhythmias, heart failure and cardiogenic shock). Direct or indirect mechanisms induced by SARS-CoV-2 could be implicated in the pathogenesis of these events. CASE PRESENTATION: We report herein the third case of COVID-19 autoimmune haemolytic anaemia (AIHA) reported so far, which occurredwithout any other possible explanations in a Caucasian patient. The patient also suffered from ST-elevation myocardial injury. CONCLUSIONS: Both complications occurred quite late after COVID-19 diagnosis and were probably precipitated by systemic inflammation, as indicated by a significant delayed increase in inflammatory markers, including interleukin-6 (IL-6).


Asunto(s)
Anemia Hemolítica Autoinmune/sangre , Infecciones Asintomáticas , Proteína C-Reactiva/inmunología , Interleucina-6/inmunología , Infarto del Miocardio con Elevación del ST/diagnóstico , Anciano de 80 o más Años , Anemia Hemolítica Autoinmune/tratamiento farmacológico , Anemia Hemolítica Autoinmune/etiología , Antibacterianos/uso terapéutico , Azitromicina/uso terapéutico , /tratamiento farmacológico , Prueba de Coombs , Electrocardiografía , Inhibidores Enzimáticos/uso terapéutico , Femenino , Glucocorticoides/uso terapéutico , Humanos , Hidroxicloroquina/uso terapéutico , Inhibidores de Agregación Plaquetaria/uso terapéutico , Prednisolona/uso terapéutico , Infarto del Miocardio con Elevación del ST/tratamiento farmacológico , Infarto del Miocardio con Elevación del ST/etiología
4.
Zhong Nan Da Xue Xue Bao Yi Xue Ban ; 45(6): 739-744, 2020 Jun 28.
Artículo en Inglés, Chino | MEDLINE | ID: mdl-32879133

RESUMEN

IgG4-related disease (IgG4-RD) is a rare autoimmune fibrosis disease characterized by elevated serum IgG4 and tissues as well as organs infiltrated with IgG4-positive cells, resulting in swelling and damage.It is currently treated as first-line treatment with glucocorticoids. Autoimmune hemolytic anemia (AIHA) is also a relatively rare disease that caused by autoreactive erythrocyte antibodies. Although both are autoimmune-related diseases, they rarely overlap. The relationship between them is not clear. A case of IgG4-RD combined with AIHA is reported. The patient has shortness of breath, cough, and sputum after physical activity. Physical examination showed appearance of anemia, yellow staining of skin and sclera, palpable neck and multiple swollen lymph nodes. Laboratory examination, bone marrow biopsy, and lymph node biopsy confirmed the diagnosis. Therefore, clinicians should develop ideas and raise awareness of such diseases.


Asunto(s)
Anemia Hemolítica Autoinmune/diagnóstico , Anemia Hemolítica Autoinmune/tratamiento farmacológico , Enfermedades Autoinmunes/complicaciones , Enfermedad Relacionada con Inmunoglobulina G4/complicaciones , Enfermedad Relacionada con Inmunoglobulina G4/diagnóstico , Biopsia , Humanos , Inmunoglobulina G
5.
J Stroke Cerebrovasc Dis ; 29(8): 104924, 2020 Aug.
Artículo en Inglés | MEDLINE | ID: mdl-32689602

RESUMEN

Dabigatran is an orally active direct thrombin inhibitor, initially approved by FDA for the prophylaxis of stroke and systemic embolism in the setting of non-valvular atrial fibrillation (NVAF). Major bleeding is its most common adverse event which is of great concern. However, other types of adverse events such as esophagitis, esophageal ulcer, exanthem and pustular eruptions were reported increasingly in recent years. We present a case of immune hemolytic anemia (IHA) due to dabigatran use in a 72-year-old male with NVAF. This new and rare reported type of adverse event associated with dabigatran suggests that dabigatran may be a new cause of drug-induced immune hemolytic anemia (DIIHI).


Asunto(s)
Anemia Hemolítica Autoinmune/inducido químicamente , Antitrombinas/efectos adversos , Dabigatrán/efectos adversos , Anciano , Anemia Hemolítica Autoinmune/diagnóstico , Anemia Hemolítica Autoinmune/tratamiento farmacológico , Anemia Hemolítica Autoinmune/inmunología , Antitrombinas/administración & dosificación , Enfermedad Crónica , Dabigatrán/administración & dosificación , Progresión de la Enfermedad , Sustitución de Medicamentos , Glucocorticoides/administración & dosificación , Humanos , Inmunoglobulinas Intravenosas/administración & dosificación , Masculino , Resultado del Tratamiento , Warfarina/administración & dosificación
6.
Zhonghua Xue Ye Xue Za Zhi ; 41(5): 412-416, 2020 May 14.
Artículo en Chino | MEDLINE | ID: mdl-32536139

RESUMEN

Objective: To explore the efficacy and safety of ibrutinib treatment for relapsed/refractory (R/R) primary autoimmune hemolytic anemia (AIHA) . Methods: Two cases of primary AIHA with relapse events were refractory to glucocorticoid, anti-CD20 monoclonal antibody, immunosuppressive drugs, and splenectomy (case 1 only) . Ibrutinib treatment was administered at an initial dose of 280 mg/d (420 mg/d for case 1 from the 3rd to 8th week) . Results: Both patients achieved transfusion independence and HGB>20 g/L above baseline after 2 weeks (partial response) . For case 1, HGB concentration restored to 113 g/L but with incomplete hemolysis recovery after 10 weeks; HGB reached the level of 118 g/L, also with incomplete hemolysis recovery, after 6 weeks in case 2. They all acquired complete response with incomplete hemolysis recovery (CRi) . The responses sustained 14 weeks and 10 weeks after follow-up at 16 weeks and 12 weeks, respectively. During the treatment, hematologic and nonhematologic toxicity is mild and acceptable. Conclusion: Ibrutinib alone is effective for the 2 R/R primary AIHA cases. We need further clinical trial to identify its efficacy and safety.


Asunto(s)
Anemia Hemolítica Autoinmune , Pirazoles/uso terapéutico , Pirimidinas/uso terapéutico , Anemia Hemolítica Autoinmune/tratamiento farmacológico , Anticuerpos Monoclonales de Origen Murino , Humanos , Proyectos Piloto , Rituximab
9.
Med. clín (Ed. impr.) ; 154(9): 331-337, mayo 2020. tab
Artículo en Español | IBECS | ID: ibc-193212

RESUMEN

INTRODUCCIÓN: Las anemias hemolíticas autoinmunes (AHAI) son enfermedades poco frecuentes y heterogéneas en su fisiopatología y comportamiento clínico, siendo el manejo de las mismas fundamentalmente empírico. PACIENTES Y MÉTODOS: Realizamos un estudio observacional, retrospectivo y multicéntrico de 93 pacientes diagnosticados de AHAI en 9 hospitales españoles entre 1987 y 2017, con una mediana de seguimiento de 28 meses. RESULTADOS: Mediana de edad de 67 años; un 85% de AHAI por anticuerpos calientes y un 64% AHAI primarias. Los valores de hemoglobina más bajos al diagnóstico se relacionaron con edad<45 años y el tipo serológico IgG+C. Un 92% recibieron tratamiento de primera línea, un 54% de segunda línea y un 27% de tercera línea. Las AHAI calientes fueron tratadas en primera línea con esteroides, con respuestas globales del 83% y completas del 58%. El rituximab en monoterapia o asociado a esteroides se administró a 34 pacientes con respuestas globales cercanas al 100% (respuestas completas 40-60%), relegando la esplenectomía a tercera línea. El tratamiento inmunosupresor se administró en pacientes con enfermedades autoinmunes o en dependientes de corticoides. DISCUSIÓN: Encontramos altas tasas de respuesta a esteroides, con tratamientos muy prolongados que provocan efectos secundarios y corticodependencia en un tercio de los pacientes. La asociación de esteroides con rituximab en primera línea podría estar indicada en pacientes con bajos niveles de hemoglobina y tipo serológico IgG+C. Las altas tasas de recaída hacen necesario el desarrollo de estudios aleatorizados con nuevos fármacos o la asociación con los ya existentes, que permitan mayor duración de las respuestas y con menores efectos secundarios


INTRODUCTION: Autoimmune haemolytic anaemia (AIHA) is an infrequent and heterogeneous disease in its pathophysiology and clinical behaviour, therefore it is generally managed empirically. PATIENTS AND METHODS: We conducted an observational, retrospective and multicentre study of 93 patients diagnosed with AHAI in 9 Spanish hospitals between 1987 and 2017, with a median follow-up of 28 months. RESULTS: Median age of 67 years; 85% AHAI for hot antibodies and 64% primary AHAI. The lowest haemoglobin values at diagnosis related to patients under 45 years of age and serological type IgG+C. Of the patients, 92% received first line treatment, 54% second line, and 27% third line. The warm AHAI were treated in first line with steroids, with overall responses of 83% and complete of 58%. Rituximab in monotherapy or in association with steroids was administered to 34 patients with overall responses close to 100% (complete responses 40-60%), relegating splenectomy to the third line. The immunosuppressive treatment was administered in patients with autoimmune diseases or in corticoid-dependent patients. DISCUSSION: We found high rates of response to steroids, with very prolonged treatments that cause side effects and corticoid dependence in a third of patients. The combination of steroids with rituximab in the first line, could be indicated in patients with low levels of haemoglobin and serological type IgG+C. The high relapse rates make necessary the development of randomised studies with new drugs or the combination with existing ones, which allow longer response times and with fewer side effects


Asunto(s)
Humanos , Masculino , Femenino , Lactante , Preescolar , Niño , Adolescente , Adulto Joven , Adulto , Persona de Mediana Edad , Anciano , Anciano de 80 o más Años , Anemia Hemolítica Autoinmune/diagnóstico , Anemia Hemolítica Autoinmune/tratamiento farmacológico , Inmunosupresores , Resultado del Tratamiento , Anemia Hemolítica Autoinmune/fisiopatología , Estudios Retrospectivos , Enfermedades Autoinmunes/tratamiento farmacológico , Efectos Colaterales y Reacciones Adversas Relacionados con Medicamentos , Glucocorticoides/administración & dosificación
10.
BMC Cardiovasc Disord ; 20(1): 106, 2020 03 04.
Artículo en Inglés | MEDLINE | ID: mdl-32131747

RESUMEN

BACKGROUND: PE (pulmonary embolism) is a life-threatening complication rarely seen in the AIHA (autoimmune haemolytic anaemia) patients. Herein we reported a rare and serious AIHA-PE patient characterised by extensive peripheral pulmonary embolism on CTPA. CASE PRESENTATION: A 59-year-old woman presented to our ED (emergency department) complaining of acute chest pain and dyspnea. During her presentation in ED she experienced a sudden syncope and soon developed CA (cardiac arrest). Laboratory studies showed a increase of CK-MB,troponin T,myoglobin and D-dimer. Computed tomography pulmonary angiography (CTPA) showed no large central or segment pulmonary emboli but increased RV (right ventricle)size,enlarged main pulmonary artery and invisible peripheral pulmonary artery. She was diagnosed with acute PE and alteplase was delivered intravenously. After thrombolytic therapy she remained hypotension and developed worsening anaemia. Detailed examination for anaemia revealed AIHA. She was discharged in a stable condition after 5 weeks with methylprednisolone and warfarin. Hb, D-dimer and transthoracic echocardiography showed complete recovery at 3-months follow up. CONCLUSION: PE attributed to AIHA is characterized by subsegment and distal pulmonary artery embolism which is easily neglected but always life-threatening. This case also highlights the PE as a secondary diagnosis should be evaluated comprehensively in order to identify the underlying pathogenesis.


Asunto(s)
Anemia Hemolítica Autoinmune/complicaciones , Embolia Pulmonar/etiología , Enfermedad Aguda , Anemia Hemolítica Autoinmune/diagnóstico , Anemia Hemolítica Autoinmune/tratamiento farmacológico , Anticoagulantes/uso terapéutico , Femenino , Fibrinolíticos/administración & dosificación , Glucocorticoides/uso terapéutico , Humanos , Metilprednisolona/uso terapéutico , Persona de Mediana Edad , Embolia Pulmonar/diagnóstico por imagen , Embolia Pulmonar/tratamiento farmacológico , Choque Cardiogénico/etiología , Síncope/etiología , Terapia Trombolítica , Activador de Tejido Plasminógeno/administración & dosificación , Resultado del Tratamiento , Warfarina/uso terapéutico
12.
J Immunother Cancer ; 8(1)2020 03.
Artículo en Inglés | MEDLINE | ID: mdl-32169870

RESUMEN

BACKGROUND: The combination of the immune checkpoint inhibitors (ICIs) ipilimumab and nivolumab is a mainstay of treatment for selected patients with metastatic melanoma. This combination also results in more frequent immune-related adverse events (irAEs) than either ICI alone. These irAEs can be severe and their pathogenesis is poorly understood. CASE PRESENTATION: We report a case of a woman with metastatic melanoma, treated with combined ipilimumab and nivolumab, who developed severe anaemia. While initial workup revealed autoimmune haemolytic anaemia, the anaemia persisted despite corticosteroids and paradoxical reticulocytopenia was observed. Bone marrow biopsy demonstrated a CD8+ T cell-mediated destruction of the red cell precursors implying concurrent pure red cell aplasia. Both processes resolved after the addition of cyclosporine A. CONCLUSIONS: This report describes a rare case of two concurrent mechanisms of haematological irAE in a patient treated with combined ICI therapy. Successful treatment resulted only after the second underlying mechanism of toxicity was uncovered. Prompt recognition of these unusual presentations of rare irAEs is now key to effective irAE management.


Asunto(s)
Anemia Hemolítica Autoinmune/inmunología , Protocolos de Quimioterapia Combinada Antineoplásica/efectos adversos , Melanoma/tratamiento farmacológico , Adulto , Anemia Hemolítica Autoinmune/inducido químicamente , Anemia Hemolítica Autoinmune/tratamiento farmacológico , Anemia Hemolítica Autoinmune/patología , Antifúngicos/administración & dosificación , Protocolos de Quimioterapia Combinada Antineoplásica/uso terapéutico , Ciclosporina/administración & dosificación , Femenino , Humanos , Ipilimumab/administración & dosificación , Ipilimumab/efectos adversos , Melanoma/inmunología , Melanoma/patología , Nivolumab/administración & dosificación , Nivolumab/efectos adversos , Pronóstico
13.
Am J Case Rep ; 21: e920760, 2020 Feb 07.
Artículo en Inglés | MEDLINE | ID: mdl-32029698

RESUMEN

BACKGROUND Evans syndrome is characterized by 'warm' autoimmune hemolytic anemia and autoimmune thrombocytopenia, and is more common in the pediatric population than in adults. Evans syndrome is often associated with underlying autoimmune disease, connective tissue disease, immune deficiency disorders, lymphoproliferative disorders, or malignancy of the immune system. A case is presented of acute kidney injury due to hemoglobin cast nephropathy in an adult man with Evans syndrome. CASE REPORT A 60-year-old man was diagnosed with Evans syndrome, which was complicated by acute renal failure that required treatment with hemodialysis. Laboratory tests and renal histology confirmed a diagnosis of hemolysis-associated hemoglobin cast nephropathy. CONCLUSIONS The diagnosis of Evans syndrome is important as it may be associated with underlying hematological and immunological disorders. Although rare, hemoglobin cast nephropathy due to hemolysis can be a cause of acute kidney injury in patients with Evans syndrome.


Asunto(s)
Lesión Renal Aguda/tratamiento farmacológico , Lesión Renal Aguda/etiología , Anemia Hemolítica Autoinmune/complicaciones , Anemia Hemolítica Autoinmune/tratamiento farmacológico , Hemólisis/efectos de los fármacos , Trombocitopenia/complicaciones , Trombocitopenia/tratamiento farmacológico , Antineoplásicos Inmunológicos/uso terapéutico , Glucocorticoides/uso terapéutico , Humanos , Masculino , Persona de Mediana Edad , Prednisona/uso terapéutico , Rituximab/uso terapéutico
14.
Acta Haematol ; 143(5): 491-495, 2020.
Artículo en Inglés | MEDLINE | ID: mdl-31962320

RESUMEN

Thymoma is an uncommon neoplasia derived from the epithelial cells of the thymus, which leads to immune dysregulation and is associated with a series of autoimmune diseases. However, the concurrence of these disease entities is rare, and the exact mechanisms of these diseases are still unclear. We have admitted several cases who were diagnosed with thymoma, autoimmune haemolytic anaemia, and pure red cell aplasia. These cases were the first to report the concurrence of these three disorders. After thymectomy, anaemia improved, haemolytic cells decreased, and haemoglobin was normalized.


Asunto(s)
Anemia Hemolítica Autoinmune/diagnóstico , Aplasia Pura de Células Rojas/diagnóstico , Neoplasias del Timo/diagnóstico , Corticoesteroides/uso terapéutico , Adulto , Anciano , Anemia Hemolítica Autoinmune/tratamiento farmacológico , Anemia Hemolítica Autoinmune/etiología , Médula Ósea/patología , Transfusión de Eritrocitos , Femenino , Hemoglobinas/análisis , Humanos , Masculino , Persona de Mediana Edad , Aplasia Pura de Células Rojas/terapia , Tórax/diagnóstico por imagen , Neoplasias del Timo/complicaciones , Tomografía Computarizada por Rayos X
17.
Acta Haematol ; 143(3): 244-249, 2020.
Artículo en Inglés | MEDLINE | ID: mdl-31665725

RESUMEN

The combination of rituximab, cyclophosphamide, and dexamethasone (RCD) is highly effective in the treatment of warm autoimmune hemolytic anemia (WAIHA) associated with chronic lymphocytic leukemia (CLL). We treated a cohort of patients with relapsed/refractory WAIHA, without CLL, with RCD. The primary objective was to evaluate the overall response (OR) of RCD therapy. Complete response (CR) was defined as a hemoglobin (Hgb) ≥12 g/dL. Partial response (PR) was defined as Hgb 10-11.9 g/dL or ≥2 g/dL increase in Hgb. Sustained response was defined as Hgb ≥10 g/dL with no treatment changes. A total of 16 patients with relapsed/refractory WAIHA received RCD (7 primary WAIHA, 9 secondary WAIHA) for a median of 4 cycles (range: 2-6). The median pretreatment Hgb was 10.0 g/dL (range: 4.3-12.2). The median best Hgb achieved was 12.5 g/dL (range: 10.6-15.1) with a median of 2 cycles until best Hgb response. The OR was 94% (11 CR, 4 PR). Two immunocompromised patients were admitted for infections during RCD treatment. There were no deaths during the treatment or follow-up period. Following a response to RCD, 4 patients received noncorticosteroid immune modulation therapy and 4 patients continued on corticosteroid therapy. Seven patients received no additional treatment.


Asunto(s)
Anemia Hemolítica Autoinmune/tratamiento farmacológico , Ciclofosfamida/uso terapéutico , Dexametasona/uso terapéutico , Rituximab/uso terapéutico , Corticoesteroides/uso terapéutico , Adulto , Anciano , Anemia Hemolítica Autoinmune/sangre , Anemia Hemolítica Autoinmune/cirugía , Terapia Combinada , Ciclofosfamida/administración & dosificación , Dexametasona/administración & dosificación , Quimioterapia Combinada , Femenino , Estudios de Seguimiento , Hemoglobinas/análisis , Humanos , Factores Inmunológicos/uso terapéutico , Estimación de Kaplan-Meier , Masculino , Persona de Mediana Edad , Recurrencia , Estudios Retrospectivos , Rituximab/administración & dosificación , Esplenectomía , Resultado del Tratamiento
19.
Transfus Med Rev ; 33(4): 256-265, 2019 10.
Artículo en Inglés | MEDLINE | ID: mdl-31703946

RESUMEN

Dysregulation of the complement system underlies the pathophysiology of many diseases. Renewed interest in complement occurred with the recognition that its therapeutic inhibition was possible. Terminal complement blockade with the anti-C5 monoclonal antibody eculizumab significantly changed management and clinical outcomes of patients with paroxysmal nocturnal hemoglobinuria, and served as a proof of concept for other complement-mediated diseases. Eculizumab is also approved for atypical hemolytic uremic syndrome and myasthenia gravis. Multiple new disease indications have been identified, and novel complement inhibitors are in various stages of development, with several currently in human trials. Beyond C5, these new drugs block proximal complement, pathway-specific targets, convertase activity, and anaphylatoxin function. Though monoclonal antibodies are still common, peptides, RNAi, and small molecule inhibitors provide the opportunity for different administration routes and schedules. Several challenges still exist or will soon present themselves, including mitigation of infection risk, effective monitoring strategies, and how to choose between therapeutics when more than one is available. In this review, we will describe the lessons learned from the "eculizumab era," present many of the novel therapeutics currently or soon to be in trials, and highlight some of the challenges that will require attention as the field progresses.


Asunto(s)
Anticuerpos Monoclonales Humanizados/uso terapéutico , Inactivadores del Complemento/uso terapéutico , Anemia Hemolítica Autoinmune/tratamiento farmacológico , Anticuerpos Monoclonales Humanizados/administración & dosificación , Síndrome Hemolítico Urémico Atípico/tratamiento farmacológico , Proteínas del Sistema Complemento/inmunología , Proteínas del Sistema Complemento/fisiología , Hemoglobinuria Paroxística/tratamiento farmacológico , Humanos , Miastenia Gravis/tratamiento farmacológico , Microangiopatías Trombóticas/tratamiento farmacológico
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