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1.
J Assoc Physicians India ; 71(10): 94-95, 2023 Oct.
Artigo em Inglês | MEDLINE | ID: mdl-38716532

RESUMO

Anemia is a common complication of chronic kidney disease (CKD) that has been classically attributed to inadequate production of endogenous erythropoietin.1 Though there are many other common causes of refractory anemia in CKD like iron deficiency, vitamin B12, and folic acid deficiency, noncompliance to dialysis and erythropoietin therapy rare causes like blood loss, bone marrow failure, infections causing aplastic crisis like CMV, parvovirus B19 should be ruled out. Parvovirus has an extreme tropism for erythroid cells and is an uncommon cause of anemia in patients with CKD on maintenance dialysis (MHD) and on erythropoietin.2 Here we are reporting a rare case of refractory anemia in a patient of CKD on MHD secondary to parvovirus-related aplastic crisis. How to cite this article: Gade K, Londhe C, Pednekar S, et al. A Case of Refractory Anemia in Patient of Chronic Kidney Disease and the Challenges in its Management. J Assoc Physicians India 2023;71(10):94-95.


Assuntos
Anemia Refratária , Insuficiência Renal Crônica , Humanos , Insuficiência Renal Crônica/complicações , Insuficiência Renal Crônica/terapia , Anemia Refratária/etiologia , Anemia Refratária/terapia , Anemia Refratária/diagnóstico , Anemia Refratária/complicações , Diálise Renal , Masculino , Infecções por Parvoviridae/complicações , Infecções por Parvoviridae/diagnóstico , Eritropoetina/uso terapêutico , Anemia Aplástica/complicações , Anemia Aplástica/terapia , Pessoa de Meia-Idade
2.
United European Gastroenterol J ; 7(2): 217-224, 2019 03.
Artigo em Inglês | MEDLINE | ID: mdl-31080606

RESUMO

Background: Gastric antral vascular ectasia (GAVE) is a rare cause of gastrointestinal bleeding, often causing iron deficiency anaemia. Previous studies have looked at the management of this with argon plasma coagulation, laser therapy and endoscopic band ligation. Methods: This was a single-centre prospective study to evaluate the efficacy and safety of radiofrequency ablation (RFA) in patients with GAVE with persistent anaemia refractory to at least one session of first-line endoscopic therapy. Patients were treated with a through-the-scope (TTS) radiofrequency catheter at two endoscopic sessions six weeks apart. The primary outcome was change in haemoglobin at six months posttreatment. The secondary outcomes were reduction in blood or iron requirements, endoscopic surface area regression and complications. Results: Twenty patients were treated. The mean change in haemoglobin at six months was +12.6 g/l (95% confidence interval 11.7-24.3 g/l), paired t test p < 0.001. At six months, three of 14 individuals who had required blood transfusions had ongoing blood transfusions and five of 17 who had required iron had ongoing iron needs. Surface area regression was scored as 74% ± 25% but no correlation was seen between this and other outcomes. Three of 20 patients experienced pain which was managed with oral analgesia. Of the 14 patients who had reached 12-month follow-up, three required retreatment (21%). Discussion: This small study suggests that RFA is a safe and effective treatment for GAVE. Our study uses the TTS catheter compared to other studies, and demonstrates prolonged improvement in haemoglobin and reduction in blood and iron requirements with a novel assessment of surface area regression.


Assuntos
Anemia Refratária/etiologia , Anemia Refratária/terapia , Ectasia Vascular Gástrica Antral/complicações , Ablação por Radiofrequência , Idoso , Idoso de 80 Anos ou mais , Anemia Refratária/diagnóstico , Feminino , Ectasia Vascular Gástrica Antral/diagnóstico , Hemorragia Gastrointestinal/complicações , Hemorragia Gastrointestinal/etiologia , Gastroscopia , Humanos , Masculino , Pessoa de Meia-Idade , Ablação por Radiofrequência/métodos , Fatores de Tempo , Resultado do Tratamento
3.
Am J Hematol ; 94(4): 475-488, 2019 04.
Artigo em Inglês | MEDLINE | ID: mdl-30618061

RESUMO

DISEASE OVERVIEW: Ring sideroblasts (RS) are erythroid precursors with abnormal perinuclear mitochondrial iron accumulation. Two myeloid neoplasms defined by the presence of RS, include refractory anemia with ring sideroblasts (RARS), now classified under myelodysplastic syndromes with RS (MDS-RS) and RARS with thrombocytosis (RARS-T); now called myelodysplastic/myeloproliferative neoplasm with RS and thrombocytosis (MDS/MPN-RS-T). DIAGNOSIS: MDS-RS is a lower-risk MDS, with single or multilineage dysplasia (SLD/MLD), <5% bone marrow (BM) blasts and ≥ 15% BM RS (≥5% in the presence of SF3B1 mutations). MDS/MPN-RS-T, now a formal entity in the MDS/MPN overlap syndromes, has diagnostic features of MDS-RS-SLD, along with a platelet count ≥450 × 10(9)/L and large atypical megakaryocytes. MUTATIONS AND KARYOTYPE: Mutations in SF3B1 are seen in ≥80% of patients with MDS-RS-SLD and MDS/MPN-RS-T, and strongly correlate with the presence of BM RS; MDS/MPN-RS-T patients also demonstrate JAK2V617F, ASXL1, DNMT3A, SETBP1, and TET2 mutations. Cytogenetic abnormalities are uncommon in both. RISK STRATIFICATION: Most patients with MDS-RS-SLD are stratified into lower-risk groups by the revised-IPSS. Disease outcome in MDS/MPN-RS-T is better than that of MDS-RS-SLD, but worse than that of essential thrombocythemia. Both diseases have a low risk of leukemic transformation. TREATMENT: Anemia and iron overload are complications seen in both and are managed similar to lower-risk MDS and MPN. The advent of luspatercept, a first-in-class erythroid maturation agent will tremendously boost the ability to manage anemia. Aspirin therapy is reasonable in MDS/MPN-RS-T, especially in the presence of JAK2V617F, but the value of platelet-lowering drugs remains uncertain.


Assuntos
Anemia Refratária , Mutação , Anemia Refratária/sangue , Anemia Refratária/diagnóstico , Anemia Refratária/genética , Anemia Refratária/terapia , Anemia Sideroblástica/sangue , Anemia Sideroblástica/diagnóstico , Anemia Sideroblástica/genética , Anemia Sideroblástica/terapia , Eritroblastos/metabolismo , Feminino , Humanos , Sobrecarga de Ferro/sangue , Sobrecarga de Ferro/diagnóstico , Sobrecarga de Ferro/genética , Sobrecarga de Ferro/terapia , Masculino , Doenças Mieloproliferativas-Mielodisplásicas , Trombocitose/sangue , Trombocitose/diagnóstico , Trombocitose/genética , Trombocitose/terapia
4.
Mayo Clin Proc ; 93(2): 155-166, 2018 02.
Artigo em Inglês | MEDLINE | ID: mdl-29395350

RESUMO

OBJECTIVE: To present a multiyear clinical experience with intravenous bevacizumab for the management of severe gastrointestinal bleeding and/or epistaxis in patients with hereditary hemorrhagic telangiectasia (HHT). PATIENTS AND METHODS: All patients treated with intravenous bevacizumab for severe hereditary hemorrhagic telangiectasia-related bleeding from June 1, 2013, through January 31, 2017, were included in this report. Severity of epistaxis (determined using the Epistaxis Severity Score questionnaire); hemoglobin, iron, and ferritin levels; and quality of life data were collected serially in all patients. RESULTS: Intravenous bevacizumab was administered to 34 patients using a standardized treatment protocol. Anemia was primarily related to severe epistaxis (n=15, 44%), severe gastrointestinal bleeding (n=4, 12%), or both (n=15, 44%), with a median baseline hemoglobin level of 9.1 g/dL (range, 8.3-10.5 gm/dL; to convert to mmol/L, multiply by 0.62). Red blood cell (RBC) transfusions had been administered to 28 patients (82%). Of these, 16 patients (47%) were RBC transfusion dependent and had received a median of 75 RBC transfusions (range, 4->500 RBC units) before bevacizumab initiation. The median length of follow-up was 17.6 months from the beginning of bevacizumab treatment (range, 3-42.5 months). There was a significant reduction in epistaxis severity scores (P<.001) and RBC transfusion requirements (P=.007) after completion of the initial bevacizumab treatment cycle. New-onset or worsened hypertension was noted in 4 patients, with 1 patient experiencing hypertensive urgency with a temporary decline in renal function. CONCLUSION: Intravenous bevacizumab is an effective treatment option for patients with severe anemia related to epistaxis and/or gastrointestinal bleeding. Further studies are needed to establish a dose-response relationship as well as clinical, genetic, and biomarker predictors of response.


Assuntos
Anemia Refratária , Bevacizumab/administração & dosagem , Epistaxe , Hemorragia Gastrointestinal , Qualidade de Vida , Telangiectasia Hemorrágica Hereditária , Administração Intravenosa , Idoso , Anemia Refratária/diagnóstico , Anemia Refratária/etiologia , Anemia Refratária/terapia , Inibidores da Angiogênese/administração & dosagem , Epistaxe/diagnóstico , Epistaxe/etiologia , Epistaxe/terapia , Feminino , Ferritinas/sangue , Hemorragia Gastrointestinal/diagnóstico , Hemorragia Gastrointestinal/etiologia , Hemorragia Gastrointestinal/terapia , Humanos , Masculino , Pessoa de Meia-Idade , Minnesota , Estudos Retrospectivos , Índice de Gravidade de Doença , Telangiectasia Hemorrágica Hereditária/sangue , Telangiectasia Hemorrágica Hereditária/complicações , Telangiectasia Hemorrágica Hereditária/diagnóstico , Telangiectasia Hemorrágica Hereditária/psicologia , Resultado do Tratamento
5.
J Gastrointestin Liver Dis ; 26(4): 369-374, 2017 Dec.
Artigo em Inglês | MEDLINE | ID: mdl-29253051

RESUMO

BACKGROUND AND AIMS: Patients with small bowel angioectasias (SBAs) can be difficult to manage as they are generally elderly with multiple co-morbidities. Angioectasias are multiple and tend to recur. Argon plasma coagulation (APC), despite being a commonly used method to treat these patients has an associated persistent rate of re-bleeding necessitating additional treatment to manage these patients. METHODS: All patients with refractory iron deficiency anaemia secondary to SBAs were retrospectively subdivided into two groups. Patients in group 1 were managed with double balloon enteroscopy (DBE) and APC alone and those in group 2 received Lanreotide in addition to DBE and APC. RESULTS: A total of 49 patients were included in this study: group 1: 37 patients (75.5%), group 2: 12 patients (24.5%). All had significant comorbidities and the mean duration of anaemia was 114.3, SD 307.0 months. Significant improvements in haemoglobin (Hb) (11g/L vs 3.2g/L p=0.043), transfusion requirements per month (0.8 vs 4.7 p=0.052) and mean bleeding episodes (1.08 vs 2.6 p=0.032) were demonstrated in group 2 when compared to group 1. One patient developed symptomatic gallstone disease and one patient stopped Lanreotide due to a lack of response. CONCLUSIONS: This is the first study comparing endotherapy to a combination of endotherapy and pharmacotherapy. It shows a significantly better outcome in patients receiving a combination of endotherapy and Lanreotide. Lanreotide can be a safe additional treatment in patients not responding to APC alone.


Assuntos
Anemia Refratária/terapia , Angiodisplasia/terapia , Coagulação com Plasma de Argônio/métodos , Intestino Delgado , Idoso , Idoso de 80 Anos ou mais , Anemia Refratária/etiologia , Angiodisplasia/complicações , Transfusão de Sangue , Endoscopia por Cápsula , Terapia Combinada , Enteroscopia de Duplo Balão/métodos , Feminino , Hemorragia Gastrointestinal/etiologia , Hemorragia Gastrointestinal/terapia , Hemoglobinas/metabolismo , Humanos , Masculino , Pessoa de Meia-Idade , Peptídeos Cíclicos/uso terapêutico , Recidiva , Estudos Retrospectivos , Somatostatina/análogos & derivados , Somatostatina/uso terapêutico
7.
Curr Probl Cancer ; 41(6): 413-418, 2017.
Artigo em Inglês | MEDLINE | ID: mdl-29129340

RESUMO

Multiple primary malignant neoplasms (MPMNs) are rare malignant neoplasms that simultaneously or successively occur in the same patient as 2 or more primary malignancies. Currently, an increasing number of cases are being reported. In general, MPMNs more commonly occur as 2 solid tumors or 2 hematological malignancies. Cases of MPMN that involve a solid tumor and a hematological malignancy are rare. Here, we report a case of synchronous colorectal cancer (CRC) and multiple myeloma (MM) with chest wall involvement. After reviewing the literature, we believe that there may be a distinct syndrome involving CRC and MM. The patient in our case study suffered refractory anemia following surgery and 2 cycles of chemotherapy. Initially, the anemia was considered to be a common manifestation of CRC in this patient. Interestingly, although he received a blood transfusion, his hemoglobin levels remained low. He later developed hematuria, proteinuria, multiple osteoporosis in the costal bones, and thrombocytopenia. These new symptoms drew our attention, and we considered a diagnosis of synchronous primary CRC and MM, with the anemia as a symptom of MM. Based on the results of a bone marrow aspirate, MM was confirmed. Therefore, when CRC is associated with refractory anemia, we should not only assume that anemia is a classical symptom of CRC, a result of chronic blood loss, nutritional deficiencies, or myelosuppression due to chemotherapy, but we should also consider that it may reflect the possibility of a coexisting hematologic malignancy. As the treatment of these 2 malignancies is different, early diagnosis and treatment based on definitive diagnosis as early as possible will be beneficial to overall prognosis.


Assuntos
Adenocarcinoma/terapia , Anemia Refratária/terapia , Protocolos de Quimioterapia Combinada Antineoplásica/efeitos adversos , Neoplasias Colorretais/terapia , Mieloma Múltiplo/terapia , Neoplasias Primárias Múltiplas/terapia , Adenocarcinoma/sangue , Adenocarcinoma/diagnóstico , Adenocarcinoma/patologia , Anemia Refratária/sangue , Anemia Refratária/diagnóstico , Anemia Refratária/etiologia , Biópsia , Quimioterapia Adjuvante/efeitos adversos , Colectomia , Colonoscopia , Neoplasias Colorretais/sangue , Neoplasias Colorretais/diagnóstico , Neoplasias Colorretais/patologia , Detecção Precoce de Câncer , Fluoruracila/efeitos adversos , Humanos , Leucovorina/efeitos adversos , Masculino , Pessoa de Meia-Idade , Mieloma Múltiplo/sangue , Mieloma Múltiplo/diagnóstico , Mieloma Múltiplo/patologia , Neoplasias Primárias Múltiplas/sangue , Neoplasias Primárias Múltiplas/diagnóstico , Neoplasias Primárias Múltiplas/patologia , Compostos Organoplatínicos/efeitos adversos , Prognóstico , Síndrome , Parede Torácica/patologia , Tomografia Computadorizada por Raios X
8.
Int J Hematol ; 105(6): 720-731, 2017 Jun.
Artigo em Inglês | MEDLINE | ID: mdl-28466384

RESUMO

Spliceosomal mutations, especially mutations in SF3B1, are frequently (>80%) identified in patients with refractory anemia with ringed sideroblasts (RARS) and myelodysplastic/myeloproliferative neoplasms with ringed sideroblasts and thrombocytosis (MDS/MPN-RS-T; previously known as RARS-T), and SF3B1 mutations have a high positive predictive value for disease phenotype with ringed sideroblasts. These observations suggest that SF3B1 mutations play important roles in the pathogenesis of these disorders and formation of ringed sideroblasts. Here we will review recent insights into the molecular mechanisms of mis-splicing caused by mutant SF3B1 and the pathogenesis of RSs in the context of congenital sideroblastic anemia as well as RARS with SF3B1 mutations. We will also discuss therapy of SF3B1 mutant MDS, including novel approaches.


Assuntos
Anemia Refratária , Anemia Sideroblástica , Mutação , Doenças Mieloproliferativas-Mielodisplásicas , Proteínas de Neoplasias , Fosfoproteínas , Fatores de Processamento de RNA , Trombocitose , Anemia Refratária/genética , Anemia Refratária/metabolismo , Anemia Refratária/terapia , Anemia Sideroblástica/genética , Anemia Sideroblástica/metabolismo , Anemia Sideroblástica/terapia , Humanos , Doenças Mieloproliferativas-Mielodisplásicas/genética , Doenças Mieloproliferativas-Mielodisplásicas/metabolismo , Doenças Mieloproliferativas-Mielodisplásicas/terapia , Proteínas de Neoplasias/genética , Proteínas de Neoplasias/metabolismo , Fosfoproteínas/genética , Fosfoproteínas/metabolismo , Splicing de RNA/genética , Fatores de Processamento de RNA/genética , Fatores de Processamento de RNA/metabolismo , RNA Neoplásico/genética , RNA Neoplásico/metabolismo , Trombocitose/genética , Trombocitose/metabolismo , Trombocitose/patologia
9.
Hematology Am Soc Hematol Educ Program ; 2016(1): 83-89, 2016 Dec 02.
Artigo em Inglês | MEDLINE | ID: mdl-27913466

RESUMO

Nontransplant therapeutic options for acquired and constitutional aplastic anemia have significantly expanded during the last 5 years. In the future, transplant may be required less frequently. That trilineage hematologic responses could be achieved with the single agent eltrombopag in refractory aplastic anemia promotes new interest in growth factors after years of failed trials using other growth factor agents. Preliminary results adding eltrombopag to immunosuppressive therapy are promising, but long-term follow-up data evaluating clonal evolution rates are required before promoting its standard use in treatment-naive disease. Danazol, which is traditionally less preferred for treating cytopenias, is capable of preventing telomere attrition associated with hematologic responses in constitutional bone marrow failure resulting from telomere disease.


Assuntos
Anemia Aplástica/terapia , Anemia Refratária/terapia , Benzoatos/uso terapêutico , Danazol/uso terapêutico , Hidrazinas/uso terapêutico , Terapia de Imunossupressão/métodos , Pirazóis/uso terapêutico , Humanos
11.
Transfus Apher Sci ; 55(1): 105-8, 2016 Aug.
Artigo em Inglês | MEDLINE | ID: mdl-27102761

RESUMO

Hemophagocytic lymphohistiocytosis (HLH) is a life-threatening derangement of the immune system in which host macrophages phagocytose the patient's own blood cells. Herein, we present the case of a patient with HLH and associated refractory anemia who developed rapid iron deposition in the liver after transfusion of sixteen units of packed red blood cells (RBCs). Before transfusion, neither a liver biopsy nor computed tomography scan demonstrated iron deposition in the organ parenchyma. After receiving sixteen units of packed RBCs, liver iron concentration rose to 6.7 mg/g dry weight, which is highly unusual in other diseases requiring transfusional support.


Assuntos
Anemia Refratária/terapia , Transfusão de Eritrócitos/efeitos adversos , Sobrecarga de Ferro/etiologia , Linfo-Histiocitose Hemofagocítica/terapia , Adulto , Anemia Refratária/diagnóstico por imagem , Anemia Refratária/metabolismo , Feminino , Humanos , Sobrecarga de Ferro/diagnóstico por imagem , Linfo-Histiocitose Hemofagocítica/diagnóstico por imagem , Linfo-Histiocitose Hemofagocítica/metabolismo
12.
Artigo em Inglês | MEDLINE | ID: mdl-26637696

RESUMO

The sideroblastic anemias are a heterogeneous group of inherited and acquired disorders characterized by the presence of ring sideroblasts in the bone marrow. X-linked sideroblastic anemia (XLSA) is caused by germline mutations in ALAS2. Hemizygous males have a hypochromic microcytic anemia, which is generally mild to moderate and is caused by defective heme synthesis and ineffective erythropoiesis. XLSA is a typical iron-loading anemia; although most patients are responsive to pyridoxine, treatment of iron overload is also important in the management of these patients. Autosomal recessive sideroblastic anemia attributable to mutations in SLC25A38, a member of the mitochondrial carrier family, is a severe disease: patients present in infancy with microcytic anemia, which soon becomes transfusion dependent. Conservative therapy includes regular red cell transfusion and iron chelation, whereas allogenic stem cell transplantation represents the only curative treatment. Refractory anemia with ring sideroblasts (RARS) is a myelodysplastic syndrome characterized mainly by anemia attributable to ineffective erythropoiesis. The clinical course of RARS is generally indolent, but there is a tendency to worsening of anemia over time, so that most patients become transfusion dependent in the long run. More than 90% of these patients carry somatic mutations in SF3B1, a gene encoding a core component of the RNA splicing machinery. These mutations cause misrecognition of 3' splice sites in downstream genes, resulting in truncated gene products and/or decreased expression attributable to nonsense-mediated RNA decay; this explains the multifactorial pathogenesis of RARS. Variants of RARS include refractory cytopenia with multilineage dysplasia and ring sideroblasts, and RARS associated with marked thrombocytosis; these variants involve additional genetic lesions. Inhibitors of molecules of the transforming growth factor-ß superfamily have been shown recently to target ineffective erythropoiesis and ameliorate anemia both in animal models of myelodysplastic syndrome and in RARS patients.


Assuntos
Processamento Alternativo , Anemia Sideroblástica/diagnóstico , Anemia Sideroblástica/terapia , Heme/biossíntese , Anemia Refratária/diagnóstico , Anemia Refratária/genética , Anemia Refratária/metabolismo , Anemia Refratária/terapia , Anemia Sideroblástica/genética , Anemia Sideroblástica/metabolismo , Animais , Modelos Animais de Doenças , Transfusão de Eritrócitos , Eritropoese , Feminino , Hemizigoto , Humanos , Masculino , Mutação , Piridoxina/uso terapêutico , Splicing de RNA , Transplante de Células-Tronco , Resultado do Tratamento
13.
Cochrane Database Syst Rev ; (10): CD011577, 2015 Oct 05.
Artigo em Inglês | MEDLINE | ID: mdl-26436602

RESUMO

BACKGROUND: Bone marrow failure disorders include a heterogenous group of disorders, of which myelodysplastic syndrome (MDS), forms the largest subgroup. MDS is predominantly a disease of the elderly, with many elderly people managed conservatively with regular allogeneic red blood cell (RBC) transfusions to treat their anaemia. However, RBC transfusions are not without risk. Despite regular transfusions playing a central role in treating such patients, the optimal RBC transfusion strategy (restrictive versus liberal) is currently unclear. OBJECTIVES: To assess the efficacy and safety of a restrictive versus liberal red blood cell transfusion strategy for patients with myelodysplasia, acquired aplastic anaemia, and other inherited bone marrow failure disorders. SEARCH METHODS: We searched for randomised controlled trials (RCTs) in the Cochrane Central Register of Controlled Trials (CENTRAL) (The Cochrane Library 2015, Issue 4), Ovid MEDLINE (from 1946), Ovid EMBASE (from 1974), EBSCO CINAHL (from 1937), the Transfusion Evidence Library (from 1980) and ongoing trial databases to 26th May 2015. SELECTION CRITERIA: RCTs including patients with long-term bone marrow failure disorders that require allogeneic blood transfusion, who are not being actively treated with a haematopoietic stem cell transplant, or intensive chemotherapy. DATA COLLECTION AND ANALYSIS: We used standard Cochrane review methodology. One author initially screened all references, and excluded any that were clearly irrelevant or duplicates. Two authors then independently screened all abstracts of articles, identified by the review search strategy, for relevancy. Two authors independently assessed the full text of all potentially relevant articles for eligibility, completed the data extraction and assessed the studies for risk of bias using The Cochrane Collaboration's 'Risk of bias' tool. MAIN RESULTS: We included one trial (13 participants) and identified three ongoing trials that assess RBC transfusion strategies in people with MDS.The quality of the evidence was very low across different outcomes according to GRADE methodology.The one included study randomised participants to a restrictive [haemoglobin (Hb) transfusion trigger < 72 g/L, 8 participants] or liberal [Hb trigger < 96 g/L, 5 participants] transfusion policy. There was insufficient evidence to determine a difference in all-cause mortality (1 RCT; 13 participants; RR 0.13, 95% CI 0.01 to 2.32; very low quality evidence). There was insufficient evidence to determine a difference in the number of red blood cell transfusions (1 RCT; 13 participants; 1.8 units per patient per month in the liberal group, compared to 0.8 in the restrictive arm, no standard deviation was reported; very low quality evidence). There were no anaemia-related complications reported (cardiac failure) and no reported effect on activity levels (no statistics provided). The study did not report: mortality due to bleeding/infection/transfusion reactions or iron overload, quality of life, frequency and length of hospital admissions, serious infections (requiring admission to hospital), or serious bleeding (e.g. WHO/CTCAE grade 3 (or equivalent) or above). AUTHORS' CONCLUSIONS: This review indicates that there is currently a lack of evidence for the recommendation of a particular transfusion strategy for bone marrow failure patients undergoing supportive treatment only. The one RCT included in this review was only published as an abstract and contained only 13 participants. Further randomised trials with robust methodology are required to develop the optimal transfusion strategy for such patients, particularly as the incidence of the main group of bone marrow failure disorders, MDS, rises with an ageing population.


Assuntos
Doenças da Medula Óssea/terapia , Transfusão de Eritrócitos/métodos , Anemia Aplástica/terapia , Anemia Refratária/terapia , Protocolos Clínicos , Humanos , Leucemia Mielomonocítica Crônica/terapia , Síndromes Mielodisplásicas/terapia , Ensaios Clínicos Controlados Aleatórios como Assunto
15.
J Pediatr Hematol Oncol ; 37(4): 285-9, 2015 May.
Artigo em Inglês | MEDLINE | ID: mdl-25374284

RESUMO

Ghosal hematodiaphyseal dysplasia (GHDD) is a recently recognized cause of steroid-responsive anemia. We would like to report 3 cases of GHDD who presented in early childhood with moderate to severe anemia, splenomegaly, and a hypocellular marrow with increased reticulin. They were easily diagnosed with long-bone x-rays showing diaphyseal and metaphyseal widening and loss of diaphyseal constriction. All cases dramatically responded to oral steroid and no longer needed blood transfusion. They required steroid at low doses for long term (up to 5 y). GHDD is easy to diagnose with long-bone radiography and consistently responds to steroid. It should therefore be considered as a differential diagnosis of unusual anemia in early childhood, especially in children from the Middle East or the Indian subcontinent.


Assuntos
Corticosteroides/uso terapêutico , Anemia Refratária/complicações , Anemia/etiologia , Osteocondrodisplasias/complicações , Anemia/tratamento farmacológico , Anemia Refratária/diagnóstico , Anemia Refratária/terapia , Pré-Escolar , Diagnóstico Diferencial , Feminino , Humanos , Masculino , Osteocondrodisplasias/diagnóstico , Osteocondrodisplasias/terapia
17.
Biol Blood Marrow Transplant ; 20(11): 1711-6, 2014 Nov.
Artigo em Inglês | MEDLINE | ID: mdl-25016195

RESUMO

New transplant approaches are urgently needed for patients with refractory severe aplastic anemia (SAA) who lack a matched sibling or unrelated donor (UD) or who have failed UD or cord blood transplant. Patients with refractory SAA are at risk of later clonal evolution to myelodysplastic syndrome and acute leukemia. We report our pilot findings with haploidentical hematopoietic stem cell transplantation (haploHSCT) using uniform reduced-intensity conditioning with postgraft high-dose cyclophosphamide in 8 patients with refractory SAA or patients who rejected a prior UD or cord blood transplant. Six of 8 patients engrafted. Graft failure was associated with donor-directed HLA antibodies, despite intensive pre-HSCT desensitization with plasma exchange and rituximab. There was only 1 case of grade II skin graft-versus-host disease. We show that haploHSCT can successfully rescue refractory SAA patients who lack donor-directed HLA antibodies but not in the presence of donor-directed HLA antibodies. This novel protocol for haploHSCT for SAA has been adopted by the European Group for Blood and Marrow Transplantation Severe Aplastic Anaemia Working Party for a future noninterventional, observational study to further evaluate its efficacy.


Assuntos
Anemia Aplástica/terapia , Anemia Refratária/terapia , Transplante de Células-Tronco Hematopoéticas/métodos , Condicionamento Pré-Transplante/métodos , Adulto , Estudos de Coortes , Feminino , Humanos , Masculino , Pessoa de Meia-Idade , Doadores de Tecidos , Transplante Homólogo , Adulto Jovem
18.
Blood ; 123(3): 326-33, 2014 Jan 16.
Artigo em Inglês | MEDLINE | ID: mdl-24215034

RESUMO

Endoscopic gastrointestinal workup fails to establish the cause of iron deficiency anemia (IDA) in a substantial proportion of patients. In patients referred for hematologic evaluation with unexplained or refractory IDA, screening for celiac disease, autoimmune gastritis, Helicobacter pylori, and hereditary forms of IDA is recommended. About 4% to 6% of patients with obscure refractory IDA have celiac disease, and autoimmune gastritis is encountered in 20% to 27% of patients. Stratification by age cohorts in autoimmune gastritis implies a disease presenting as IDA many years before the establishment of clinical cobalamin deficiency. Over 50% of patients with unexplained refractory IDA have active H pylori infection and, after excluding all other causes of IDA, 64% to 75% of such patients are permanently cured by H pylori eradication. In young patients with a history suggestive of hereditary iron deficiency with serum ferritin higher than expected for IDA, mutations involving iron trafficking and regulation should be considered. Recognition of the respective roles of H pylori, autoimmune gastritis, celiac disease, and genetic defects in the pathogenesis of iron deficiency should have a strong impact on the current diagnostic workup and management of unexplained, or refractory, IDA.


Assuntos
Anemia Ferropriva/terapia , Anemia Refratária/terapia , Hematologia/métodos , Administração Oral , Doenças Autoimunes/complicações , Endoscopia Gastrointestinal/efeitos adversos , Endoscopia Gastrointestinal/métodos , Feminino , Ferritinas/sangue , Gastrite/complicações , Genótipo , Infecções por Helicobacter/diagnóstico , Helicobacter pylori/metabolismo , Humanos , Ferro/administração & dosagem , Deficiências de Ferro , Masculino , Mutação
19.
Rev Med Brux ; 34(4): 323-7, 2013 Sep.
Artigo em Francês | MEDLINE | ID: mdl-24195247

RESUMO

Refractory anemia, also known as myelodysplastic syndromes, forms a group of clonal diseases characterized by cytopenias with mostly rich bone marrow. Preferentially reaching an older population, the prognosis depends on both comorbidities and characteristics of the disease, which have been grouped into a score established in 1997 ("IPSS = International Prognostic Scoring System") and revised in 2012 ("R-IPSS = Revised IPSS"). Overall survival and risk of transformation into acute nonlymphoblastic leukemia can now be estimated fairly accurately. Based on these characteristics, the treatment will be mainly supportive or will use several new molecules: growth factors, lenalidomide, 5-azacitidine, etc. A minority of patients may also benefit from allogeneic BMT or sometimes immunosuppressive therapy.


Assuntos
Anemia Refratária , Idoso , Idoso de 80 Anos ou mais , Anemia Refratária/diagnóstico , Anemia Refratária/epidemiologia , Anemia Refratária/terapia , Transplante de Medula Óssea , Humanos , Imunossupressores/uso terapêutico , Prognóstico , Medição de Risco
20.
Blood ; 122(22): 3561-7, 2013 Nov 21.
Artigo em Inglês | MEDLINE | ID: mdl-24052548

RESUMO

Refractory aplastic anemia (AA) is defined as a lack of response to first-line immunosuppressive therapy (IST) with antithymocyte globulin and cyclosporin and is manifested as persistence of severe cytopenias at 6 months after IST. Although supportive care is critical for AA patients, it is of paramount importance for refractory disease in view of the longer duration of pancytopenia and susceptibility to life-threatening infections due to IST. Improvements in supportive care have largely contributed to better outcome over the past 2 decades, with 5-year overall survival reaching 57% during 2002 to 2008 for patients with AA unresponsive to initial IST. Exclusion of hypocellular myelodysplastic syndrome and constitutional BM failure masquerading as apparent idiopathic AA should be done in conjunction with centers of excellence. Hematopoietic stem cell transplantation is indicated if refractory AA patients are fit and have a suitably matched donor, either a sibling (>40-50 years) or unrelated donor. Patients lacking a fully matched donor should be considered for a second course of antithymocyte globulin plus cyclosporin, although response in the refractory setting is only ∼30% to 35%. Response may also occur with alemtuzumab or the thrombopoietin mimetic eltrombopag in refractory AA. The emerging data for alternate donor (cord or haploidentical) transplantation in AA has provided additional therapeutic choices to consider in refractory disease.


Assuntos
Anemia Aplástica/terapia , Anemia Refratária/terapia , Soro Antilinfocitário/uso terapêutico , Transfusão de Sangue , Transplante de Células-Tronco de Sangue do Cordão Umbilical , Transplante de Células-Tronco Hematopoéticas , Humanos , Imunossupressores/uso terapêutico , Controle de Infecções , Resultado do Tratamento , Doadores não Relacionados
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