Abstract:
:Immunoglobulin light chain amyloidosis (AL amyloidosis) is a rare, life-threatening disease characterized by the deposition of misfolded proteins in vital organs such as the heart, the lungs, the kidneys, the peripheral nervous system, and the gastrointestinal tract. This causes a direct toxic effect, eventually leading to organ failure. The underlying B-cell lymphoproliferative disorder is almost always a clonal plasma cell disorder, most often a small plasma cell clone of <10%. Current therapy is directed toward elimination of the plasma cell clone with the goal of preventing further organ damage and reversal of the existing organ damage. Autologous stem cell transplantation has been shown to be a very effective treatment in patients with AL amyloidosis, although it cannot be widely applied as patients are often frail at presentation, making them ineligible for transplantation. Treatment with cyclophosphamide, bortezomib, and dexamethasone has emerged as the standard of care for the treatment of AL amyloidosis. Novel anti-plasma cell therapies, such as second generation proteasome inhibitors, immunomodulators, monoclonal antibodies targeting a surface protein on the plasma cell (daratumumab, elotuzumab), and the small molecular inhibitor venetoclax, have continued to emerge and are being evaluated in combination with the standard of care. However, there is still a need for therapies that directly target the amyloid fibrils and reverse organ damage. In this review, we will discuss current and emerging nonchemotherapy treatments of AL amyloidosis, including antifibril directed therapies under current investigation.
journal_name
Acta Haematoljournal_title
Acta haematologicaauthors
Van Doren L,Lentzsch Sdoi
10.1159/000507724subject
Has Abstractpub_date
2020-01-01 00:00:00pages
373-380issue
4eissn
0001-5792issn
1421-9662pii
000507724journal_volume
143pub_type
杂志文章,评审abstract::We investigated double (specific and nonspecific) esterase (DE) staining in marrow cells of 237 patients with the myelodysplastic syndromes (MDS). Additional abnormalities of neutrophilic granules were examined cytochemically and immunocytochemically for myeloperoxidase activity and antigen elastase, lactoferrin and C...
journal_title:Acta haematologica
pub_type: 杂志文章
doi:10.1159/000040855
更新日期:1998-01-01 00:00:00
abstract::Recent molecular biological investigations have shown that genetic information on the short arm of chromosome 17 is frequently deleted or inactivated in acute myeloid leukaemias and a number of solid tumours. In earlier studies we have found clonal occurrence of a 17p deletion (17p-) in patients with refractory anaemi...
journal_title:Acta haematologica
pub_type: 杂志文章
doi:10.1159/000205033
更新日期:1990-01-01 00:00:00
abstract::The detection of cellular antigens in bone marrow sections depends on the method of embedding, the nature of antigen and antibody, antigen retrieval techniques and the sensitivity of the immunohistochemical method. This study evaluated a fluorescyl-tyramide-enhanced immunostaining method on methyl-methacrylate-embedde...
journal_title:Acta haematologica
pub_type: 杂志文章
doi:10.1159/000097458
更新日期:2007-01-01 00:00:00
abstract::It has been suggested that CD44 mediates adhesive interactions between hematopoietic progenitor cells and the stromal microenvironment. Ligands of CD44 include several extracellular matrix components, such as hyaluronic acid and fibronectin. Antibodies against CD44 have been shown to induce homotypic T cell aggregatio...
journal_title:Acta haematologica
pub_type: 杂志文章
doi:10.1159/000203886
更新日期:1996-01-01 00:00:00
abstract::A 69-year-old man with a rare case of lymphoma-type alpha-chain disease was admitted to the hospital with marked cervical and inguinal lymph node swelling. Lymph node biopsy showed marked infiltration of plasma cells, plasmacytoid cells and immunoblastoid cells, alone or in combination. Immunoelectrophoresis and immun...
journal_title:Acta haematologica
pub_type: 杂志文章
doi:10.1159/000204814
更新日期:1991-01-01 00:00:00
abstract::The frequency of chronic lymphocytic leukemia (CLL) varies a great deal from one population to another. We have undertaken to compare the aspects of CLL in Maghreb and Europe through two series of comparable importance, studied during the same period and under similar conditions in Algiers and Amiens. This comparison ...
journal_title:Acta haematologica
pub_type: 杂志文章
doi:10.1159/000206896
更新日期:1983-01-01 00:00:00
abstract::A case of lymphoma presenting with features shared by hairy cell leukemia (HCL) and its variant, intermediate lymphocytic lymphoma (ILL) and monocytoid B-cell lymphoma (MBCL) is described. Clinical presentation and the morphological findings observed in peripheral blood and in bone marrow biopsy suggested an HCL; howe...
journal_title:Acta haematologica
pub_type: 杂志文章
doi:10.1159/000204496
更新日期:1993-01-01 00:00:00
abstract::Automated cytochemistry by the Hemalog D carries out leukocyte differential counts in a continuous flow mode on whole blood EDTA-collected samples, through the optical measurement of enzyme activity and cell size. This operative principle thus parallels the basis of the FAB classification of acute leukemias. Although ...
journal_title:Acta haematologica
pub_type: 杂志文章
doi:10.1159/000206394
更新日期:1984-01-01 00:00:00
abstract::In the last 18 years, we have observed 24 cases of hypoparathyroidism (HPT) in beta-thalassemia major. At present, 4.5% of patients followed regularly in our department have this complication. HPT is thought to be mainly the consequence of iron deposition in the parathyroid glands. The age of our patients when HPT was...
journal_title:Acta haematologica
pub_type: 杂志文章
doi:10.1159/000204662
更新日期:1992-01-01 00:00:00
abstract::The clinical pattern of haemolysis associated with Donath-Landsteiner antibodies has undergone a change over the years. In the current study 13 patients developed the acute form of the disease whilst only 1 presented with the classical picture of chronic paroxysmal cold haemoglobinuria. The acute illness typically occ...
journal_title:Acta haematologica
pub_type: 杂志文章
doi:10.1159/000206992
更新日期:1982-01-01 00:00:00
abstract::Electron-microscopic examination of peripheral blood from a patient with 'hairy cell' leukemia revealed classical 'hairy cells', atypical lymphoid cells and numerous pathological plasma cells. Osmiophilic granular material coated the cell surface of 'hairy cells' and lymphoid cells but not the plasmalemma of the plasm...
journal_title:Acta haematologica
pub_type: 杂志文章
doi:10.1159/000208089
更新日期:1975-01-01 00:00:00
abstract::Two unrelated patients with Bernard-Soulier syndrome and their relatives were studied. The patients demonstrated severe bleeding diathesis, the relatives were asymptomatic. The propositi showed the characteristic abnormalities of the syndrome: thrombocytopenia, a percentage of giant platelets higher than 65%, prolonge...
journal_title:Acta haematologica
pub_type: 杂志文章
doi:10.1159/000206125
更新日期:1986-01-01 00:00:00
abstract::Differentiation of rapidly binding coagulation factor inhibitors from antiphospholipid antibodies is a challenge for the hemostaseologic laboratory, especially with respect to the different therapeutic consequences. Several immunological and functional assays for the diagnosis of these disorders have been proposed. He...
journal_title:Acta haematologica
pub_type: 杂志文章
doi:10.1159/000040821
更新日期:1998-01-01 00:00:00
abstract::We describe a B cell chronic lymphocytic leukemia/lymphoma (B-CLL) patient with an adrenocortical adenoma. He was treated initially with oral cyclophosphamide and prednisolone for 2 months, followed by low-dose prednisolone for an additional 2 years, which resulted in the prompt disappearance of CLL cells. After a per...
journal_title:Acta haematologica
pub_type: 杂志文章
doi:10.1159/000203692
更新日期:1997-01-01 00:00:00
abstract::Multiple myeloma and benign monoclonal gammopathies (BMG) are regarded as monoclonal B cell proliferations in which B lymphocyte maturation is blocked in the final stages of the differentiation cycle. Further studies with monoclonal antibodies (MoAbs) of the FMC series and of the 791T/36 MoAb will perhaps provide a fi...
journal_title:Acta haematologica
pub_type: 杂志文章
doi:10.1159/000205903
更新日期:1987-01-01 00:00:00
abstract::The immunological phenotypes of lymphocytes and myeloma cells in 48 patients with multiple myeloma (MM) were analyzed using a panel of monoclonal antibodies (mAbs). Myeloma cells were positive for OKT10, BL3, PCA1 and BA2. In a few cases, they were also positive for the B cell-associated antigens J5, B1 and I2. Eight ...
journal_title:Acta haematologica
pub_type: 杂志文章
doi:10.1159/000205187
更新日期:1990-01-01 00:00:00
abstract::Blastic plasmacytoid dendritic cell neoplasm (BPDCN) is a rare and highly aggressive hematological malignancy derived from precursors of plasmacytoid dendritic cells. This disease typically presents with cutaneous involvement as the first manifestation, with subsequent or simultaneous spread to bone marrow and periphe...
journal_title:Acta haematologica
pub_type: 杂志文章,评审
doi:10.1159/000334703
更新日期:2012-01-01 00:00:00
abstract::B-cell chronic lymphocytic leukaemia (B-CLL) is characterized by a high frequency of infections, including those of viral aetiology. Previous reports have demonstrated a specific immunologic response to influenza virus vaccine in B-CLL patients with normal IgG levels. In this study, we have evaluated different immunop...
journal_title:Acta haematologica
pub_type: 杂志文章
doi:10.1159/000040709
更新日期:1998-01-01 00:00:00
abstract::Dihydrofolate reductase (FH2-R) was studied cytochemically in the bone marrow erythroblasts of 20 normal controls and 46 patients with myelodysplastic syndromes (MDSs) classified according to FAB, prior to therapy. The reaction product was quantified for the same samples with a Vickers M86 microdensitometer. The enzym...
journal_title:Acta haematologica
pub_type: 杂志文章
doi:10.1159/000205807
更新日期:1988-01-01 00:00:00
abstract::In the myelodysplastic syndromes (MDS) clonogenic marrow cell culture studies have demonstrated intrinsic hemopoietic stem cell and progenitor cell abnormalities consistent with these disorders representing clonal hemopathies. Abnormal responsiveness of these cells to stimulatory and inhibitory growth factors indicate...
journal_title:Acta haematologica
pub_type: 杂志文章,评审
doi:10.1159/000205911
更新日期:1987-01-01 00:00:00
abstract::Alpha-thalassemia is very common in the Kuwaiti population, but its influence on anemia of pregnancy has not been previously investigated. We have screened a group of 59 anemic (Hb < 11 g/dl) pregnant women for the alpha-thal-2 (-alpha-3.7 kb) deletion which is the commonest alpha-thal allele in this community, using ...
journal_title:Acta haematologica
pub_type: 杂志文章
doi:10.1159/000039757
更新日期:2000-01-01 00:00:00
abstract::Increased levels of hemoglobin A(2) (HbA(2)) are present in most beta-thalassemia carriers. The mechanism of this effect is not understood, although the increase may result from transcriptional and posttranscriptional changes. In the present study, we quantitate delta-globin mRNA levels in peripheral-blood-enriched re...
journal_title:Acta haematologica
pub_type: 杂志文章
doi:10.1159/000040959
更新日期:1999-01-01 00:00:00
abstract::Fourteen patients with hematologic neoplasia (11 acute myeloid leukemias, 2 non-Hodgkin's lymphomas and 1 blast crisis of chronic myeloid leukemia) who underwent high-dose cytosine arabinoside (HIDARAC) therapy with or without sequential asparaginase (ASNase) were investigated in order to evaluate liver toxicity and a...
journal_title:Acta haematologica
pub_type: 临床试验,杂志文章
doi:10.1159/000206094
更新日期:1986-01-01 00:00:00
abstract::It has been found possible to test ferritin concentrations in the reticulum and hemopoietic cells from human bone marrow by an immunoradiometric assay. Ferritin concentration in healthy test persons amounts to 0.92 +/- 0.38 ng/microgram protein in the reticulum and 0.084 +/- 0.031 ng/microgram protein in hemopoietic c...
journal_title:Acta haematologica
pub_type: 杂志文章
doi:10.1159/000207030
更新日期:1982-01-01 00:00:00
abstract::Preliminary results from the first 21 patients of a group of 30 with International Federation of Gynaecology and Obstetrics (FIGO) stage II-IV epithelial ovarian carcinoma and anaemia are reported. Patients entered this open-label, comparative-group, out-patient study and were randomized to receive conventional suppor...
journal_title:Acta haematologica
pub_type: 临床试验,杂志文章,随机对照试验
doi:10.1159/000204782
更新日期:1992-01-01 00:00:00
abstract::We describe a case of hemolytic anemia with subacute bacterial endocarditis due to Streptococcus sanguis. The major hematological features of the patient were a normocytic anemia with reticulocytosis, an elevation of serum lactate dehydrogenase, and a marked reduction of serum haptoglobin. All these signs of hemolytic...
journal_title:Acta haematologica
pub_type: 杂志文章
doi:10.1159/000203981
更新日期:1995-01-01 00:00:00
abstract::The fibrinolytic response to surgical trauma was studied longitudinally in 7 pigs by measuring the fibrinolytic activity in the blood and vein wall simultaneously. The results indicated a marked suppression in fibrinolytic activity in the blood which was closely correlated with the activity in the vein wall. This corr...
journal_title:Acta haematologica
pub_type: 杂志文章
doi:10.1159/000207397
更新日期:1980-01-01 00:00:00
abstract::We examined the in vivo effect of granulocyte colony-stimulating factor (G-CSF) on the surface expression of putative counterligands for endothelial selectins on neutrophils in healthy volunteers. G-CSF (50 microg/m2/day) was administered subcutaneously to 5 healthy volunteers for 4 days. The expression of surface ant...
journal_title:Acta haematologica
pub_type: 杂志文章
doi:10.1159/000040901
更新日期:1998-01-01 00:00:00
abstract::Neurologic complications of allogeneic hematopoietic cell transplantation (allo-HCT) include infections, cerebrovascular events, therapy-induced neurotoxicity, recurrent malignancies, and neurologic manifestations of graft-versus-host disease (GVHD). Anti-glutamic acid decarboxylase (GAD) antibody-associated cerebella...
journal_title:Acta haematologica
pub_type: 杂志文章
doi:10.1159/000494423
更新日期:2019-01-01 00:00:00
abstract::Non-transfusion-dependent thalassemia (NTDT) is associated with various forms of thalassemia and genetic modifiers. We report the molecular basis of NTDT in hemoglobin (Hb) E-β-thalassemia disease. This study was done in 73 adult patients encountered at the prenatal diagnosis center of Khon Kaen University, Northeast ...
journal_title:Acta haematologica
pub_type: 临床试验,杂志文章
doi:10.1159/000449120
更新日期:2016-01-01 00:00:00