Abstract:
:Acquired aplastic anemia (AA) and paroxysmal nocturnal hemoglobinuria (PNH) are interrelated ultra-rare diseases. Quality of life (QoL) evaluation tools used in studies for AA and PNH are unspecific and designed for cancer patients (e.g., the European Organization for Research and Treatment of Cancer Quality of Life Questionnaire, EORTC QLQ-C30). Given the complexity of AA and PNH, variation in symptoms and treatments, younger age of many patients, and the fact that AA and PNH are not classified as malignant diseases, it is likely that cancer-specific questionnaires are inappropriate. We generate an AA/PNH-specific QoL questionnaire (QLQ-AA/PNH), performed according to EORTC guidelines. QoL issues were obtained from the literature and interviews with patients and physicians (phase I), then ranked by patients and physicians. In phase II, items were created. Patients in more than 25 German and Swiss cities were interviewed face to face. In phase I, interviews of 19 patients and 8 physicians specialized in AA/PNH treatment resulted in 649 QoL issues; these were condensed to 175 and graded according to their importance by 30 patients and 14 physicians (phase II). Five physicians took part in phases I and II. Altogether, 97 issues were rated important. Twelve EORTC QLQ-C30 items were not rated important, while several new QoL aspects were brought up. Modifications in wording and phrasing led to two questionnaires with 77 items regarding general QoL aspects and 20 items regarding medical care. Important QoL aspects of PNH/AA patients are inappropriately captured with available QoL tools. Developing a new QoL questionnaire specific for this patient group is warranted.
journal_name
Ann Hematoljournal_title
Annals of hematologyauthors
Groth M,Singer S,Niedeggen C,Petermann-Meyer A,Röth A,Schrezenmeier H,Höchsmann B,Brümmendorf TH,Panse Jdoi
10.1007/s00277-016-2867-8subject
Has Abstractpub_date
2017-02-01 00:00:00pages
171-181issue
2eissn
0939-5555issn
1432-0584pii
10.1007/s00277-016-2867-8journal_volume
96pub_type
杂志文章abstract::This study retrospectively analyzed the clinical outcome of 172 children with newly diagnosed severe aplastic anemia (SAA) between January 2008 and April 2018, who received rabbit antithymocyte globulin (ATG) and cyclosporine (CsA) as first-line treatment. The median age at diagnosis was 5 years (range, 1-14). The ove...
journal_title:Annals of hematology
pub_type: 杂志文章
doi:10.1007/s00277-020-04296-9
更新日期:2021-01-01 00:00:00
abstract::Second-generation azoles may be more effective than first-generation azoles in the prevention of fungal infections in hematology patients. We performed a systematic review with meta-analysis of randomized controlled trials comparing second- with first-generation azoles in hematology patients with respect to proven or ...
journal_title:Annals of hematology
pub_type: 杂志文章,meta分析,评审
doi:10.1007/s00277-013-1693-5
更新日期:2013-06-01 00:00:00
abstract::We have studied the effects of recombinant human thrombopoietin (TPO, mpl ligand) on the megakaryocyte colony formation from control human bone marrow cells, human leukemia cells at diagnosis, and human bone marrow cells after induction chemotherapy for acute leukemias. In the control human bone marrow cells from four...
journal_title:Annals of hematology
pub_type: 杂志文章
doi:10.1007/s002770050408
更新日期:1998-07-01 00:00:00
abstract::The impact of intraocular involvement (IOL) in primary CNS lymphoma (PCNSL) has not been sufficiently evaluated. Here, we present the analysis of IOL in the only completed randomized phase III trial in PCNSL. The G-PCNSL-SG1 study evaluated the role of whole-brain radiotherapy in primary therapy of PCNSL. Data of the ...
journal_title:Annals of hematology
pub_type: 杂志文章,多中心研究,随机对照试验
doi:10.1007/s00277-014-2212-z
更新日期:2015-03-01 00:00:00
abstract::Polycythemia vera (PV) is a myeloproliferative neoplasm characterized by overproduction of red blood cells. We have performed a comprehensive characterization of blood immune cells for expression of naïve and memory receptors as well as β2m-associated and β2m-free MHC class I heavy chains, also known as closed and ope...
journal_title:Annals of hematology
pub_type: 杂志文章
doi:10.1007/s00277-018-3332-7
更新日期:2018-09-01 00:00:00
abstract::The rarity of severe complications of this disease in children makes randomized clinical trials in immune thrombocytopenia (ITP) unfeasible. Therefore, the current management recommendations for ITP are largely dependent on clinical expertise and observations. As part of its discussions during the Intercontinental Coo...
journal_title:Annals of hematology
pub_type: 共识发展会议,杂志文章
doi:10.1007/s00277-010-0941-1
更新日期:2010-07-01 00:00:00
abstract::Therapy-induced autoimmunity may mediate the destruction of cancer cells. Previous studies have demonstrated that presence of autoimmune thyroid disorder is associated with favorable outcome in patients with solid cancer. Patients with diffuse large B cell lymphoma (DLBCL) who achieved complete response on positron em...
journal_title:Annals of hematology
pub_type: 杂志文章
doi:10.1007/s00277-015-2311-5
更新日期:2015-06-01 00:00:00
abstract::We analyzed the prognostic factors for a successful mobilization and peripheral blood stem cell collection in a series of 57 consecutive patients with multiple myeloma (MM); a new scoring system to predict an adequate mobilization in this subset of patients was also constructed. A total of 221 aphereses were performed...
journal_title:Annals of hematology
pub_type: 杂志文章
doi:10.1007/s002770100351
更新日期:2001-10-01 00:00:00
abstract::Multiple myeloma (MM) is an incurable disease accompanied by low plasma levels of low-density lipoprotein cholesterol (LDL-c). The significance of altered cholesterol metabolism in the pathophysiology of MM remains elusive. Although it has been hypothesized that myeloma cells depend on exogenous cholesterol for its su...
journal_title:Annals of hematology
pub_type: 杂志文章
doi:10.1007/s00277-011-1246-8
更新日期:2012-01-01 00:00:00
abstract::The development of thrombocytopenia following exposure to the platelet glycoprotein (GP) IIb/ IIIa receptor antagonist abciximab (c7E3 Fab, ReoPro) is associated with adverse clinical outcome and excessive bleeding. Pseudothrombocytopenia is an important differential diagnosis in sudden onset of thrombocytopenia in a ...
journal_title:Annals of hematology
pub_type: 杂志文章
doi:10.1007/s002770050573
更新日期:2000-03-01 00:00:00
abstract::A 57-year-old man with acute myeloid leukemia (AML) French-American-British (FAB) 4 developed disseminated invasive cerebral and pulmonary aspergillosis during postinduction aplasia. According to international consensus, infection was categorized as probable (two host factors: deep neutropenia for >10 days and refract...
journal_title:Annals of hematology
pub_type: 杂志文章
doi:10.1007/s00277-003-0792-0
更新日期:2004-06-01 00:00:00
abstract::Recombinant human granulocyte colony-stimulating factor (rhG-CSF) and erythropoietin (rhE-PO) were used to treat ten patients with myelodysplastic syndromes (MDS). None of the patients showed a favorable response in erythrocyte and platelet counts following 10 weeks' treatment, although favorable responses in neutroph...
journal_title:Annals of hematology
pub_type: 杂志文章
doi:10.1007/BF01834360
更新日期:1994-04-01 00:00:00
abstract::Ruxolitinib, a JAK1 and JAK2 inhibitor, has been tested and approved for the treatment of primary and secondary myelofibrosis (MF). Aim of our study is to report safety and efficacy of ruxolitinib in 98 patients affected by MF treated outside clinical trials and collected and treated consecutively by the Lazio Coopera...
journal_title:Annals of hematology
pub_type: 杂志文章,多中心研究
doi:10.1007/s00277-016-2884-7
更新日期:2017-03-01 00:00:00
abstract::Haplo-identical transplants (Haplo-Tx) are an important alternative for patients with hematological malignancies who lack a HLA-identical donor. Seventy-one T-replete Haplo-Tx were performed in 70 high-risk patients at our center; 22/70 (31%) patients with refractory/relapsed leukemia received sequential salvage thera...
journal_title:Annals of hematology
pub_type: 临床试验,杂志文章
doi:10.1007/s00277-018-3433-3
更新日期:2018-11-01 00:00:00
abstract::To characterize the clinical features, associated disorders, and treatment of necrobiotic xanthogranuloma (NXG), a rare non-Langerhans cell histiocytosis, we conducted a retrospective review of pathologically confirmed NXG at Mayo Clinic Arizona from 1987 to June 2017. Data on clinical findings, laboratory findings, a...
journal_title:Annals of hematology
pub_type: 杂志文章
doi:10.1007/s00277-018-3301-1
更新日期:2018-08-01 00:00:00
abstract::Immunoglobulin D (IgD) myeloma is a rare subtype that used to lead to a poor outcome. To investigate the current clinical features, cytogenetic changes and survival of patients with IgD myeloma under novel treatments, we analysed 47 patients with IgD myeloma, 31 men and 16 women, with a median age of 54.5 years. We fo...
journal_title:Annals of hematology
pub_type: 杂志文章
doi:10.1007/s00277-018-3582-4
更新日期:2019-04-01 00:00:00
abstract::Follicular lymphoma (FL) is a low-grade lymphoma that is usually characterized by generalized lymphadenopathy. Extranodal invasion by FL generally involves the bone marrow, skin, and duodenum; splenic infiltration often occurs in the advanced stages. However, primary splenic FL is very rare. Hence, few studies have be...
journal_title:Annals of hematology
pub_type: 临床试验,杂志文章
doi:10.1007/s00277-017-3139-y
更新日期:2017-12-01 00:00:00
abstract::B cell-activating factor (BAFF) is a cytokine that plays a major role in the maintenance of normal B-cell development and homeostasis. It has been suggested that in multiple myeloma (MM) it might have regulatory effects on the proliferation and viability of malignant plasma cells. The aim of this study was to evaluate...
journal_title:Annals of hematology
pub_type: 杂志文章
doi:10.1007/s00277-012-1470-x
更新日期:2012-09-01 00:00:00
abstract::Extreme thrombocytosis (ExT) has been associated with an increased bleeding risk in myeloproliferative neoplasm (MPN) patients and is included in the high risk category in treatment guidelines. Treatment of patients with ExT has not been studied in prospective trials. To study physicians' approaches to ExT, we distrib...
journal_title:Annals of hematology
pub_type: 杂志文章
doi:10.1007/s00277-016-2826-4
更新日期:2017-01-01 00:00:00
abstract::Although retreatment with alemtuzumab in relapsing B-cell chronic lymphocytic leukemia (CLL) may be beneficial, there has thus far been no thorough analysis available on this topic. Data were collected from 30 chemotherapy-pretreated patients with progressive CLL who had received alemtuzumab twice in consecutive, dist...
journal_title:Annals of hematology
pub_type: 杂志文章,多中心研究
doi:10.1007/s00277-011-1192-5
更新日期:2011-09-01 00:00:00
abstract::Due to their homing properties, extranodal marginal zone B-cell lymphoma (MZBL) of mucosa-associated lymphoid tissue (MALT) type remain localized for long periods of time, and therefore have an excellent prognosis. However, if generalization and/or transformation into a diffuse large-cell lymphoma occurs, the prognosi...
journal_title:Annals of hematology
pub_type: 杂志文章
doi:10.1007/s002770000203
更新日期:2000-12-01 00:00:00
abstract::While much has been learned about the basic immunology and clinical characteristics of immune thrombocytopenia, many important questions remain with regard to pathogenesis, disease progression, identification of novel therapeutic targets and approaches, and clinical trials that rationalize and optimize use of existing...
journal_title:Annals of hematology
pub_type: 杂志文章
doi:10.1007/s00277-010-0917-1
更新日期:2010-07-01 00:00:00
abstract::Early cytomegalovirus (CMV) replication (eCMV) after allogeneic hematopoietic stem cell transplantation (allo-HSCT) has been suggested as an independent factor that reduces leukemia relapse risk. We retrospectively analyzed 74 patients with acute myeloid leukemia (AML) who underwent allo-HSCT between August 2006 and S...
journal_title:Annals of hematology
pub_type: 杂志文章
doi:10.1007/s00277-014-2190-1
更新日期:2015-02-01 00:00:00
abstract::A 24-year-old woman with a large cell anaplastic CD 30-positive T-cell non-Hodgkin's lymphoma (NHL) developed downbeat nystagmus, anisocoria, and oscillopsia. Prior to overt cerebral invasion by NHL, she had a thiamine deficiency with very low thiamine concentrations in the CSF, probably caused by protracted vomiting ...
journal_title:Annals of hematology
pub_type: 杂志文章
doi:10.1007/s002770050484
更新日期:1999-02-01 00:00:00
abstract::Patients previously diagnosed with invasive aspergillosis (IA) have been considered to be at risk for relapse of mycosis during subsequent hematopoietic transplant. Even with prophylactic measures, reactivation of the infection occurs in 29% of patients undergoing bone marrow transplantation (BMT). A period of neutrop...
journal_title:Annals of hematology
pub_type: 杂志文章
doi:10.1007/s002770100311
更新日期:2001-08-01 00:00:00
abstract::The production of erythropoietin (Epo), the glycoprotein hormone which controls red blood cell formation, is regulated by feedback mechanisms sensing tissue oxygenation. The mechanism of the putative oxygen sensor has yet to be elucidated. There is evidence that at least two pathways participate in hypoxia signal tran...
journal_title:Annals of hematology
pub_type: 杂志文章
doi:10.1007/s002770050514
更新日期:1999-06-01 00:00:00
abstract::Second allogeneic hematopoietic stem cell transplantation (allo-HSCT) has a low survival outcome and a high non-relapse mortality (NRM) rate which is a major obstacle to this treatment. We hypothesized that the status of malnourishment after first allo-HSCT as represented by the geriatric nutritional risk index (GNRI)...
journal_title:Annals of hematology
pub_type: 杂志文章
doi:10.1007/s00277-020-04089-0
更新日期:2020-07-01 00:00:00
abstract::Information regarding prognostic determinants of outcome after splenectomy for adult immune thrombocytopenic purpura (ITP) and the management of postsplenectomy relapse is limited. Among 140 adult patients with ITP who had therapeutic splenectomy at our institution, 88% achieved either a complete (platelets > 150 x 10...
journal_title:Annals of hematology
pub_type: 杂志文章
doi:10.1007/s00277-002-0461-8
更新日期:2002-06-01 00:00:00
abstract::In spite of their recognized risk of thrombosis, patients with myeloproliferative neoplasms (MPN) show little or no abnormalities of traditional coagulation tests, perhaps because these are unable to represent the balance between pro- and anticoagulants nor the effect of platelets and blood cells. We investigated whet...
journal_title:Annals of hematology
pub_type: 杂志文章
doi:10.1007/s00277-013-1834-x
更新日期:2013-12-01 00:00:00
abstract::In this report we show that serum has differentiation-inducing effects on primitive hematopoietic progenitor cells with the CD34++CD38- immunophenotype. Using the pre-colony forming unit (pre-CFU) assay as a model for early myelopoiesis, we compared the effects of serum-containing and serum-free media and evaluated di...
journal_title:Annals of hematology
pub_type: 杂志文章
doi:10.1007/s002770000227
更新日期:2001-01-01 00:00:00