RESUMO
Refractory anaemia (RA) among myelodysplastic syndrome (MDS) is associated with a partial functional iron deficit and may require transfusions. In low-risk lymphoma and solid tumour patients, iron support improves erythropoietin (EPO) cost-effectiveness in treating anaemia. The aim of this study is to see if oral sucrosomial iron support improves the cost-effectiveness of EPO treatment in MDS patients affected by low-risk RA. We treated patients with EPO only or with EPO plus oral sucrosomial iron or intravenous (i.v.) iron. The need for transfusions was lowest in the group taking oral iron (p = 0.016) or not receiving supplementation at all (p = 0.022). We compared costs of EPO with i.v. ferric gluconate or oral sucrosomial iron supplementation or no iron supplementation. The oral iron group had fewer side effects, fewer patient medical visits in the out-patient setting, and fewer transfusions; this led to higher savings on direct hospital costs and indirect patient costs (lost days at work) and translated into a 50% abatement of overall expenditures. EPO treatment-related expenditures in MDS-RA patients were lowest with oral sucrosomial iron supplementation (Sideral®), with a longer interval between EPO administration in maintenance treatment, quicker hemoglobin recovery, lower ferritin increase and fewer blood transfusions.
Assuntos
Anemia Refratária/tratamento farmacológico , Eritropoetina/uso terapêutico , Ferro/administração & dosagem , Síndromes Mielodisplásicas/patologia , Idoso , Idoso de 80 Anos ou mais , Anemia Refratária/complicações , Anemia Refratária/economia , Suplementos Nutricionais , Progressão da Doença , Eritropoetina/economia , Feminino , Compostos Férricos/administração & dosagem , Ferritinas/sangue , Custos de Cuidados de Saúde , Humanos , Itália , Masculino , Síndromes Mielodisplásicas/complicações , Resultado do TratamentoRESUMO
INTRODUCTION: Gastrointestinal angiodysplasias are an important cause of difficult-to-manage bleeding, especially in older patients. Endoscopic coagulation of angiodysplasias is the mainstay of treatment, but may be difficult for small bowel angiodysplasias because of the inability to reach them for endoscopic intervention. Some patients are red blood cell (RBC) transfusion dependent due to frequent rebleeding despite endoscopic treatment. In small cohort studies, octreotide appears to decrease the number of bleeding episodes in patients with RBC transfusion dependency due to gastrointestinal angiodysplasias. This trial will assess the efficacy of octreotide in decreasing the need for RBC transfusions and parenteral iron in patients with anaemia due to gastrointestinal bleeding of small bowel angiodysplasias despite endoscopic intervention. STUDY DESIGN: Randomised controlled, superiority, open-label multicentre trial. PARTICIPANTS: 62 patients will be included with refractory anaemia due to small bowel angiodysplasias, who are RBC transfusion or iron infusion dependent despite endoscopic intervention and oral iron supplementation. INTERVENTION: Patients will be randomly assigned (1:1) to standard care or 40â mg long-acting octreotide once every 4â weeks for 52â weeks, in addition to standard care. The follow-up period is 8â weeks. MAIN OUTCOME MEASURES: The primary outcome is the difference in the number of blood and iron infusions between the year prior to inclusion and the treatment period of 1â year. Important secondary outcomes are the per cent change in the number of rebleeds from baseline to end point, adverse events and quality of life. ETHICS AND DISSEMINATION: The trial received ethical approval from the Central Committee on Research Involving Human Subjects and from the local accredited Medical Research Ethics Committee of the region Arnhem-Nijmegen, the Netherlands (reference number: 2014-1433). Results will be published in a peer-reviewed journal and presented at international conferences. TRIAL REGISTRATION NUMBER: NCT02384122; Pre-results.
Assuntos
Anemia Refratária/tratamento farmacológico , Angiodisplasia/complicações , Hemorragia Gastrointestinal/etiologia , Octreotida/uso terapêutico , Projetos de Pesquisa , Padrão de Cuidado , Anemia Refratária/etiologia , Fármacos Gastrointestinais/uso terapêutico , Humanos , Intestino Delgado , Pessoa de Meia-Idade , Países Baixos , Resultado do TratamentoAssuntos
Anemia Refratária/complicações , Anemia Sideroblástica/complicações , Idoso , Anemia Refratária/diagnóstico , Anemia Refratária/tratamento farmacológico , Anemia Sideroblástica/diagnóstico , Anemia Sideroblástica/tratamento farmacológico , Terapia por Quelação/efeitos adversos , Desferroxamina/efeitos adversos , Desferroxamina/uso terapêutico , Diagnóstico Diferencial , Transfusão de Eritrócitos , Feminino , Ferritinas/sangue , Humanos , Quelantes de Ferro/efeitos adversos , Quelantes de Ferro/uso terapêutico , Sobrecarga de Ferro/etiologia , Sobrecarga de Ferro/prevenção & controle , Imageamento por Ressonância Magnética/métodos , Tomografia Computadorizada por Raios X , Resultado do TratamentoRESUMO
OBJECTIVE: To observe the clinical efficacy of Qinghuang Powder combined with Chinese herbs for reinforcing Shen and strenghening Pi in treating myelodysplastic syndrome (MDS). METHODS: fifty-five patients with diagnosis fitting to MDS were treated with Qinghuang Powder and decoction for strengthening Pi and reinforcing Shen, in combination with Stanozololum. RESULTS: Eleven patients (20.0%) out of the 55 were completely remitted (CR), the total effective rate being 74.5% (41/55 cases). By FAB typing, 9 (26. 5%) in the 34 patients of type RA/RAS were CR, with the total effective rate of 82.4% (28/34 cases), and in the 21 patients of type RAEB, 9.5% (2/21 cases) were CR with the total effective rate of 61.9% (13/21 cases), showing insignificant statistical difference between the two types (P > 0.05). By international prognostic scoring system (IPSS), the treatment was evaluated as CR in 10 patients, effective in 25 and ineffective in 11 in 36 patients of moderate risk group I , the responding numbers were 1, 4, 2 in 7 patients of moderate risk group II and 0, 3, 3 in 6 patients of high risk group, respectively, also showing insignificant difference between groups (P > 0.05). Levels of Hb, WBC and platelet significantly increased after treatment (P < 0. 05). By cytogenetics, the effective rate was 68.8% (11/16 cases) in patients with abnormal chromosome and 72.7% (24/33 cases) in those with normal chromosome, with insignificant difference (P > 0.05). CONCLUSION: The comprehensive therapy with TCM treatment for reinforcing Shen and dissolving stasis in dominance has a definite clinical effect in treating MDS, it was not significantly associated with FAB typing, IPSS score, and chromosome abnormality of patients.
Assuntos
Arsenicais/uso terapêutico , Medicamentos de Ervas Chinesas/uso terapêutico , Medicina Tradicional Chinesa , Síndromes Mielodisplásicas/tratamento farmacológico , Adolescente , Adulto , Idoso , Anemia Refratária/tratamento farmacológico , Diagnóstico Diferencial , Quimioterapia Combinada , Feminino , Humanos , Masculino , Pessoa de Meia-Idade , Síndromes Mielodisplásicas/diagnóstico , Síndromes Mielodisplásicas/fisiopatologia , Fitoterapia , Estanozolol/uso terapêutico , Resultado do Tratamento , Adulto JovemRESUMO
Myelodysplastic syndromes (MDSs) are a group of hematologic diseases that present unique challenges for oncology nurses, especially because patients with the disorders are being seen more often in oncology practices. An increasing array of therapeutic options are available, and the National Comprehensive Cancer Network published its first clinical practice guidelines for MDSs in 2004. This article provides oncology nurses with the most recent data on supportive care as well as emerging therapies for patients with low- to intermediate-risk MDS.
Assuntos
Síndromes Mielodisplásicas/tratamento farmacológico , Idoso , Algoritmos , Anemia Refratária/tratamento farmacológico , Anemia Refratária/terapia , Drogas em Investigação , Feminino , Humanos , Masculino , Síndromes Mielodisplásicas/diagnóstico , Síndromes Mielodisplásicas/enfermagem , Síndromes Mielodisplásicas/terapia , Guias de Prática Clínica como Assunto , Medição de RiscoRESUMO
Five, repeatedly transfused, patients with refractory anemia (RA) or RA with ringed sideroblast (RARS) subtypes of myelodysplastic syndrome (MDS), with serum ferritin (SF) levels of > 2,000 microg/L, and one female with Hb E [beta26(B8)Glu --> Lys]/beta0-thalassemia (thal) with an SF level of 1,760 microg/ L, were treated with deferiprone (L1) at the dose of 4-6 g per day for at least 26 months. Beginning in the second month, all patients received recombinant human erythropoietin (rHuEPO) at the dose of 150 IU/kg thrice weekly, subcutaneously for 24 months. A significant increase in iron excretion after combined administration of L1 and rHuEPO compared to treatment with L1 as a single agent, was observed in all patients. The amount of excreted iron in urine ranged from 7.5 to almost 20 mg per day. In one patient, a response to rHuEPO resulted in transfusion independence and her SF decreased from 2086 to 879 microg/L. In four MDS patients, who remained dependent on red blood cell (RBC) transfusions, simultaneous administration of L1 and rHuEPO enabled the stabilization of SF levels, despite continuing iron load from the transfusions. Combined administration of rHuEPO and oral iron chelators may potentiate mobilization of storage iron and maintain iron balance in transfusion-dependent iron overloaded early MDS patients.
Assuntos
Anemia Refratária/tratamento farmacológico , Eritropoetina/administração & dosagem , Quelantes de Ferro/administração & dosagem , Ferro/metabolismo , Síndromes Mielodisplásicas/tratamento farmacológico , Administração Oral , Anemia Refratária/diagnóstico , Deferiprona , Esquema de Medicação , Quimioterapia Combinada , Feminino , Ferritinas/sangue , Humanos , Injeções Subcutâneas , Quelantes de Ferro/uso terapêutico , Síndromes Mielodisplásicas/diagnóstico , Piridonas/administração & dosagem , Proteínas RecombinantesRESUMO
This study was aimed to examine whether a combination of all-trans retinoic acid (ATRA), 1, 25-dihydroxyvitamin D(3) and androgen possesses the therapeutic value for the MDS-refractory anemia (MDS-RA), and to analyze the mechanisms in detail. 62 cases receiving a scheme of combination of ATRA, 1, 25-dihydroxyvitamin D(3) and androgen (group A) were monitored. The remaining 33 cases (group B) were provided with vitamin supplementation, chalybeate drugs, and one or two of the combination. Bone marrow aspiration and biopsy were performed for collecting the specimens at the baseline and afterwards. The conditions of the patients were monitored by means of weekly complete blood counts and the monthly examination, including toxicity test, physical examination, electrocardiography, and biochemistry panel. The results showed that after treating for 8 weeks in group A, 4 out of 62 patients showed complete remission and 12 patients showed partial remission according to the defined response criteria, and 43 patients (69.35%) showed hematological improvement (HI). The further treatment for 16 out of 62 patients (25.81%), 13 failures (10 deaths, 2 RAEB and 1 RAEB-T) and 3 transformations (M(2), M(3), M(5)) with a median survival interval of 26.25 months, were observed and interrupted for some reasons. However, partial remission was observed only in 3 patients in group B, and HI amounted to 51.51%. Furthermore, the disease progression was observed in 12 out of 33 patients (36.36%) with a median survival interval of 16 months, 9 failures (including 6 deaths, 2 RAEB and 1 RAEB-T) and 3 transformations (M(2), M(3), M(4)). The overall ratios of survival for 3 and 5 years in group A, which received the combination, reached to 69.24% and 53.72% respectively, in comparison with 52.23% and 31.34% in the patients of group B (log-rank, P = 0.016). The following requirements, if were met, would be significant for prognosis: the combination regiment, no transformation, children, no complication, female, 90-120 g/L of hemoglobin concentration, normal cellular bone marrow and uni-cytopenias (P < 0.05). Moreover, Cox regression showed that therapy, transformation and age are all the independent factors (P < 0.05). It is concluded that the combination of above mentioned 3 drugs may be effective and safe treatment for the patients with MDS-RA. Its relevant mechanisms can be involved in the combination, that elicits a wide range of pharmacological effects, such as differentiation, anti-tumor-promotion, anti-apoptosis, anti-angiogenesis, anti-cachexia and immunoregulation.
Assuntos
Androgênios/uso terapêutico , Anemia Refratária/tratamento farmacológico , Calcitriol/uso terapêutico , Tretinoína/uso terapêutico , Adolescente , Adulto , Idoso , Idoso de 80 Anos ou mais , Androgênios/administração & dosagem , Anemia Refratária/sangue , Contagem de Células Sanguíneas , Calcitriol/administração & dosagem , Criança , Pré-Escolar , Quimioterapia Combinada , Feminino , Humanos , Masculino , Pessoa de Meia-Idade , Síndromes Mielodisplásicas/sangue , Síndromes Mielodisplásicas/tratamento farmacológico , Estudos Retrospectivos , Análise de Sobrevida , Fatores de Tempo , Resultado do Tratamento , Tretinoína/administração & dosagemRESUMO
20 cases of myelodysplastic syndrome (MDS) patients were treated with Yisuikang (YSK) including MDS-RA 17 cases, MDS-RAEB 2 cases, MDS-RAEB-T 1 case. 14 patients were treated with YSK only, 6 cases were treated with both YSK and Western medicine. The result showed that 4 cases were remitted, 5 cases markedly improved, 6 cases improved and 5 cases ineffective, the total effective rate was 75.0%. It was found that YSK was effective in the treatment of MDS-RA or Qi-Blood Deficiency and Yu-Re Syndrome in TCM, while any other types were ineffective. They also found that types of TCM might be correlated with MDS.
Assuntos
Diagnóstico Diferencial , Medicamentos de Ervas Chinesas/uso terapêutico , Medicina Tradicional Chinesa , Síndromes Mielodisplásicas/tratamento farmacológico , Adolescente , Adulto , Anemia Refratária/tratamento farmacológico , Anemia Refratária com Excesso de Blastos/tratamento farmacológico , Cápsulas , Feminino , Humanos , Masculino , Pessoa de Meia-IdadeRESUMO
Different response patterns to recombinant human erythropoietin (rHuEpo) administration to anemic patients with myelodysplastic syndromes (MDS) are described. The biology of rHuEpo effect on erythropoiesis in patients with MDS has not been elucidated. However, until more biological information is obtained, it could be prudent to consider these response patterns as guidelines in the treatment of MDS. In the small but interesting series of nine patients with MDS only one responded to rHuEpo within the treatment period of eight weeks. Two additional patients continued the treatment on their own, and after 16 weeks a response was noted for the first time. A third patient was treated for only six weeks and a delayed response was recorded while off treatment for ten weeks. This response was also recorded 16 weeks from treatment initiation-as in the other two patients. A fourth patient with MDS developed transfusion related hemosiderosis and during iron chelation therapy the RBC transfusion rate dropped to a rate lower than the rate needed before the rHuEpo treatment. It is emphasized that in non responders, non-routine approaches should be considered.
Assuntos
Eritropoetina/uso terapêutico , Síndromes Mielodisplásicas/tratamento farmacológico , Idoso , Idoso de 80 Anos ou mais , Anemia Refratária/tratamento farmacológico , Anemia Sideroblástica/tratamento farmacológico , Transfusão de Sangue/estatística & dados numéricos , Terapia por Quelação , Terapia Combinada , Desferroxamina/uso terapêutico , Contagem de Eritrócitos/efeitos dos fármacos , Eritropoetina/efeitos adversos , Eritropoetina/farmacologia , Feminino , Seguimentos , Hemocromatose/etiologia , Hemocromatose/terapia , Humanos , Masculino , Síndromes Mielodisplásicas/sangue , Síndromes Mielodisplásicas/terapia , Mielofibrose Primária/tratamento farmacológico , Proteínas Recombinantes/efeitos adversos , Proteínas Recombinantes/farmacologia , Proteínas Recombinantes/uso terapêutico , Indução de Remissão , Reação Transfusional , Resultado do TratamentoAssuntos
Eritropoetina/uso terapêutico , Anemia/fisiopatologia , Anemia Refratária/tratamento farmacológico , Anemia Refratária/etiologia , Preservação de Sangue , Transfusão de Sangue Autóloga , Eritropoese , Eritropoetina/efeitos adversos , Eritropoetina/biossíntese , Eritropoetina/fisiologia , Doenças Hematológicas/tratamento farmacológico , Humanos , Inflamação/tratamento farmacológico , Neoplasias/sangue , Neoplasias/complicações , Receptores da Eritropoetina/fisiologia , Proteínas Recombinantes/farmacocinética , Diálise RenalRESUMO
We have used recombinant human erythropoietin (rHuEPO) in a phase I/II clinical trial to evaluate its ability to reverse refractory anemia in hematologic disorders. rHuEPO was administered subcutaneously 5 days per week at escalating doses (50 to 150 U/kg per day). The aim of treatment was a hemoglobin (Hb) level greater than or equal to 10 g/dL without blood transfusion. Of 25 patients treated, 17 were evaluable, most of them with a regular need for transfusion. Eight of these had lymphoproliferative disorders (three cases of malignant lymphoma and five of monoclonal gammopathy) and were exposed to cytotoxic therapy. The other nine patients had hematopoietic stem cell disorders (four cases of myelodysplastic syndrome, three of idiopathic myelofibrosis, and two of chronic myelogenous leukemia). All patients with lymphoproliferative disorder had serum EPO levels inappropriately low for the degree of anemia, while patients with stem cell disorder showed variable values. Erythroid marrow activity was inadequate in all cases. Seven of eight patients with lymphoproliferative disorder responded to treatment maintaining Hb above 10 g/dL without transfusion. The median dose of rHuEPO required for correction of anemia was 75 U/kg. In four cases response was maintained with 50 U/kg, three times per week. There was no complete response among patients with hematopoietic stem cell disorder, although transfusion requirement was eliminated or reduced in four cases. Four patients developed functional iron deficiency during rHuEPO treatment and required iron supplementation to obtain response. Aggravation of splenomegaly was observed in two cases of myeloproliferative disorder. We conclude that: (1) subcutaneous administration of rHuEPO can be effective and safe in patients with lymphoproliferative disorder exposed to chemotherapy and showing inappropriate EPO response to anemia; (2) this is less likely in hematopoietic stem cell disorders, although favorable responses may be observed in occasional patients; and (3) functional iron deficiency as a cause of nonresponse to rHuEPO is frequent also in nonrenal anemia.