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1.
Acta Haematol ; 146(5): 408-412, 2023.
Artigo em Inglês | MEDLINE | ID: mdl-37231772

RESUMO

We describe a case of acute myeloid leukemia with NPM1 mutation and disseminated leukemia cutis in a very old patient, who achieved a long-lasting response to the azacitidine/venetoclax combination with molecular complete remission, given the potential value of this rarely observed clinical outcome.


Assuntos
Leucemia Mieloide Aguda , Humanos , Leucemia Mieloide Aguda/diagnóstico , Leucemia Mieloide Aguda/tratamento farmacológico , Leucemia Mieloide Aguda/genética , Proteínas Nucleares/genética , Azacitidina/uso terapêutico , Mutação , Protocolos de Quimioterapia Combinada Antineoplásica/uso terapêutico
2.
Chemotherapy ; 66(3): 78-81, 2021.
Artigo em Inglês | MEDLINE | ID: mdl-34102636

RESUMO

An unmet clinical need currently exists for elderly patients with relapsed/resistant (R/R) Philadelphia (Ph) positive acute lymphoblastic leukemia (ALL), nearly all who have a very poor prognosis. This includes patients already exposed to the first or second generation tyrosine kinase inhibitors (TKIs) and therefore has few treatment options available. New immunotherapies and targeted agents have shown encouraging activity in R/R ALL irrespective of age. Inotuzumab (InO), a humanized anti-CD22 monoclonal antibody, has potentially beneficial clinical effects in patients with resistant and difficult-to-treat disease in whom prior TKIs have failed. However, InO, as a single agent, did not show durable response and longer progression free survival and overall survival in R/R Ph positive ALL patients compared with those treated with standard chemotherapy. We observed a durable molecular remission (7 months) in an elderly patient affected by Ph'+ ALL with T315I and concomitant p190 and p210 expression achieved by concomitant therapy of InO (for 4 cycles) and ponatinib (15 mg/daily) followed by ponatinib (15 mg/daily) only as maintenance therapy. These findings suggest that elderly R/R Ph positive patients who cannot proceed to the transplant might benefit by concomitant immunotherapy and TKIs aimed to deepen the responses and prolong CR and outcomes.


Assuntos
Anticorpos Monoclonais Humanizados/uso terapêutico , Proteínas de Fusão bcr-abl/genética , Imidazóis/uso terapêutico , Cromossomo Filadélfia , Leucemia-Linfoma Linfoblástico de Células Precursoras/tratamento farmacológico , Inibidores de Proteínas Quinases/uso terapêutico , Piridazinas/uso terapêutico , Idoso , Proteínas de Fusão bcr-abl/metabolismo , Humanos , Masculino , Polimorfismo de Nucleotídeo Único , Leucemia-Linfoma Linfoblástico de Células Precursoras/genética , Recidiva , Resultado do Tratamento
3.
Hematol Oncol ; 38(2): 189-196, 2020 Apr.
Artigo em Inglês | MEDLINE | ID: mdl-31891213

RESUMO

Pulmonary infections (PIs) are a major complication of patients with myelodysplastic syndromes (MDS). We retrospectively evaluated 234 MDS patients treated with azacytidine (AZA). The total number of AZA cycles was 2886 (median 8 cycles per patient). There were 111 episodes of PI (3.8% of AZA cycles) in 81 patients (34.6%). PIs were considered of fungal origin in 27 cases (24.3%), associated to bacteremia in 11 cases (9.9%), to influenza infection in two cases (1.8%) and of unknown origin in the remaining 71 cases (64.0%). Forty-five PI episodes were documented in cycles 1 to 4 of AZA (5.1% of 875 cycles) and the remaining 66 episodes beyond the fourth cycle (3.2% of 2011 cycles) (P = .017). Overall, a fungal PI was documented in 13/875 (1.5%) cycles 1 to 4 and in 13/2011 (0.6%) cycles beyond the fourth cycle (P = .001). A baseline chronic pulmonary disease was significantly associated to a higher risk of severe PIs. In the survival analysis, cases of PI in patients who progressed to acute leukemia (PAL) were excluded, in view of the predominant influence of PAL on the outcome of the patients. A PI unrelated to PAL documented during the first 4 AZA cycles was an independent factor predicting lower survival (OR, 2.13; 95% CI, 1.37-3.33; P = .001). In conclusion, PIs are common in MDS patients receiving AZA, in particular during the first cycles of treatment and are associated with an unfavorable outcome. The results of our study raise the issue of the need of a tailored infection prevention strategy.


Assuntos
Antimetabólitos Antineoplásicos/efeitos adversos , Azacitidina/efeitos adversos , Pulmão/microbiologia , Síndromes Mielodisplásicas/tratamento farmacológico , Infecções Respiratórias/etiologia , Idoso , Feminino , Seguimentos , Humanos , Pulmão/efeitos dos fármacos , Masculino , Pessoa de Meia-Idade , Síndromes Mielodisplásicas/patologia , Prognóstico , Infecções Respiratórias/induzido quimicamente , Estudos Retrospectivos , Taxa de Sobrevida
4.
Hematol Oncol ; 37(3): 291-295, 2019 Aug.
Artigo em Inglês | MEDLINE | ID: mdl-31050810

RESUMO

There have been some reports on a possible role of azacytidine (AZA) in the treatment of accelerated/blastic phase evolved from Philadelphia-negative myeloproliferative neoplasms (MPN-AP/BP), but results are conflicting. In this study, we analyzed a cohort of 39 patients with MPN-AP/BP treated frontline with AZA at the standard dosage (75 mg/m2 ). Median time from diagnosis to AP/BP evolution was 92.3 months (IR 29.9-180.1). All patients were evaluable for hematologic response: two patients (5.2%) died early after AZA initiation, 13 patients (33.3%) had a progressive or stable disease, nine (23.1%) had a hematologic improvement (HI), seven (17.9%) achieved a partial response (PR), and eight (20.5%) a complete response (CR). Overall, 24 patients achieved a clinical hematologic response (HI + PR + CR), with an overall response rate of 61.5%. Median overall survival (OS) from AZA start of the whole cohort was 13.5 months (95% CI, 8.2-18.7). There was no difference in median OS among patients with HI, PR, or CR (P = .908). These three subgroups as "responders" having been considered, a significantly better OS was observed in responder compared with nonresponder patients, with a median OS of 17.6 months (95% CI, 10.1-25.0) versus 4.1 months (95% CI, 0.4-10.0) (P = .001) Only female gender was significant for both achievement of response (.010) and OS duration (P = .002). In conclusion, AZA is useful for the management of MPN-AP/BP, with an overall response rate (HI + PR + CR) of 61.5% and a longer OS in responders.


Assuntos
Antimetabólitos Antineoplásicos/uso terapêutico , Azacitidina/uso terapêutico , Crise Blástica/tratamento farmacológico , Transtornos Mieloproliferativos/tratamento farmacológico , Idoso , Crise Blástica/diagnóstico , Feminino , Humanos , Hidroxiureia/uso terapêutico , Leucemia Mieloide Crônica Atípica BCR-ABL Negativa/diagnóstico , Leucemia Mieloide Crônica Atípica BCR-ABL Negativa/tratamento farmacológico , Masculino , Melfalan/uso terapêutico , Pessoa de Meia-Idade , Mutação , Transtornos Mieloproliferativos/diagnóstico , Pipobromano/uso terapêutico , Policitemia Vera/diagnóstico , Policitemia Vera/tratamento farmacológico , Mielofibrose Primária/diagnóstico , Mielofibrose Primária/tratamento farmacológico , Prognóstico , Indução de Remissão , Estudos Retrospectivos , Trombocitemia Essencial/diagnóstico , Trombocitemia Essencial/tratamento farmacológico , Resultado do Tratamento
6.
Value Health ; 22(11): 1303-1310, 2019 11.
Artigo em Inglês | MEDLINE | ID: mdl-31708068

RESUMO

OBJECTIVES: We investigated the validity of the recently developed European Organisation for Research and Treatment of Cancer Quality of Life Questionnaire Core 30 (EORTC QLQ-C30) summary score in patients with hematologic malignancies. Specifically, we evaluated the adequacy of a single-factor measurement model for the QLQ-C30, and its known-groups validity and responsiveness to change over time. METHODS: We used confirmatory factor analysis to test the single-factor model of the QLQ-C30, using baseline QLQ-C30 data (N = 2134). The QLQ-C30 summary score was compared to the original QLQ-C30 scales using general (age, sex, Eastern Cooperative Oncology Group performance status, comorbidity) and disease-specific (red blood cell transfusion dependency) groups. Repeated measurements allowed us to investigate responsiveness to change in a subgroup of patients with acute myeloid leukemia. RESULTS: The single-factor model of the QLQ-C30 exhibited adequate fit in patients with hematologic malignancies. Known-group comparisons generally supported the construct validity of the summary score when using more general grouping variables (sociodemographics, broad clinical parameters). Nevertheless, when groups were formed on the basis of disease-specific variables (eg, transfusion dependency), the summary score performed less well the some of the original, separate scales of the QLQ-C30. CONCLUSION: Our findings provide support for the validity of the single-factor model of the EORTC QLQ-C30 in patients with hematologic malignancies. Specifically, the results suggest that the summary score can be used as an endpoint in this population when symptom- or other health domain-specific hypotheses are not available.


Assuntos
Neoplasias Hematológicas/psicologia , Qualidade de Vida , Inquéritos e Questionários/normas , Adulto , Idoso , Europa (Continente) , Análise Fatorial , Feminino , Nível de Saúde , Humanos , Relações Interpessoais , Masculino , Saúde Mental , Pessoa de Meia-Idade , Desempenho Físico Funcional , Reprodutibilidade dos Testes
7.
Support Care Cancer ; 27(8): 2755-2756, 2019 Aug.
Artigo em Inglês | MEDLINE | ID: mdl-31053971

RESUMO

This commentary deals with the need of an early integration between hematologist and palliative care specialists as well as pain therapists as a routine basis in order to ensure the best management of patients affected by acute leukemia from the onset of the disease and in the stages of causal therapy. This strategy could limit the burden of painful symptoms and, in addition, avoid unnecessary suffering to patients, ensuring the best conditions for optimal outcome of these patients with extremely high clinical complexity and symptomatology who receive intensive treatments or who are managed with novel treatment approaches.


Assuntos
Dor do Câncer/complicações , Dor do Câncer/terapia , Hematologia/métodos , Leucemia Mieloide Aguda/terapia , Oncologia/métodos , Manejo da Dor/métodos , Cuidados Paliativos/métodos , Dor do Câncer/epidemiologia , Hematologia/organização & administração , Hematologia/normas , Humanos , Comunicação Interdisciplinar , Leucemia Mieloide Aguda/complicações , Oncologia/organização & administração , Oncologia/normas , Dor/complicações , Dor/etiologia , Manejo da Dor/efeitos adversos , Equipe de Assistência ao Paciente/organização & administração , Equipe de Assistência ao Paciente/normas
8.
Support Care Cancer ; 27(3): 793-803, 2019 Mar.
Artigo em Inglês | MEDLINE | ID: mdl-30564934

RESUMO

INTRODUCTION: High-dose melphalan (HDMel) is the most common conditioning chemotherapy regimen for autologous stem cell transplantation (SCT) in patients affected by multiple myeloma (MM). No consensus exists for the emetogenicity or prophylaxis of chemotherapy-induced nausea and vomiting (CINV) in this regimen. METHODS: Data on the incidence and efficacy/safety of CINV prophylaxis among patients affected by MM undergoing autologous SCT with the HDMel regimen was extracted from electronic databases and analyzed. RESULTS: Eleven studies involving multiple CINV prophylaxis regimens were identified and included. No consensus on HDMel emetogenicity was reached, but most studies summarized the emetogenicity as moderate-high risk. An aprepitant-based three-drug regimen (aprepitant + serotonin receptor antagonist (5HT3RA) + dexamethasone) showed better efficacy than a two-drug regimen (5HT3RA + dexamethasone) for CINV prevention without increasing the frequency in adverse events. CONCLUSIONS: The aprepitant-based three-drug regimen should be the regimen of choice for CINV prophylaxis for MM patients undergoing autologous SCT with HDMel conditioning.


Assuntos
Antineoplásicos Alquilantes/efeitos adversos , Transplante de Células-Tronco Hematopoéticas/efeitos adversos , Melfalan/efeitos adversos , Náusea/prevenção & controle , Vômito/prevenção & controle , Adulto , Antieméticos/uso terapêutico , Antineoplásicos Alquilantes/administração & dosagem , Aprepitanto/administração & dosagem , Dexametasona/administração & dosagem , Feminino , Humanos , Quimioterapia de Indução , Masculino , Melfalan/administração & dosagem , Mieloma Múltiplo/tratamento farmacológico , Náusea/etiologia , Qualidade de Vida , Antagonistas da Serotonina/administração & dosagem , Antagonistas da Serotonina/uso terapêutico , Condicionamento Pré-Transplante/efeitos adversos , Condicionamento Pré-Transplante/métodos , Transplante Autólogo , Vômito/etiologia
9.
Cancer ; 124(6): 1251-1259, 2018 03 15.
Artigo em Inglês | MEDLINE | ID: mdl-29231969

RESUMO

BACKGROUND: Current prognostic systems for myelodysplastic syndromes (MDS) are based on clinical, pathologic, and laboratory indicators. The objective of the current study was to develop a new patient-centered prognostic index for patients with advanced MDS by including self-reported fatigue severity into a well-established clinical risk classification: the International Prognostic Scoring System (IPSS). METHODS: A total of 469 patients with advanced (ie, IPSS intermediate-2 or high-risk) MDS were analyzed. Untreated patients (280 patients) were recruited into an international prospective cohort observational study to create the index. The index then was applied to an independent cohort including pretreated patients with MDS from the Dana-Farber Cancer Institute in Boston, Massachusetts (189 patients). At baseline, patients completed the European Organization for Research and Treatment of Cancer Quality of Life Questionnaire-Core 30 (EORTC QLQ-C30). RESULTS: A new prognostic index was developed: the FA-IPSS(h), in which FA stands for fatigue and h for higher-risk. This new risk classification enabled the authors to distinguish 3 subgroups of patients with distinct survival outcomes (ie, risk-1, risk-2, and risk-3). Patients classified as FA-IPSS(h) risk-1 had a median overall survival (OS) of 23 months (95% confidence interval [95% CI], 19-29 months), whereas those with risk-2 had a median OS of 16 months (95% CI, 12-17 months) and those with risk-3 had a median OS of 10 months (95% CI, 4-13 months). The predictive accuracy of this new index was higher than that of the IPSS alone in both the development cohort as well as in the independent cohort including pretreated patients. CONCLUSIONS: The FA-IPSS(h) is a novel patient-centered prognostic index that includes patients' self-reported fatigue severity. The authors believe its use might enhance physicians' ability to predict survival more accurately in patients with advanced MDS. Cancer 2018;124:1251-9. © 2017 American Cancer Society.


Assuntos
Síndromes Mielodisplásicas/mortalidade , Medidas de Resultados Relatados pelo Paciente , Qualidade de Vida , Autorrelato , Adulto , Idoso , Idoso de 80 Anos ou mais , Terapia Combinada , Feminino , Seguimentos , Humanos , Agências Internacionais , Masculino , Pessoa de Meia-Idade , Síndromes Mielodisplásicas/patologia , Síndromes Mielodisplásicas/terapia , Prognóstico , Estudos Prospectivos , Fatores de Risco , Taxa de Sobrevida
10.
Health Qual Life Outcomes ; 16(1): 127, 2018 Jun 18.
Artigo em Inglês | MEDLINE | ID: mdl-29914509

RESUMO

BACKGROUND: Treatment decision-making in patients with relapsed/refractory multiple myeloma (RRMM) is challenging for a number of reasons including, the heterogeneity of disease at relapse and the number of possible therapeutic approaches. This study broadly aims to generate new evidence-based data to facilitate clinical decision-making in RRMM patients. The primary objective is to investigate the prognostic value of patient self-reported fatigue severity for overall survival. METHODS: This multicenter prospective observational study will consecutively enroll 312 patients with multiple myeloma who have received at least 1 prior line of therapy and are considered as RRMM according to the International Myeloma Working Group (IMWG) criteria. Eligible RRMM participants will be adults (≥ 18 years old) patients and will be enrolled irrespective of comorbidities and performance status. At the time of study inclusion, data to calculate the frailty score are to be available. Patients will be followed up for 30 months and patient-reported outcome (PRO) assessment is planned at baseline and thereafter at 3, 6, 12, and 24 months. The following PRO validated questionnaires will be used: the European Organisation for the Research and Treatment of Cancer Quality of Life Questionnaire-Core 30 (EORTC QLQ-C30), the EORTC QLQ-MY20 and the EORTC QLQ-INFO25. Satisfaction with care and preference for involvement in treatment decisions will also be evaluated. Clinical, laboratory and treatment related information will be prospectively collected in conjunction with pre scheduled PRO assessments. Cox regression analyses will be used to assess the prognostic value of baseline fatigue severity (EORTC QLQ-C30) and other patient-reported health-related quality of life parameters. DISCUSSION: Clinical decision-making in RRMM is a challenge and outcome prediction is also an important aspect to enhance personalized treatment planning. Given the paucity of PRO data in this population, this prospective observational study aims to provide novel information that may facilitate patients' management in routine practice. TRIAL REGISTRATION: This trial is registered as identifier NCT03190525 .


Assuntos
Tomada de Decisão Clínica , Protocolos Clínicos , Mieloma Múltiplo/psicologia , Qualidade de Vida/psicologia , Feminino , Humanos , Masculino , Mieloma Múltiplo/terapia , Recidiva Local de Neoplasia/psicologia , Medidas de Resultados Relatados pelo Paciente , Satisfação Pessoal , Prognóstico , Estudos Prospectivos
11.
Support Care Cancer ; 26(6): 1699-1701, 2018 Jun.
Artigo em Inglês | MEDLINE | ID: mdl-29502153

RESUMO

Current evidence, although limited, outlines that sexual dysfunction may represent a prominent part of the symptom burden experienced by the patients with hematologic malignancies (HM). However, despite their presumed negative effects on quality of life (QoL), sexual health is not typically considered in the QoL assessment of HM patients. In addition, very few studies have been conducted in this area. Therefore, it would be important to further investigate how newer drugs developed in recent years for patients with HM, including targeted therapies and impact on sexual health, and how this influence overall patients' QoL outcomes.


Assuntos
Neoplasias Hematológicas/complicações , Neoplasias Hematológicas/psicologia , Qualidade de Vida/psicologia , Disfunções Sexuais Fisiológicas/patologia , Saúde Sexual/tendências , Adulto , Humanos , Masculino , Pessoa de Meia-Idade
12.
Support Care Cancer ; 26(7): 2075-2090, 2018 Jul.
Artigo em Inglês | MEDLINE | ID: mdl-29560502

RESUMO

PURPOSE: We performed a systematic review to quantify the amount of evidence-based data available on patient-reported outcomes (PRO) in Relapsed/Refractory Multiple Myeloma (RRMM) patients and to examine the added value of such studies in supporting clinical decision-making. METHODS: We conducted a search in PubMed/Medline and the Cochrane Library to identify studies published between January 1990 and May 2017. All studies, regardless of the design, including patients with RRMM and also evaluating PRO were considered. For each study, we collected both PRO and traditional clinical outcomes, such as survival and toxicity information, based on a predefined data extraction form. RESULTS: After having screened 1680 records, 11 studies were identified and these included six randomized controlled trials (RCT). Overall, there were five studies focusing on proteasome inhibitors (PIs), four on immunomodulatory drugs (IMiDs), one on both PIs and IMiDs, and one on monoclonal antibodies. Considering only RCTs, it was found that primary clinical efficacy endpoints frequently favored experimental arms, while (physician-reported) toxicity data did not. However, inspection of PRO data revealed novel information that often contrasted with standard toxicity, for example, by not indicating worse quality of life outcomes or symptom severity for patients enrolled in the experimental arms. CONCLUSIONS: There is paucity of evidence-based data regarding the impact of therapies on quality of life and symptom burden of patients with RRMM. Inclusion of PRO in future studies of patients with RRMM is needed to better inform clinical decision-making.


Assuntos
Tomada de Decisão Clínica/métodos , Mieloma Múltiplo/tratamento farmacológico , Medidas de Resultados Relatados pelo Paciente , Qualidade de Vida , Anticorpos Monoclonais/uso terapêutico , Humanos , Imunomodulação/efeitos dos fármacos , Inibidores de Proteassoma/uso terapêutico , Resultado do Tratamento
13.
Br J Haematol ; 177(5): 741-750, 2017 06.
Artigo em Inglês | MEDLINE | ID: mdl-28419408

RESUMO

Iron chelation is controversial in higher risk myelodysplastic syndromes (HR-MDS), outside the allogeneic transplant setting. We conducted a retrospective, multicentre study in 51 patients with transfusion-dependent, intermediate-to-very high risk MDS, according to the revised international prognostic scoring system, treated with the oral iron chelating agent deferasirox (DFX). Thirty-six patients (71%) received azacitidine concomitantly. DFX was given at a median dose of 1000 mg/day (range 375-2500 mg) for a median of 11 months (range 0·4-75). Eight patients (16%) showed grade 2-3 toxicities (renal or gastrointestinal), 4 of whom (8%) required drug interruption. Median ferritin levels decreased from 1709 µg/l at baseline to 1100 µg/l after 12 months of treatment (P = 0·02). Seventeen patients showed abnormal transaminase levels at baseline, which improved or normalized under DFX treatment in eight cases. One patient showed a remarkable haematological improvement. At a median follow up of 35·3 months, median overall survival was 37·5 months. The results of this first survey of DFX in HR-MDS are comparable, in terms of safety and efficacy, with those observed in lower-risk MDS. Though larger, prospective studies are required to demonstrate real clinical benefits, our data suggest that DFX is feasible and might be considered in a selected cohort of HR-MDS patients.


Assuntos
Benzoatos/uso terapêutico , Terapia por Quelação/métodos , Quelantes de Ferro/uso terapêutico , Síndromes Mielodisplásicas/tratamento farmacológico , Triazóis/uso terapêutico , Administração Oral , Adulto , Idoso , Idoso de 80 Anos ou mais , Deferasirox , Transfusão de Eritrócitos/estatística & dados numéricos , Feminino , Ferritinas/metabolismo , Humanos , Masculino , Pessoa de Meia-Idade , Estudos Retrospectivos , Resultado do Tratamento
14.
Blood ; 124(19): 2930-6, 2014 Nov 06.
Artigo em Inglês | MEDLINE | ID: mdl-25232059

RESUMO

The clinical outcome, response to treatment, and occurrence of acute complications were retrospectively investigated in 308 primary autoimmune hemolytic anemia (AIHA) cases and correlated with serological characteristics and severity of anemia at onset. Patients had been followed up for a median of 33 months (range 12-372); 60% were warm AIHA, 27% cold hemagglutinin disease, 8% mixed, and 5% atypical (mostly direct antiglobulin test negative). The latter 2 categories more frequently showed a severe onset (hemoglobin [Hb] levels ≤6 g/dL) along with reticulocytopenia. The majority of warm AIHA patients received first-line steroid therapy only, whereas patients with mixed and atypical forms were more frequently treated with 2 or more therapy lines, including splenectomy, immunosuppressants, and rituximab. The cumulative incidence of relapse was increased in more severe cases (hazard ratio 3.08; 95% confidence interval, 1.44-6.57 for Hb ≤6 g/dL; P < .001). Thrombotic events were associated with Hb levels ≤6 g/dL at onset, intravascular hemolysis, and previous splenectomy. Predictors of a fatal outcome were severe infections, particularly in splenectomized cases, acute renal failure, Evans syndrome, and multitreatment (4 or more lines). The identification of severe and potentially fatal AIHA in a largely heterogeneous disease requires particular experienced attention by clinicians.


Assuntos
Anemia Hemolítica Autoimune/tratamento farmacológico , Anemia Hemolítica Autoimune/imunologia , Autoanticorpos/imunologia , Eritropoetina/uso terapêutico , Imunoglobulinas Intravenosas/uso terapêutico , Esteroides/uso terapêutico , Adulto , Idoso , Anemia Hemolítica Autoimune/cirurgia , Anticorpos Monoclonais Murinos/uso terapêutico , Feminino , Humanos , Fatores Imunológicos/uso terapêutico , Imunossupressores/uso terapêutico , Masculino , Pessoa de Meia-Idade , Valor Preditivo dos Testes , Estudos Retrospectivos , Rituximab , Índice de Gravidade de Doença , Esplenectomia , Resultado do Tratamento , Adulto Jovem
15.
Ann Hematol ; 95(7): 1059-65, 2016 Jun.
Artigo em Inglês | MEDLINE | ID: mdl-27091349

RESUMO

The Gruppo Romano Mielodisplasie (GROM) conducted a retrospective study in 543 patients with myelodysplastic syndromes (MDS) to evaluate the safety and efficacy of erythropoiesis-stimulating agents (ESAs) in "real-life" clinical practice. The 40.000-UI/week erythropoietin (EPO)-alpha and 30.000-UI/week EPO-beta starting dose were defined "standard," and 80,000 UI/week EPO-alpha and 60.000 UI/week EPO-beta were defined "high." Response was defined according to International Working Group (IWG) 2006 criteria. At ESA's start, median age was 74.2 years (interquartile range (IR) 67.8-79.5) and median hemoglobin was 8.9 g/dl (IR 8.2-9.6). Median time from diagnosis to ESAs start was 3.8 months (IR 0.8-13.2). ESA starting dose was "standard" in 361 patients (66.5 %) and "high" in 182 patients (33.5 %). Erythroid response was observed in 82/185 (44.3 %) transfusion dependent (TD) patients as compared with 226/329 (68.6 %) transfusion independent (TI) ones (p < 0.001). At multivariate analysis, in TD patients, only endogenous EPO levels <50 mU/l were significant (p = 0.046), whereas in TI patients, high-dose ESAs (p < 0.001), abnormal creatinine levels (0.009), and endogenous EPO levels <50 mU/l (p = 0.014) were predictors of response. Responders showed a higher 5-year overall survival (OS) (57.8 vs. 32.2 %, p < 0.001) and leukemia-free survival (76.0 vs. 49.8 %, p < 0.001). At multivariable analysis for OS, response to ESA, low International Prognostic Scoring System (IPSS), no transfusion need, and female sex showed an independent favorable prognostic role. Our results confirm that treatment with ESAs is effective in a real-life MDS setting, particularly at high dose and in TI patients. Prospective studies are needed to define the optimal starting dose.


Assuntos
Hematínicos/uso terapêutico , Síndromes Mielodisplásicas/diagnóstico , Síndromes Mielodisplásicas/tratamento farmacológico , Idoso , Idoso de 80 Anos ou mais , Feminino , Seguimentos , Humanos , Itália/epidemiologia , Masculino , Síndromes Mielodisplásicas/mortalidade , Estudos Retrospectivos , Taxa de Sobrevida/tendências
16.
Eur J Haematol ; 96(6): 643-9, 2016 Jun.
Artigo em Inglês | MEDLINE | ID: mdl-26277477

RESUMO

At present, very few data are available on deferasirox (DFX) in the treatment of patients with Philadelphia-negative myeloproliferative neoplasms in fibrotic phase (FP-MPN) and transfusion dependence. To address this issue, a retrospective analysis of 28 patients (22 male and 6 female) with FP-MPN and iron overload secondary to transfusion dependence was performed, based on patients enrolled in the database of our regional cooperative group who received treatment with DFX. DFX was started after a median interval from diagnosis of 12.8 months (IR 7.1-43.1) with median ferritin values of 1415 ng/mL (IR 1168-1768). Extra-hematological toxicity was reported in 16 of 28 patients (57.1%), but only two patients discontinued treatment due to toxicity. Among 26 patients evaluable for response (≥6 months of treatment), after a median treatment period of 15.4 months (IR 8.1-22.3), 11 patients (42.3%) achieved a stable and consistent reduction in ferritin levels <1000 ng/mL. As for hematological improvement, 6 of 26 patients (23%) showed a persistent (>3 months) rise of Hb levels >1.5 g/dL, with disappearance of transfusion dependence in four cases. Treatment with DFX is feasible and effective in FP-MPN with iron overload. Moreover, in this setting, an erythroid response can occur in a significant proportion of patients.


Assuntos
Benzoatos/uso terapêutico , Eritropoese/efeitos dos fármacos , Quelantes de Ferro/uso terapêutico , Sobrecarga de Ferro/tratamento farmacológico , Sobrecarga de Ferro/etiologia , Transtornos Mieloproliferativos/complicações , Transtornos Mieloproliferativos/patologia , Triazóis/uso terapêutico , Idoso , Benzoatos/administração & dosagem , Benzoatos/efeitos adversos , Terapia por Quelação , Deferasirox , Índices de Eritrócitos , Feminino , Fibrose , Seguimentos , Humanos , Quelantes de Ferro/administração & dosagem , Quelantes de Ferro/efeitos adversos , Sobrecarga de Ferro/sangue , Sobrecarga de Ferro/diagnóstico , Masculino , Pessoa de Meia-Idade , Transtornos Mieloproliferativos/mortalidade , Transtornos Mieloproliferativos/terapia , Estudos Retrospectivos , Reação Transfusional , Resultado do Tratamento , Triazóis/administração & dosagem , Triazóis/efeitos adversos
17.
Eur J Haematol ; 96(4): 344-51, 2016 Apr.
Artigo em Inglês | MEDLINE | ID: mdl-26018238

RESUMO

OBJECTIVE: Azacitidine is the standard of care for higher-risk myelodysplastic syndromes (MDS). We evaluated factors affecting the outcome of azacitidine treatment in 196 'real-world' patients, retrospectively collected by two Italian cooperative groups. METHODS: The study included 184 MDS and 12 low blast count acute myeloid leukemia (AML). Azacitidine was administered at the standard dose of 75 mg/m(2)/d for 7 d (SD) in 163 patients and 100 mg/d for 5-7 d in 33 patients. RESULTS: After a median of 4.5 azacitidine cycles (range 7-15 cycles), 182 patients were evaluable for response. Nineteen percent achieved complete remission (CR), 17% partial remission (PR), and 21% hematological improvement (HI). The disease was stable or progressive in 29% and 14% of patients, respectively. The probability of response was significantly higher in patients who received the 75 mg/m(2)/7 d compared with 100 mg through 5-7 d dose (CR/PR/HI: 63 vs. 29%, P = 0.0005). Median overall survival was 17.1 months. Low MDS-CI and achievement of CR/PR/HI were significant predictors of survival in the multivariable analysis. CONCLUSIONS: Our data show that maximal azacitidine efficacy is associated with the standard dose and with prolonged treatment, beyond 4-6 cycles, with the goal of also improving the 'quality' of response. Lower MDS-CI and IPSS-R scores, hematologic response and disease stability, are associated with longer survival. The risk of febrile events is highest during the first treatment cycles and is associated with active disease.


Assuntos
Antimetabólitos Antineoplásicos/uso terapêutico , Azacitidina/uso terapêutico , Leucemia Mieloide Aguda/tratamento farmacológico , Síndromes Mielodisplásicas/tratamento farmacológico , Adulto , Idoso , Idoso de 80 Anos ou mais , Contagem de Células , Gerenciamento Clínico , Esquema de Medicação , Feminino , Humanos , Leucemia Mieloide Aguda/diagnóstico , Leucemia Mieloide Aguda/mortalidade , Leucemia Mieloide Aguda/patologia , Masculino , Pessoa de Meia-Idade , Análise Multivariada , Síndromes Mielodisplásicas/diagnóstico , Síndromes Mielodisplásicas/mortalidade , Síndromes Mielodisplásicas/patologia , Estudos Retrospectivos , Análise de Sobrevida , Resultado do Tratamento
18.
Int J Gynecol Pathol ; 35(6): 593-597, 2016 Nov.
Artigo em Inglês | MEDLINE | ID: mdl-27167673

RESUMO

A very rare case of primary diffuse large B-cell lymphoma of the uterine cervix characterized by "spindle cell variant" morphology ("sarcomatoid subtype") is described along with a discussion of the challenging diagnosis due to its rarity and presenting clinical and pathological features.


Assuntos
Linfoma Difuso de Grandes Células B/diagnóstico , Linfoma Difuso de Grandes Células B/patologia , Sarcoma/diagnóstico , Neoplasias do Colo do Útero/diagnóstico , Neoplasias do Colo do Útero/patologia , Adulto , Biomarcadores Tumorais/análise , Biópsia , Erros de Diagnóstico , Feminino , Humanos
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