Psychometric evaluation of the NTDT-PRO questionnaire for assessing symptoms in patients with non-transfusion-dependent beta-thalassaemia.

OBJECTIVES: The non-transfusion-dependent beta-thalassaemia-patient-reported outcome (NTDT-PRO) questionnaire was developed for assessing anaemia-related tiredness/weakness (T/W) and shortness of breath (SoB) among patients with NTDT. Psychometric properties were evaluated using blinded data from the BEYOND trial (NCT03342404). DESIGN: Analysis of a phase 2, double-blind, randomised, placebo-controlled trial. SETTING: USA, Greece, Italy, Lebanon, Thailand and the UK. PARTICIPANTS: Adults (≥18 years) (N=145) with NTDT who had not received a red blood cell transfusion within 8 weeks prior to randomisation, with mean baseline haemoglobin level ≤100 g/L. MEASURES: NTDT-PRO daily scores from baseline until week 24, and scores at select time points for the 36-Item Short Form Health Survey version 2 (SF-36v2), Functional Assessment of Chronic Illness Therapy-Fatigue (FACIT-F) and Patient Global Impression of Severity (PGI-S). RESULTS: Cronbach's alpha at weeks 13-24 was 0.95 and 0.84 for the T/W and SoB domains, respectively, indicating acceptable internal consistency reliability. Among participants self-reporting no change in thalassaemia symptoms via the PGI-S between baseline and week 1, intraclass correlation coefficients were 0.94 and 0.92 for the T/W and SoB domains, respectively, indicating excellent test-retest reliability. In a known-groups validity analysis, least-squares mean T/W and SoB scores at weeks 13-24 were worse in participants with worse scores for the FACIT-F Fatigue Subscale (FS), SF-36v2 vitality or PGI-S. Indicating responsiveness, changes in T/W and SoB domain scores were moderately correlated with changes in haemoglobin levels, and strongly correlated with changes in SF-36v2 vitality, FACIT-F FS, select FACIT-F items and the PGI-S. Improvements in least-squares mean T/W and SoB scores were higher in participants with greater improvements in scores on other PROs measuring similar constructs. CONCLUSIONS: The NTDT-PRO demonstrated adequate psychometric properties to assess anaemia-related symptoms in adults with NTDT and can be used to evaluate treatment efficacy in clinical trials.

Transfusion-transmitted malaria: case report of asymptomatic donor harboring Plasmodium malariae.

Malaria in Brazil is endemic in the Amazon region, but autochthonous cases with low parasitaemia occur in the Atlantic Forest area of the country. According to Brazilian legislation no test is mandatory for blood donors from non-endemic areas. However if they have traveled to malaria transmission regions they are deferred for six months before they can donate. This report describes a transfusion-transmitted malaria case in Sao Paulo, Brazil, where one recipient received infected blood and developed the disease. He lived in Sao Paulo and had no previous transfusion or trips to endemic areas, including those of low endemicity, such as Atlantic Forest. Thick blood smears confirmed Plasmodium malariae. All donors lived in Sao Paulo and one of them (Donor 045-0) showed positive hemoscopy and PCR. This asymptomatic donor had traveled to Juquia, in the Atlantic Forest area of S ao Paulo State, where sporadic cases of autochthonous malaria are described. DNA assay revealed P. malariae in the donor's (Donor 045-0) blood. Serum archives of the recipient and of all blood donors were analyzed by ELISA using both P. vivax and P. falciparum antigens, and IFAT with P. malariae. Donor 045-0's serum was P. malariae IFAT positive and the P. vivax ELISA was reactive. In addition, two out of 44 donors' archive sera were also P. vivax ELISA reactive. All sera were P. falciparum ELISA negative. This case suggests the need of reviewing donor selection criteria and deferral strategies to prevent possible cases of transfusion-transmitted malaria.

Transfusion-transmitted malaria: case report of asymptomatic donor harboring Plasmodium malariae / Malária transfusional: relato de caso de doador assintomático infectado por Plasmodium malariae

Malaria in Brazil is endemic in the Amazon region, but autochthonous cases with low parasitaemia occur in the Atlantic Forest area of the country. According to Brazilian legislation no test is mandatory for blood donors from non-endemic areas. However if they have traveled to malaria transmission regions they are deferred for six months before they can donate. This report describes a transfusion-transmitted malaria case in Sao Paulo, Brazil, where one recipient received infected blood and developed the disease. He lived in Sao Paulo and had no previous transfusion or trips to endemic areas, including those of low endemicity, such as Atlantic Forest. Thick blood smears confirmed Plasmodiummalariae. All donors lived in Sao Paulo and one of them (Donor 045-0) showed positive hemoscopy and PCR. This asymptomatic donor had traveled to Juquia, in the Atlantic Forest area of S ao Paulo State, where sporadic cases of autochthonous malaria are described. DNA assay revealed P. malariae in the donor's (Donor 045-0) blood. Serum archives of the recipient and of all blood donors were analyzed by ELISA using both P. vivax and P. falciparum antigens, and IFAT with P. malariae. Donor 045-0's serum was P. malariae IFAT positive and the P. vivax ELISA was reactive. In addition, two out of 44 donors' archive sera were also P. vivax ELISA reactive. All sera were P. falciparum ELISA negative. This case suggests the need of reviewing donor selection criteria and deferral strategies to prevent possible cases of transfusion-transmitted malaria.

No Brasil a malária é endêmica na Amazônia, porém casos autóctones com baixas parasitemias ocorrem na área costeira de Mata Atlântica. De acordo com a legislação brasileira, não são obrigatórios testes para detecção de malária em doadores de sangue de áreas não-endêmicas; entretanto são excluídos por seis meses aqueles com relato de deslocamento para áreas de transmissão. Este trabalho descreve um caso de malária transfusional ocorrido em São Paulo, Brasil, em que um paciente recebeu sangue infectado, desenvolvendo a doença. Ele residia em São Paulo e não apresentava histórico de transfusão anterior ou deslocamentos para áreas endêmicas, incluindo as de baixa endemicidade, como a Mata Atlântica. A gota espessa revelou Plasmodium malariae. Os doadores eram residentes em São Paulo e um deles (045-0) apresentou hemoscopia e PCR positivos. Este era assintomático com PCR positiva para P. malariae e viagem para Juquiá, Mata Atlântica de São Paulo, onde são descritos casos esporádicos de malária autóctone. Amostras de soro do receptor e de todos os doadores foram ensaiadas por ELISA com antígenos de P. vivax e P. falciparum e RIFI com P. malariae. O doador 045-0 apresentou RIFI positiva para P. malariae. ELISA-P. vivax foi reagente no doador infectado (045-0) e em dois dos 44 doadores. Todos os soros foram negativos com antígeno de P. falciparum. Este caso aponta a necessidade de revisão dos critérios de triagem clínico-epidemiológica para evitar casos transfusionais e também adequar as estratégias de exclusão de doadores de sangue.

Quimioterapia em tumores de cabeça e pescoço / Chemotherapy for head and neck tumors

A cirurgia e a radioterapia säo modalidades terapêuticas eficazes no tratamento de carcinoma de cabeça e pescoço em estados precoces. Neste caso, pode se obter uma taxa de cura variando entre 50 a 100. Entretanto, em estados avançados o prognóstico é reservado e a quimioterapia surge como um alternativa que pode contribuir para o processo de cura da doença, mais especificamente quando combinada à radioterapia e/ou cirurgia(quimioterapia neoadjuvante), ou quando aplicada concomitantemente à radioterapia(quimioradioterapia). Nestes casos, deve-se avaliar qual das duas modalidades pode trazer melhor benefício, sem nos esquecermos da preservaçäo do órgäo. Em estágios avançados ou na doença recidivante, a quimioterapia é empregada somente como forma paliativa de tratemento, na tentativa de melhorar a disfagia e a dor ou de preservar o órgäo acometido. As drogas quimioterápicas que demonstram eficácia comprovada no tratamento dos tumores de cabeça e pescoço säo: Methotrexate, 5-fluorouracil, Cisplatina, Carboplatina e Bleomicina. Novas drogas estäo sendo estudadas, como a Vinorrelbina e os taxóides. A quimioterapia representa uma adiçäo importante aos algoritmos atuais de tratamento multidisciplinar dos tumores de cabeça e pescoço, seja pela possibilidade de cura ou pela melhora na qualidade de vida dos pacientes, já que pode potencialmente melhorar os resultados locais e até mesmo, em alguns casos, permitir a preservaçäo do órgäo acometido