Allogeneic hematopoietic stem cell transplantation for children and adolescents with acute myeloid leukemia in Brazil : a multicentric retrospective study

The survival rates of children with high-risk acute myeloid leukemia (AML) treated with hematopoietic stem cell transplant (HSCT) range from 60% to 70% in high-income countries. The corresponding rate for Brazilian children with AML who undergo HSCT is unknown. We conducted a retrospective analysis of 114 children with AML who underwent HSCT between 2008 and 2012 at institutions participating in the Brazilian Pediatric Bone Marrow Transplant Working Group. At transplant, 38% of the children were in first complete remission (CR1), 37% were in CR2, and 25% were in CR3þ or had persistent disease. The donors included 49 matched-related, 59 matched-unrelated, and six haploidentical donors. The most frequent source of cells was bone marrow (69%), followed by the umbilical cord (19%) and peripheral blood (12%). The 4-year overall survival was 47% (95% confidence interval [CI] 30%–57%), and the 4-year progression-free survival was 40% (95% CI 30%–49%). Relapse occurred in 49 patients, at a median of 122 days after HSCT. There were 65 deaths: 40 related to AML, 19 to infection, and six to graft versus host disease. In conclusion, our study suggests that HSCT outcomes for children with AML in CR1 or CR2 are acceptable and that this should be considered in the overall treatment planning for children with AML in Brazil. Therapeutic standardization through the adoption of multicentric protocols and appropriate supportive care treatment will have a significant impact on the results of HSCT for AML in Brazil and possibly in other countries with limited resources

Related haematopoietic progenitor cell donor care: the influence of current guidance, and pathways to improvement

Historically, relatively little regulation has been in place to safeguard the health and well-being of related HPC donors (RDs), and retrospective studies have suggested an increased incidence of adverse events in comparison to unrelated donors. Although in recent years, FACT-JACIE Standards have introduced specific requirements aiming to address this gap, accreditation is not mandatory in many countries, and the influence of such changes has never been evaluated. This thesis provides insight into current procedures for managing RDs in transplant centres internationally, examines the impact of regulatory guidance to date, and explores potential pathways to improvement. Studies in this thesis provide a detailed analysis of RD care pathways in the USA and Europe, and I am able to clearly demonstrate the potential for regulation to drive change in this field. Improvements are shown over time in aspects of care that have been addressed by regulatory standards, and management of RDs in accredited centres is shown to be more consistent with accepted best practice than that in nonaccredited centres. These studies also reveal heterogeneity in donor care at each stage of the pathway with the result that RDs who would be deemed suitable by some transplant centres would be deferred by others. In an effort to align care standards for RDs, I assessed the feasibility of alternative care pathways and showed considerable logistical and financial difficulties for care models where the entire RD pathway is managed outside the transplant centre setting. However I was able to establish a model of RD follow-up by an unrelated donor registry, which was evaluated through a successful pilot study. After I demonstrated enthusiasm for clear guidelines and medical criteria in related donor care, I led the development of national RD care guidelines and including an online tool for comprehensive RD medical suitability criteria

Managed Care’s Crimea: Medical Necessity, Therapeutic Benefit, and the Goals of Administrative Process in Health Insurance

En brotyp som är vanlig bland brokonstruktioner är plattrambron. Denna brotyp byggs både med slak- och spännarmering. Det är dock vanligare att bygga med slakarmering än spännarmering, vilket övervägs särskilt då spännvidderna är ungefär 25- 30 meter eller mer. Inledningsvis har en noggrann litteraturstudie genomförts tillsammans med en intervju av en kunnig brokonstruktör för att säkerställa en interaktion mellan teori och aktuell praxis. Detta arbete har haft ett huvudmål, vilket är att ur ett struktur- och kostnadsperspektiv jämföra och utvärdera hur behovet av slakarmering varierar i en plattrambros farbana då spännvidder varieras och betongtvärsnitt ändras. Arbetet har utförts med hjälp av finita elementprogrammet ”Brigade Standard”, vilket erbjuder en tre-dimensionell strukturanalys. Fyra olika tvärsnitt för spännvidder mellan 10-20 meter analyseras. Dimensioner på brodelar bestäms med hjälp av tumregler samt generella råd från intervjuperson; höjd i fält enligt L/20, L/25, L/30 samt L/35, där L är spännvidden. Resultatet visar främst att för ökade spännvidder mot 20 meter ökar lasteffekten på grund av egentyngd drastiskt och då särskilt det tvärsnitt som är störst, det vill säga L/20. Vidare visas även att slankare tvärsnitt resulterar i aningen högre armeringsmängd. Dock påverkas betongkostnaden i större utsträckning av slankare tvärsnitt och särskilt större spännvidder. Även om större spännvidd och slankare tvärsnitt föranleder ett större armeringsbehov, fås en lägre totalkostnad. Det bör noteras att priset på betong och stål är starkt beroende av konjunktur och tillgång till material. Denna studie kan bli ännu intressantare då priset varierar kraftigt för stål och föranleder att betongpriset blir mindre dominerande än vad det är idag. Huvudprodukten från detta arbete är att med hjälp av tabeller och nya priser kunna beräkna en preliminär totalkostnad för plattrambrons farbana för olika spännvidder och hur olika tvärsnittsalternativ påverkar denna kostnad

Advancing knowledge for haemato-oncology nursing practice

Nurses working in haemato-oncology require specialised knowledge and yet, formal education programmes and textbooks devoted to the speciality are limited. In advancing knowledge for haemato-oncology nursing practice this project endeavours to develop, synthesise, organise and disseminate available evidence making it more accessible to practising nurses. The project consists of two inter-related parts: reflection on, and critique of, the development and editing of the second edition of a nursing textbook and a Delphi study of research priorities in haemato-oncology nursing included as a chapter of the textbook. In aiming to expand and advance the knowledge base for nurses working in haematooncology the concept of knowledge, ways of acquiring knowledge and types of nursing knowledge are explored and critically analysed. A textbook is mainly a source of empirical knowledge but ways of encouraging the development of other forms of relevant knowledge are discussed in relation to the format of the textbook. Critical reflection on the development of the textbook is also undertaken to detennine usefulness and value. Development of the first edition of the textbook and analysis of existing literature in haemato-oncology nursing highlighted the paucity of research informing the speciality. This provided the incentive to undertake the Delphi study. Round 1 generated a wide range of research topics, subsequently used to develop the questionnaire for round 2. Round 2 results demonstrated close clustering and little discrimination between research topics. Round 3 was therefore undertaken to increase the reliability and credibility of results and further discriminate between research priorities. Results from rounds 2 and 3 were remarkably consistent with several strong research themes emerging. These themes provide the foundations for the development of a research strategy with the potential to further advance knowledge for haemato-oncology nursing practice

A comparison of health technology adoption in four countries (Japan, Korea, the UK, and the US)

This research empirically examines and compares the adoption of health technologies through case studies. The health technologies under review are assisted reproductive technologies, cochlear implants, haematopoietic stem cell transplantations, caesarean section deliveries, Gamma knife units and kidney transplants in four countries: Japan, Korea, the UK and the US. The interactions between the micro factors of health technologies and the macro environment in the adoption of health technologies are examined on the basis of a literature review and analysis of data. The micro factors were evaluated in terms of economic, clinical and technical aspects. In assessing the macro factors, payment systems and regulations related to the selected health technologies were taken into account. To examine the micro factors, the results of health technology assessments in earlier studies were reviewed. In order to explore the macro factors, historical changes in the payment systems affecting the selected health technologies and legal regulations, including legislation, directives, guidelines and court orders related to the technologies, were investigated. The adoption level of health technologies was evaluated in time-series and cross-sectional terms, measuring the trend of technology adoption and comparing the experience of the four countries under review. This research suggests clustering health technologies into “welfare oriented technology” and “private benefit oriented technology” by considering the economic incentives of adopters, individual desires of consumers and public concern over the technology. Private benefit oriented technologies are those which adopters expect to increase income from the providers or which meet the personal desires of the consumers. For welfare oriented technology, the decision is dominated by the aims of public welfare. As the model predicted, the adoption of welfare oriented technologies was higher in the health systems under national planning, while that of private benefit oriented technologies was higher in the systems whose health provisions accept market conditions

The Tin Man Needs a Heart: A Proposed Framework for the Regulation of Bioprinted Organs

Each day, seventeen people die in the United States while waiting for an organ transplant. At least part of this need could be met by bioprinting, a technology that allows the on-demand production of custom-sized organs from a patient’s own cells. The field of bioprinting is progressing rapidly: the first bioprinted organs have already entered the clinic. Yet, developers of bioprinted organs face significant uncertainty as to how their potentially lifesaving products will be regulated—and by which government agency. Such regulatory uncertainty has the potential to decrease investment and stifle innovation in this promising technological field. This Note examines how the current framework for the regulation of medical products and human organs might be applied to bioprinted organs. This Note concludes that the existing regulatory schemes do not sufficiently address the specific regulatory needs created by bioprinted organs, which are uniquely interdisciplinary materials. Therefore, this Note proposes a new regulatory framework to reduce uncertainty for bioprinted organ developers and to promote patient access to these bioprinted materials that might soon serve as safe and effective replacements for donor organs