Philadelphia chromosome-negative myeloproliferative neoplasms in younger adults: A critical discussion of unmet medical needs, with a focus on pregnancy

Blood Reviews ◽  
2021 ◽  
pp. 100903
Author(s):  
Emanuela Sant'Antonio ◽  
Oscar Borsani ◽  
Chiara Camerini ◽  
Cirino Botta ◽  
Marco Santoro ◽  
...  
Healthcare ◽  
2021 ◽  
Vol 9 (3) ◽  
pp. 334
Author(s):  
Eunmi Lee ◽  
Sunkyung Cha ◽  
Geun Myun Kim

We investigated the effect of predisposing, enabling, need factors, and health behaviors on health-related quality of life (HRQoL) of patients with multimorbidity according to Andersen’s model. This study is a secondary analysis of population-based cross-sectional surveys. Data from 328 patients with multimorbidity (≥3 co-occurring chronic diseases) from the 6th/7th Korea National Health and Nutrition Examination Surveys were analyzed using logistic regression. Patients ≥65 years, without private insurance, with poor subjective health, unmet medical needs, and/or limited activity were more likely to experience mobility problems. Self-care problems were more likely among those without private insurance and/or with limited activity. Patients lacking living security, with poor subjective health, limited activity, and/or who smoked were more likely to experience problems performing usual activities. Pain/discomfort was more likely among females, Medicaid beneficiaries, and patients with limited activity and/or with poor subjective health. Patients with poor subjective health, limited activity, and/or unmet medical needs were more likely to experience anxiety/depression. The investigation of HRQoL in multimorbidity should consider predisposing, enabling, need factors, and health behaviors. Interventions addressing movement restrictions and personalized care based on HRQoL domains should be prioritized.


Vaccines ◽  
2021 ◽  
Vol 9 (2) ◽  
pp. 151
Author(s):  
Alexie Mayor ◽  
Adélaïde Chesnay ◽  
Guillaume Desoubeaux ◽  
David Ternant ◽  
Nathalie Heuzé-Vourc’h ◽  
...  

Respiratorytract infections (RTIs) are frequent and life-threatening diseases, accounting for several millions of deaths worldwide. RTIs implicate microorganisms, including viruses (influenza virus, coronavirus, respiratory syncytial virus (RSV)), bacteria (Pseudomonas aeruginosa, Streptococcus pneumoniae, Staphylococcus aureus and Bacillus anthracis) and fungi (Pneumocystis spp., Aspergillus spp. and very occasionally Candida spp.). The emergence of new pathogens, like the coronavirus SARS-CoV-2, and the substantial increase in drug resistance have highlighted the critical necessity to develop novel anti-infective molecules. In this context, antibodies (Abs) are becoming increasingly important in respiratory medicine and may fulfill the unmet medical needs of RTIs. However, development of Abs for treating infectious diseases is less advanced than for cancer and inflammatory diseases. Currently, only three Abs have been marketed for RTIs, namely, against pulmonary anthrax and RSV infection, while several clinical and preclinical studies are in progress. This article gives an overview of the advances in the use of Abs for the treatment of RTIs, based on the analysis of clinical studies in this field. It describes the Ab structure, function and pharmacokinetics, and discusses the opportunities offered by the various Ab formats, Ab engineering and co-treatment strategies. Including the most recent literature, it finally highlights the strengths, weaknesses and likely future trends of a novel anti-RTI Ab armamentarium.


Leukemia ◽  
2009 ◽  
Vol 23 (9) ◽  
pp. 1577-1586 ◽  
Author(s):  
J Wang ◽  
T Ishii ◽  
W Zhang ◽  
S Sozer ◽  
Y Dai ◽  
...  

2016 ◽  
Vol 42 (2-3) ◽  
pp. 429-450 ◽  
Author(s):  
Thomas J. Hwang ◽  
Aaron S. Kesselheim

Accelerating the development and approval of novel therapeutics has emerged as a key public health priority given the mortality, morbidity, and economic costs associated with infections caused by drug-resistant bacteria. However, there is limited empirical evidence to guide policymaking, such as the factors that may disadvantage antibiotics compared to other classes of drugs. In this Article, we empirically examine characteristics of the key clinical trials underpinning FDA's approval of antibiotics and other drugs over the past decade. Despite perceptions that antibiotic trials are larger and more difficult to conduct, we find that antibiotic trials are no larger than those conducted for drugs approved in other disease areas with high unmet medical needs, suggesting that policymakers may need to target other levers to meaningfully stimulate innovation. We discuss the risks and benefits of harnessing new and existing regulatory pathways to speed the approval of new drugs, particularly those intended to treat patients with serious and life-threatening infections, and we evaluate ways that proposals for new regulatory pathways could be improved to better prioritize and expedite the approval of therapies with the greatest potential for patient health benefits.


Haematologica ◽  
2015 ◽  
Vol 100 (10) ◽  
pp. e402-e405 ◽  
Author(s):  
J. Dahlstrom ◽  
X. Zhang ◽  
M. Ghaderi ◽  
M. Hultcrantz ◽  
M. Bjorkholm ◽  
...  

2014 ◽  
Vol 60 (2) ◽  
pp. 44-48
Author(s):  
Annamária Szántó ◽  
Zsuzsanna Pap ◽  
Z Pávai ◽  
I Benedek ◽  
Judit Beáta Köpeczi ◽  
...  

Abstract Background: The elucidation of the genetic background of the myeloproliferative neoplasms completely changed the management of these disorders: the presence of the Philadelphia chromosome and/or the BCR-ABL oncogene is pathognomonic for chronic myeloid leukemia and identification of JAK2 gene mutations are useful in polycytemia vera (PV), essential thrombocytemia (ET) and myelofibrosis (PMF). The aim of this study was to investigate the role of molecular biology tests in the management of myeloproliferative neoplasms. Materials and methods: We tested the blood samples of 117 patients between April 2008 and February 2013 at the Molecular Biology of UMF Târgu Mureș using RQ-PCR (for M-BCR-ABL oncogene) and/or allele-specific PCR (for JAK2V617F mutation). Results: Thirty-two patients presented the M-BCR-ABL oncogene, 16 of them were regularly tested as a follow-up of the administered therapy: the majority of chronic phase patients presented decreasing or stable values, while in case of accelerated phase and blast phase the M-BCR-ABL values increased or remained at the same level. Twenty patients were identified with the JAK2V617F mutation: 8 patients with PV, 4 with ET, 3 with PMF, 4 with unclassifiable chronic myeloproliferative disease and 1 patient with chronic myelomonocytic leukemia. There was no case of concomitant occurance of both molecular markers. Conclusions: Molecular biology testing plays an important role in the management of myeloproliferative neoplasms: identification of the molecular markers confirms the final diagnosis, excluding secondary causes of abnormal blood count parameters. Regular monitoring of MBCR- ABL expression level is useful in the follow-up of therapeutic efficiency.


2007 ◽  
Vol 39 (5) ◽  
pp. 769-778 ◽  
Author(s):  
NURUL ALAM

SummaryWhile a country’s health policy aims to provide health services to all who need them, very little in known about unmet need for additional medical care from users’ perspectives in Bangladesh. This study examined unmet medical need (defined as whether a mother felt that, to manage sickness, her child had required medical care that was not available, regardless of reasons and medical care sought) of 2123 under-15 sick children by illness and child’s socioeconomic characteristics in rural Bangladesh. The 1996 Health and Socioeconomic Survey conducted in Matlab recorded children’s chronic (a disease or a condition lasting 3 months or more) and acute (a disease or a condition with a rapid onset and a short, severe course) morbidity, medical care sought to combat illness and unmet needs for additional medical services in mothers’ views to manage the illness. The survey also recorded household socioeconomic data. Logistic regression was used to examine the data. The results reveal that unmet needs for additional medical care were 5·4% for children with acute illnesses, and 30·2% for children with chronic illnesses. For chronic illnesses, seeking medical care to manage illness from any health provider outside the home reduced unmet medical needs. Economic inequalities existed for both acute and chronic illnesses: the odds ratio of unmet medical needs for sick children of the least poor households was 0·42 (95% CI: 0·28–0·64) times that for sick children of the very poor households. The critically high unmet needs for children’s chronic morbidity reveal that the chronic disease control programme in Bangladesh needs urgent revisiting and strengthening.


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