Session 5 - Primary Prophylaxis

Author(s):  
Juan Carlos Garcia-Pagan ◽  
Norman D. Grace
Keyword(s):  

2009 ◽  
Vol 101 (04) ◽  
pp. 674-681 ◽  
Author(s):  
Massimo Franchini ◽  
Annarita Tagliaferri ◽  
Antonio Coppola

SummaryA four-decade clinical experience and recent evidence from randomised controlled studies definitively recognised primary prophylaxis, i.e. the regular infusion of factor concentrates started after the first haemarthrosis and/or before the age of two years, as the first-choice treatment in children with severe haemophilia. The available data clearly show that preventing bleeding since an early age enables to avoid or reduce the clinical impact of muscle-skeletal impairment from haemophilic arthropathy and the related consequences in psycho-social development and quality of life of these patients. In this respect, the aim of secondary prophylaxis, defined as regular long-term treatment started after the age of two years or after two or more joint bleeds, is to avoid (or delay) the progression of arthropathy. The clinical benefits of secondary prophylaxis have been less extensively studied, especially in adolescents and adults; also in the latter better outcomes and quality of life for earlier treatment have been reported. This review summarises evidence from literature and current clinical strategies for prophylactic treatment in patients with severe haemophilia, also focusing on challenges and open issues (optimal regimen and implementation, duration of treatment, long-term adherence and outcomes, cost-benefit ratios) in this setting.



Author(s):  
Claudio Cerchione ◽  
Davide Nappi ◽  
Giovanni Martinelli

AbstractMultiple myeloma (MM) survival rates have been substantially increased thanks to novel agents that have improved survival outcomes and shown better tolerability than treatments of earlier years. These new agents include immunomodulating imide drugs (IMiD) thalidomide and lenalidomide, the proteasome inhibitor bortezomib (PI), recently followed by new generation IMID pomalidomide, monoclonal antibodies daratumumab and elotuzumab, and next generation PI carfilzomib and ixazomib. However, even in this more promising scenario, febrile neutropenia remains a severe side effect of antineoplastic therapies and can lead to a delay and/or dose reduction in subsequent cycles. Supportive care has thus become key in helping patients to obtain the maximum benefit from novel agents. Filgrastim is a human recombinant subcutaneous preparation of G-CSF, largely adopted in hematological supportive care as “on demand” (or secondary) prophylaxis to recovery from neutropenia and its infectious consequences during anti-myeloma treatment. On the contrary, pegfilgrastim is a pegylated long-acting recombinant form of granulocyte colony-stimulating factor (G-CSF) that, given its extended half-life, can be particularly useful when adopted as “primary prophylaxis,” therefore before the onset of neutropenia, along chemotherapy treatment in multiple myeloma patients. There is no direct comparison between the two G-CSF delivery modalities. In this review, we compare data on the two administrations’ modality, highlighting the efficacy of the secondary prophylaxis over multiple myeloma treatment. Advantage of pegfilgrastim could be as follows: the fixed administration rather than multiple injections, reduction in neutropenia and febrile neutropenia rates, and, finally, a cost-effectiveness advantage.





Vaccines ◽  
2021 ◽  
Vol 9 (4) ◽  
pp. 372
Author(s):  
Léna Royston ◽  
Eva Royston ◽  
Stavroula Masouridi-Levrat ◽  
Nathalie Vernaz ◽  
Yves Chalandon ◽  
...  

Background: Real-life data on the administration of letermovir as cytomegalovirus (CMV) primary prophylaxis after allogeneic hematopoietic cell transplantation (HCT) remain limited. Methods: We conducted a retrospective single-center matched cohort study, comparing consecutive high-risk allogeneic HCT recipients (cases) receiving primary prophylaxis with letermovir and untreated matched historical controls, during a study period of 180 days. The primary outcome was the incidence of clinically significant (cs) CMV infection. Secondary outcomes included duration and costs of CMV-antiviral treatments, hospital resource utilization, hematology and laboratory parameters. Results: Letermovir prophylaxis decreased csCMV infection incidence from 82.7% (controls) to 34.5% (cases; p-value < 0.0001). Controls were more likely to have >1 episode of csCMV infection (59.6%) compared to cases (11.5%; p-value < 0.0001). Letermovir was associated with: shorter overall CMV-associated treatment duration (49 days vs. 77.8 days; p-value: 0.02) and a trend for lower costs of CMV-associated treatments ($4096 vs. $9736; p-value: 0.07) and reduced length of stay (44.8 days vs. 59.8 days; p-value: 0.16). Letermovir administration was associated with significantly shorter duration (27.3 days vs. 57.1 days; p-value: 0.008) and lower costs ($1089 vs. $2281; p-value: 0.008) of valganciclovir treatment. Compared to controls, higher platelet counts were observed in cases (138 G/L vs. 92 G/L; p-value: 0.03) and renal function was improved (94 mL/min/1.73 m2 vs. 74 mL/min/1.73 m2; p-value: 0.006). Conclusions: Primary anti-CMV letermovir prophylaxis decreased the incidence of csCMV infection and the administration of CMV-associated treatments and costs, particularly those associated with valganciclovir. An effect of letermovir on platelet reconstitution and renal function of csCMV post-HCT was observed and needs further investigation.





2010 ◽  
Vol 63 (3-4) ◽  
pp. 227-230
Author(s):  
Slobodanka Petrovic ◽  
Jelena Tomic ◽  
Radmila Ljustina-Pribic

Introduction The natural course of asthma is unpredictable and appears to be unaffected by any therapeutic strategy. Under such circumstances, the attention must be focused on the opportunities for prevention of a disease which is chronic, life long and incurable, even thought it can be very effectively controlled. During the past decades, a lot of a studies have been performed and started, in which relatively large numbers of children were included and followed prospectively to determine the incidence of risk factors for asthma in childhood. All these studies have contributed significant new information. The levels of prevention must be considered in all patients. There are two main separate components to the strategy. Primary prophylaxis Primary prophylaxis (time course of allergic sensitization, timing of exposure to allergens, influence of tobacco smoke, maternal health and allergen exposure) is introduced before there is any evidence of sensitization to factors which might have caused the disease. There is increasing evidence that allergic sensitization is a very common precursor to the development of asthma. Secondary prophylaxis Secondary prophylaxis (allergen avoidance, hygiene hypothesis) is important after primary sensitization to allergen has occurred, but before there is any evidence of asthma. Conclusion In this article the authors reviewed all results of studies about primary and secondary prophylaxis of asthma and its influence on the course of disease.



Hepatology ◽  
2003 ◽  
Vol 38 ◽  
pp. 296-296 ◽  
Author(s):  
C ARACIL ◽  
J LOPEZBALAGUER ◽  
D MONFORT ◽  
M PIQUERAS ◽  
B GONZALEZ ◽  
...  


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