Quality of life of patients with severe chronic neutropenia receiving long-term treatment with granulocyte colony-stimulating factor

JAMA ◽  
1993 ◽  
Vol 270 (9) ◽  
pp. 1132-1133 ◽  
Author(s):  
E. A. Jones
Blood ◽  
2014 ◽  
Vol 124 (21) ◽  
pp. 1399-1399
Author(s):  
Cornelia Zeidler ◽  
Anna Nickel ◽  
Ulrike A.H. Grote ◽  
Sabine Mellor-Heineke ◽  
Karl Welte

Abstract Congenital neutropenias include a heterogeneous group of diseases characterized by a decrease in circulating neutrophils and different underlying germ-line gene mutations. Since 1988 recombinant human G-CSF is available for the treatment of severe chronic neutropenia patients. In phase I/II/III studies in patients with severe congenital and cyclic neutropenia, treatment with recombinant human granulocyte colony-stimulating factor (r-metHuG-CSF) resulted in a rise in the absolute neutrophil counts (ANC) and a reduction in infections. The Severe Chronic Neutropenia International Registry collects clinical information on patients suffering from severe chronic neutropenia since 1994. 312 of 379 CN and 65 of 79 CyN patients receive long-term G-CSF treatment for a median duration of 8,26 years in CN and 9,37 in CyN. Median G-CSF doses vary by neutropenia subtype and gene mutation. Patients with congenital neutropenia revealing ELANE mutations require the highest G-CSF doses compared to other subtypes (median G-CSF dose 5 µg/kg/day in 88 patients). SCNIR follow-up data suggest that pediatric ELANE-CN patients were maintained at a particular G-CSF dose per kg body weight for longer than expected by the gain of body weight. We therefore analysed the reported yearly G-CSF doses in all treated ELANE-CN patients to evaluate the dose trend: From 88 G-CSF treated patients with ELANE-CN we excluded 16 patients with nonresponse (ANC remained below 0.5 x 109 /L) and partial response (ANC remained between 0.5 and 0.99 x 109 /L). Since the gain of body weight is highest during the first 5 years of life with and 10-fold increase we divided G-CSF good responders by age at G-CSF initiation (0-5 years vs above 6 years) and compared G-CSF doses at the end of the dose finding period with the last dose reported. 51 of the remaining 72 patients started G-CSF treatment between the first and 5th year of life with a median G-CSF treatment duration of 9.76 years. 37 of the 51 patients were treated for at least 5 years. In 21 of the 72 patients G-CSF treatment was initiated after their 10thbirthday with a median follow up of 20.45 years. 19 of the 21 patients were treated for at least 5 years. All patients with a treatment duration of less than 5 years were excluded from further analysis. In ELANE-CN patients with treatment start at an age of 0-5 years the mean G-CSF dose decreased significantly from 13.47 µg/kg/day at the time of ANC-response to 7.96 µg/kg/day at the last report (median G-CSF dose decreased from 6.85 µg/kg/day to 4.42 µg/kg/day) during a treatment duration of at least 5 years. In summary, a significant decrease in the individual G-CSF doses could be observed in ELANE-CN patients who started G-CSF treatment during the first five years of their lives suggesting an age dependent alleviation of the severity of the disease as judged by the response to G-CSF. Disclosures No relevant conflicts of interest to declare.


Blood ◽  
2002 ◽  
Vol 100 (4) ◽  
pp. 1499-1501 ◽  
Author(s):  
Madeleine Carreau ◽  
Lili Liu ◽  
Olga I. Gan ◽  
Johann K. Hitzler ◽  
John E. Dick ◽  
...  

Transient treatment with cytokines appears to improve hematopoietic function in Fanconi anemia; however, the effectiveness or adverse effect of long-term treatment is not known. The mitomycin C–treatedFancc−/− mouse provides a valuable model to address long-term efficacy of such treatment.Fancc−/− mice injected with granulocyte colony-stimulating factor, erythropoietin, or both cytokines showed a delay in mitomycin C (MMC)–induced bone marrow (BM) failure compared to untreated mice. However, long-term cytokine exposure followed by MMC challenges did not protect mice from the reduction of peripheral blood counts or the number of early myeloid progenitors. These results suggest that cytokine treatment may be beneficial only in the short-term, while long-term treatment is not protective for BM aplasia.


2013 ◽  
Vol 6 (11) ◽  
pp. 681-687 ◽  
Author(s):  
Robert A Jones ◽  
Brian Quilty

Unlike many other forms of inflammatory arthritis, the crystal arthropathies are routinely diagnosed and managed in primary care. Gout, in particular, is relatively commonplace and rates of other types of crystal-related arthritis are predicted to increase. These are, therefore, conditions that GPs and trainees will regularly encounter during routine practice. While the clinical features and pathophysiology of gout and pseudo-gout are well described, the long-term treatment goals and options of management are often less well understood, and opportunities to assess for associated co-morbidities can easily be missed. GPs can be central in optimising management by promptly and appropriately addressing acute symptoms, preventing recurrent attacks, minimising disability and work absences, reducing cardiovascular risk factors, improving general health and enhancing quality of life.


2019 ◽  
Author(s):  
Gek Phin Chua ◽  
Quan Sing Ng ◽  
Hiang Khoon Tan ◽  
Whee Sze Ong

Abstract Background The aim of this study is to determine the main concerns of survivors at various stages of the cancer survivorship of the cancer survivorship trajectory and to assess whether these concerns have any effect on their quality of life (QOL). The overall goal was to use the insights from the study to guide practice on patient care. Methods A cross-sectional survey of 1107 cancer survivors diagnosed with colorectal, breast, lung, gynaecological, prostate or liver cancers from a cancer centre in Singapore. Eligible patients self-completed a questionnaire adapted from the Mayo Clinic Cancer Centre’s Cancer Survivors Survey of Needs. Results The top 5 concerns among all survivors were cancer treatment and recurrence risk (51%), followed by long-term treatment effects (49%), fear of recurrence (47%), financial concerns (37%) and fatigue (37%). Cancer treatment and recurrence risk, long-term treatment effects and fear of recurrence were amongst the top concerns across the survivorship trajectory. Mean QOL was 7.3 on a scale of 0 – 10. Completed treatment patients had higher QOL score than the newly diagnosed and on treatment patients and the patients dealing with recurrence or second cancer patients. Predictors for QOL included the economic status and housing type of patients and whether patients were concerned with pain and fatigue Conclusion This study confirms that cancer survivors in Singapore face multiple challenges and had various concerns at various stages of cancer survivorship, some of which negatively affect their QOL It is critical to design patient care delivery that appropriately address the various concerns of cancer survivors in order for them to cope and improve their QOL.


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