Therapeutic guidelines for the treatment of cystic fibrosis

Cystic fibrosis (CF) is a complex, systemic autosomal recessive disease that affects the functions of the respiratory system, the digestive tract and all exocrine glands. The frequency for Europe averages 1: 2500 to 1: 3500 live births. The total number of patients with cystic fibrosis in Bulgaria is about 180. About 10% of the patients are diagnosed at birth. About 60–70% of patients are diagnosed before they reach one year of age. Respiratory symptoms predominate in the clinical picture in patients with cystic fibrosis and determine the prognosis in more than 90% of the patients. The treatment of patients with cystic fibrosis is strictly individualized, pharmacological and non-pharmacological and requires a comprehensive therapeutic approach. The complex therapy also includes bronchodilators, NSAIDs, corticosteroids, respiratory rehabilitation in combination with general body massage. Continued courses of broad-spectrum antibiotics are required to suppress chronic infection. With the progression of the disease, complications such as atelectasis, pneumothorax and pulmonary hemorrhages are observed. The establishment of specialized centers with trained and experienced professionals is essential in order to provide optimal patient care. These include frequent clinical evaluations, follow-up of complications, and early interventions for the treatment of patients with cystic fibrosis. The aim of the article is to familiarize the audience with the therapeutic measures applied in the treatment of patients with cystic fibrosis.

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357 Factors associated with changes in health-related quality of life in children with cystic fibrosis during one-year follow-up

Journal of Cystic Fibrosis ◽

10.1016/s1569-1993(17)30694-x ◽

2017 ◽

Vol 16 ◽

pp. S154

Author(s):

M. Van Horck ◽

B. Winkens ◽

G. Wesseling ◽

K. de Winter-de Groot ◽

I. De Vreede ◽

...

Keyword(s):

Quality Of Life ◽

Cystic Fibrosis ◽

Health Related Quality ◽

Factors Associated ◽

Related Quality ◽

Health Related ◽

One Year

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Improved Biochemical Parameters in Patients Who Undergo Early Resection in Isolated Ileocaecal Crohn’s Disease

Surgery Research and Practice ◽

10.1155/2017/4396573 ◽

2017 ◽

Vol 2017 ◽

pp. 1-5

Author(s):

Mirzafaraz Saeed ◽

Hari Hullur ◽

Amro Salem ◽

Abbas Ali ◽

Yousif Sahib ◽

...

Keyword(s):

Crohn’S Disease ◽

Crohn's Disease ◽

Surgical Resection ◽

Complete Blood Count ◽

Long Term Results ◽

Number Of Patients ◽

Long Term Follow Up ◽

One Year

Introduction. The aim of this study is to evaluate the outcome of introduction of early surgery in the course of isolated ileocecal Crohn’s disease, where there is no absolute indication of surgery. Methods. Observational study involving patients with isolated ileocecal Crohn’s disease who underwent early surgical resection (within one year of the presentation of the hospital). A complete blood count, ESR, and CRP were done and compared between the preoperative value, 1st postoperative visit (3-4 weeks), and last follow-up visit. Statistical analysis was done using Analysis of Variance (ANOVA) to compare the different figures. Results. There was a statistically significant increase in the hemoglobin levels between preoperative, postoperative, and long-term follow-up and a significant decrease in leukocyte count between the pre- and postoperative values (F=19.8, p<0.001 and F=8.9, p=0.002, resp.). Similarly, the ESR and CRP values were decreased significantly at long-term follow-up (F=8.5, p=0.019 and F=8.3, p=0.013, resp.). Conclusion. Early surgical resection in isolated ileocaecal Crohn’s disease achieved significant biochemical improvements. These successful results in this small number of patients indicate that early surgical intervention may provide better outcomes. These initial results encourage larger and comparative studies of long-term results versus long-term use of biological agents.

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A study on symptom improvement and efϑicacy of formoterol plus budesonide in the asthma management in northern parts of Tamil Nadu

International Journal of Research in Pharmaceutical Sciences ◽

10.26452/ijrps.v12i1.4177 ◽

2021 ◽

Vol 12 (1) ◽

pp. 742-748

Author(s):

Mani Dhandayuthapani ◽

Murugesh Shivashankar ◽

Uma K

Keyword(s):

Tamil Nadu ◽

Asthma Management ◽

Symptom Improvement ◽

Asthmatic Patients ◽

Severe Asthmatic ◽

Number Of Patients ◽

One Year ◽

Experienced Symptom

The purpose is to study the asthma symptom improvement and efficacy of formoterol (LABA) and Budesonide (ICS) combination in the asthma management in northern districts of Tamilnadu. It is a multicentric, non-comparative, questionnaire and random sampling study was conducted in 145 mild to severe asthmatic patients. They were in Formoterol plus budesonide combination inhalation drugs available in DPI and pMDI. Among the 145 asthmatic patients, 45 patients, 6 patients and 94 patients were in DPI, nebuliser and pMDI devices respectively. During drug initiation, The Asthmatics were in mild (27%), Moderate (57%) and Severe (16%) stages. After one year of follow-up, the number of patients in the mild is 84%, moderate 14% and severe 2%. Among 45 DPI Asthmatic patients, 29 patients, 15 patients and 1 patient have reported the handling the devices as easy, medium and hard respectively. On the other hand, 94 pMDI asthmatic patients, 38 patients, 48 patients and 8 patients have reported the handling of devices as easy, medium and hard respectively. The treatment resulted in 77 patients as good, 65 as satisfactory and 3 as same. After one year, all the 145 asthmatic patients adhered with the treatment and experienced symptom improvement with 53% patients as good, 45% patients as satisfactory and 2% patients as same. The treatment of formoterol and budesonide combination in the northern districts of Tamilnadu have effectively controlled the asthmatic symptoms and improved the quality of life in asthmatics. Moreover, the patient's adherence to the treatment is good in the northern parts of Tamilnadu.

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Characterisation of Victims Of Violence in the A & E Department and Analysis of the Acceptance of a Medico-Legal Expertise Centre After its Implementation vs. Multi-Year Consolidation

Zeitschrift für Orthopädie und Unfallchirurgie ◽

10.1055/a-0755-2296 ◽

2018 ◽

Vol 157 (04) ◽

pp. 426-433 ◽

Cited By ~ 1

Author(s):

David Latz ◽

Anja Bergermann ◽

Jeannie Jungnitsch ◽

Jan Peter Grassmann ◽

Erik Schiffner ◽

...

Keyword(s):

Trauma Surgery ◽

Social Impairment ◽

Number Of Patients ◽

Medical Reports ◽

Close Cooperation ◽

Victims Of Violence ◽

One Year ◽

Trauma Surgeons ◽

Attending Physician

Abstract Background People who have become victims of domestic or public violence often suffer long-term physical, psychological and social impairment. Due to physical injury, the first contact with the health care system is frequently an A & E Department. Thus, physicians and especially surgeons play a key role in detecting victims of domestic or public violence. The specific needs of victims are adequate medical treatment of injuries, forensic documentation, as well as interdisciplinary medical support to prevent further morbidity and violence. To take this into account, so-called expertise centres for victims of violence have been established at several locations in Germany in recent years. In this study: I. We tried to define the characteristics of victims of domestic and public violence to ensure better identification by physicians/surgeons. II. We elucidate the acceptance and effectiveness of such an expertise centre one year after its implementation and for a period of three years (2007 – 2009) and for a follow-up period of three years (2014 – 2016) after establishment. Material and Methods Patients were prospectively classified as victims of violence by the attending physician at the A & E Department and further treatment was initiated by the expertise centre for victims of violence. Medical reports from the A & E Department were analysed anonymously and compared with the number of patients of the expertise centre for victims of violence who had been referred from A & E Department. Results Orthopaedic and trauma surgery is the main referring discipline for the expertise centre for victims of violence. 0.9% of patients (2007 – 2009) and in the follow-up period (2014 – 2016) even 1.6% of patients were identified as victims of violence. However, the acceptance of such a centre fell from 22.2% (2007 – 2009) to 17.2% (2014 – 2016). Conclusion Physicians and especially trauma surgeons are responsible for identifying victims of domestic or public violence and ensuring further treatment. Accordingly, it is crucial that the expertise centre should characterise the victims of violence and be aware of their different needs, if the expertise centre is to be accepted. The results of this study indicate that interdisciplinary training and close cooperation between traumatology and legal medicine are the main prerequisites for continuous improvement in the treatment of victims of violence.

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Robot-Asssisted Laparoscopic Radical Prostatectomy (Ralp). Oncological and Functional Findings after 90 Cases

Urologia Journal ◽

10.1177/039156030907600207 ◽

2009 ◽

Vol 76 (2) ◽

pp. 87-89 ◽

Cited By ~ 1

Author(s):

C. Giberti ◽

M. Schenone ◽

P. Cortese ◽

F. Gallo ◽

E Gastaldi ◽

...

Keyword(s):

Disease Free Survival ◽

Functional Results ◽

Modern Technology ◽

Nerve Sparing ◽

Number Of Patients ◽

Before And After ◽

Disease Free Survival Rate ◽

One Year ◽

The One

The RALP is the most modern technology available for the treatment of intracapsular prostate cancer (CaP), which can produce a shorter learning curve and better results than the traditional techniques. Methods Between March 2005 and March 2008, 90 patients (64.3 ys, range 52–71) with intracapsular CaP underwent RALP at our institute. Before surgery the patients underwent routine examinations and filled in IIEF, IPSS and EORTC-QLQC30/PR 25 questionnaires. Patients were followed up with PSA assay, physical examination and compilation of the questionnaires. Median follow-up was 12.5 months (range 1–35 months). Results Mean operative time was 230 min. Discharge and catheter removal were at day 7.4 and 8.2, respectively, after surgery. Pathological staging reported pT2 and pT3 in 57 (63%) and 33 patients (37%), respectively. Positive surgical margins were assessed in 30 patients (33%), particularly 8.7% in pT2 tumors. The one-year biochemical disease-free survival rate was 90%. Regarding the functional results, 81 patients (90%) were perfectly continent while a mild and a moderate incontinence were reported in 7 (8%) and 2 (2%) patients, respectively. Mean IPSS score decreased from 8 to 4; among the patients who underwent bilateral nerve sparing RALP and no adjuvant therapy, 31 (70.4%) reported satisfactory sexual intercourses. Concerning postoperative quality of life, mean EORTC-QLQC30/PR 25 questionnaires scores were very similar before and after RALP. Conclusions After 90 cases of RALP the oncological and functional results are definitely promising. However, a wider number of patients and a greater follow-up are needed to confirm these data particularly as regards the functional results.

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Well-differentiated Thyroid Carcinoma of Childhood and Adolescence- A Long Term Experience at a Single Institute

Bangladesh Journal of Nuclear Medicine ◽

10.3329/bjnm.v20i2.37345 ◽

2018 ◽

Vol 20 (2) ◽

pp. 99

Author(s):

Faria Nasreen ◽

Nurun Nahar ◽

Sadia Sultana

Keyword(s):

Thyroid Carcinoma ◽

Papillary Carcinoma ◽

Differentiated Thyroid Carcinoma ◽

Whole Body ◽

Childhood And Adolescence ◽

Children And Adolescent ◽

Number Of Patients ◽

Well Differentiated ◽

One Year

Background: Thyroid carcinoma is rare in childhood and adolescence. The presentation is more advanced in case of children as compared to adults. However, the prognosis for survival in children is reportedly excellent. The aim of the study was to see the outcome of patients with well differentiated thyroid carcinoma during childhood and adolescence treated at a single institute.Patients and methods: A retrospective study of 61 children and adolescent patients (age ≤18years) with well differentiated thyroid carcinoma (DTC) enrolled in the National Institute of Nuclear Medicine and Allied Sciences, Dhaka during the period of January1986 up to December 2007 was carried out. To allow for a theoretical follow up of at least 5 years the last inclusion year was 2007 and follow up was carried on up to June 2013. All patients were treated by thyroidectomy followed by radioiodine ablation therapy. Whole body scans, Tg, anti Tg Ab and neck ultrasound findings were recorded six months to one year after initial therapy to classify patients into remission, persistent or recurrent disease. Status on last follow up was noted to estimate the survival rate.Results: A total of 40 patients had papillary carcinoma, 18 had follicular variant of papillary carcinoma (FVPCT) and three had follicular carcinoma. Age range at diagnosis was nine to 18 years with a mean of 15± 2 years. The number of patients ≤10 years were five and > 10 years were 56. There were 12 males and 49 females giving a M: F ratio of about 1:4. Among the 61 patients 30 patients had lymph node metastases and two had both nodal and lung metastases at initial presentation. After one year follow up from the initial radioiodine therapy, 30 patients were in remission and 31 patients had persistent disease. Eventually recurrence occurred in five patients. Three patients died during the whole observation period and all of them were cancer related giving a cancer specific mortality ratio of 0.049%, 95% CI 0 to 0.105%. Cancer specific survival by Kaplan-Meier curve was 98.2%, 98.2% and 66% at 5, 10 and 15 years respectively.Conclusion: DTC in children and adolescent has a good prognosis in the presence of neck and distant metastases. Awareness of the patient and regular follow up with life long surveillance is essential to obtain a favorable outcome.Bangladesh J. Nuclear Med. 20(2): 99-104, July 2017

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ASTHMA IN CYSTIC FIBROSIS

Romanian Journal of Pediatrics ◽

10.37897/rjp.2015.2.2 ◽

2015 ◽

Vol 64 (2) ◽

pp. 118-121

Author(s):

Marcela Daniela Ionescu ◽

◽

Ioana-Alina Anca ◽

Mihaela Balgradean ◽

◽

...

Keyword(s):

Cystic Fibrosis ◽

Lung Disease ◽

Airway Obstruction ◽

Autosomal Recessive ◽

Pulmonary Infection ◽

Autosomal Recessive Disease ◽

Personal History ◽

Common Symptom ◽

Infection And Inflammation ◽

History Of

Cystic fibrosis (CF) is the most common autosomal recessive disease in Caucasians. Lung disease is characterized by impaired mucocilliary clearance with airway obstruction and chronic pulmonary infection and inflammation. Wheeze is a common symptom in CF, but in some cases the wheeze is due to the presence of concomitant asthma. There is no consensus on how to define CF asthma, but the diagnosis is predominantly based on the patient’s strong family and personal history of atopy.

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CONGENITAL AFIBRINOGENAEMIA: DIAGNOSIS, CLINICAL FEATURES, FOLLOW-UP STUDY

10.1055/s-0038-1644856 ◽

1987 ◽

Author(s):

A M JakloyazAy ◽

Oa H dnagy

Keyword(s):

Autosomal Recessive ◽

Autosomal Recessive Disease ◽

Renal Calculi ◽

Bleeding Tendency ◽

Fresh Blood ◽

Severe Injuries ◽

Surgical Interventions ◽

Follow Up Study ◽

Rare Autosomal Recessive Disease

Authors followed 6 cases of congenital afibrinogenae- mia (CA) by offsprings of two apparently unrelated families from the same village. The sex ratio was4.m/3.f. CA is a rare autosomal recessive disease. Controlling 76 family members authors detected 11 cases of moderate and 2 cases of severe hypofibrinogeneemia.Among them-without any bleeding tendency-the mother of one case and both parents of two siblings with CA*The lack of fibrinogen was confirmed biochemically and immunologically too. The only symptom ofthe Illness are the severe posttraumatic bleeding. They appear as epistax- is, bleeding of the gums, or anyother bleeding aiter minor or severe injuries*Intraarticular bleeding, as in haemophilia rarely occurs inCA. One of our patients had profuse haematurias, causedby renal calculi. The only therapy is the substitution with transfusions of fresh blood, plasma, or fibrinogen concentrates*The rise of posttransfusional lllnisses grows with the number of transfusions*Stomatological or surgical interventions could be performed only after correction of the dotting abnormalitySo, one of our patients was submitted to splenectomyfor spontaneous rupture at 12 years and to nephrectomy for severe pyelo-caliceal cal- culosis with 19.He recovered fully after both interventions but died at 21 years after a bicycle accidenti The five other patients deceased at the age of 5«resp. 10 months and at 6-lo-resp 12 years. In 3 cases there was a subdural hammorrhage, once an intracranial blee- dingCnon autopsiated)and once a severe intraabdoml- nal haemorrhage after an accidental traumatism of the abdominal wall. The care of the CA cases is mostly a pediatric proble. The frequency of the pottraumatic bleeding decrease with the growth*The schoolchildren are paying more attention to avoid injuries

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Significance of Abnormal Liver Function Tests in Hemophiliacs: A Follow-Up Study

10.1055/s-0039-1682583 ◽

1977 ◽

Author(s):

P.M. Mannucci ◽

Z.M. Ruggeri ◽

A. Capitanio ◽

F. Pareti

Keyword(s):

Liver Function ◽

Clinical Evidence ◽

Liver Function Tests ◽

Abnormal Liver Function ◽

Function Tests ◽

Number Of Patients ◽

High Incidence ◽

Therapeutic Measures ◽

Observation Period

It was previously shown (1) that hemophiliacs have a high incidence of abnormal liver function tests unaccompanied by clinical evidence of illness. Since single measurements are of little use to access the significance of such abnormalities and their possible relationship with chronic hepatitis, hemophiliacs with abnormal liver function tests were regularly followed with measurements carried out at least once a year. 20 patients with raised SGOT-SGPT were followed for up to 3 years; in 9, the abnormalities persisted throughout the whole observation period, whereas in 2 the enzymes were occasionally increased and in 9 they were always normal since the initial abnormal measurement. Out of 39 patients with increased serum gamma-globulins, abnormal values were 1 persistently observed in 11 followed for 3 years. In 28 patients followed for 2 years, persistently abnormal values were found in 21, whereas in 7 normal values were invariably found since the initial abnormal measurement. These findings show that liver function tests are persistently abnormal in a number of patients; in these, biopsy should be considered in order to undertake suitable therapeutic measures.

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EVALUATION OF CFTR CHANNEL FUNCTION AND RESPONSES TO MODULATORS FOR CYSTIC FIBROSIS PATIENTS WITH GENETIC VARIANT F508DEL

BIOTECHNOLOGY: STATE OF THE ART AND PERSPECTIVES ◽

10.37747/2312-640x-2020-18-232-234 ◽

2020 ◽

pp. 232-234

Author(s):

A. Efremova ◽

T. Bukharova ◽

N. Petrova ◽

N.Kashirskaya Kashirskaya ◽

Y. Melyanovskaya ◽

...

Keyword(s):

Cystic Fibrosis ◽

Autosomal Recessive ◽

Genetic Variant ◽

Autosomal Recessive Disease ◽

Target Therapy ◽

Allelic Frequency ◽

Common Life ◽

Life Threatening ◽

Cftr Protein ◽

The Russian Federation

Cystic fibrosis (CF) is the most common life-threatening autosomal recessive disease. CF is caused by mutations in the CFTR gene that encodes an anionic channel, which is expressed in epithelial cells. The most common genetic variant is F508del, its allelic frequency in the Russian Federation 52.81% (2017). This variant belongs to class II, leads to incorrect folding of the CFTR protein and its premature degradation. Currently, the effectiveness of target therapy can be individually evaluated by forskolin-induced swelling (FIS) assay on intestinal organoids obtained from CF patients with different genotypes.

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