Pharmacotherapy assignment presentation.pptx

Best ppt

1. PHARMACOTHERAPY ASSIGNMENT Cystic and Fibrosis

2. Assignment Outline Introduction

3. Introduction to Cystic Fibrosis What is Cystic Fibrosis (CF)? A genetic disorder affecting multiple organs, primarily the lungs, pancreas, and digestive system.
Caused by mutations in the CFTR gene. Prevalence : Affects approximately 30,000 people in the United States. Life Expectancy : Significantly improved in recent decades due to advances in treatment, with average lifespan now over 40 years.

4. Pathophysiology of CF CFTR Gene and Protein:**     * Encodes a protein responsible for transporting chloride ions across cell membranes, particularly in epithelial tissues. * **Consequences of Defective CFTR:**     * Thick, sticky mucus buildup in airways, pancreas, and intestines.     * Chronic lung infections.     * Pancreatic insufficiency.     * Malabsorption and malnutrition.     * Increased sweat chloride levels.

5. Symptoms and Diagnosis of CF** Common Symptoms:**
* Recurrent respiratory infections.
* Persistent cough with phlegm.
* Wheezing and shortness of breath.
* Poor weight gain and growth.
* Salty sweat. * **Diagnosis:**
* Newborn screening.
* Sweat chloride test.
* Genetic testing.

6. Treatment Goals for CF* Improve Lung Function:** Clear mucus, prevent and treat infections, and reduce inflammation.
* **Manage Pancreatic Insufficiency:** Replace digestive enzymes and ensure adequate nutrition.
* **Treat Other Symptoms:** Address constipation, manage pain, and address fertility issues.
* **Enhance Quality of Life:** Support physical, emotional, and social well-being.

7. Targeting the Defective CFTR Protein** CFTR Modulators:**  Aim to improve the function of the existing defective CFTR protein.     * ** Potentiators :**  Enhance the function of the remaining CFTR protein.         * Ivacaftor ( Kalydeco )         * Lumacaftor (VX-809)         * Tezacaftor (VX-770)         * Elexacaftor (VX-661)     * **Correctors:**  Help the CFTR protein fold correctly and reach the cell membrane.         * Lumacaftor (VX-809)         * Tezacaftor (VX-770)         * Elexacaftor (VX-661)

8. Combinations of CFTR Modulators** Triple Combinations:** Offer improved efficacy for patients with specific CFTR mutations.
* ** Trikafta ( Elexacaftor / Tezacaftor / Ivacaftor ):** Approved for patients with at least one F508del mutation.
* ** Symdeko ( Elexacaftor / Tezacaftor ):** Approved for patients with at least one F508del mutation.
* **Individualized Treatment Plans:** Based on specific CFTR mutations and patient needs

9. Managing Lung Disease * **Airway Clearance Techniques:**     * **Chest Physiotherapy:**  Manual techniques to mobilize mucus.     * **Inhaled Hypertonic Saline:**  Increases mucus hydration.     * **Nebulized Medications:**  Bronchodilators and mucolytics . * **Antibiotics:**  Treat and prevent infections.     * Tobramycin (TOBI)     * Azithromycin (Zithromax)     * Ciprofloxacin (Cipro) * **Anti-inflammatory Medications:**  Reduce inflammation.     * Budesonide ( Pulmicort )     * Fluticasone ( Flovent )

10. Treating Pancreatic Insufficiency** Pancreatic Enzyme Replacement Therapy (PERT):**  Replaces missing digestive enzymes.     * **Capsules or Tablets:**  Taken with every meal and snack.     * **Dosage adjusted based on individual needs.** * **Nutritional Support:**  High-calorie diets, supplemental vitamins, and nutritional counseling

11. Managing Other Symptoms** **Constipation:** Laxatives and stool softeners.
* **Pain Management:** Over-the-counter or prescription pain relievers.
* **Infertility:** Assisted reproductive techniques may be needed.
* **Psychological Impact:** Therapy and support groups

12. Future Directions in CF Treatment** Gene Therapy:**  Replace or correct the faulty CFTR gene. * **CRISPR-Cas9 Technology:**  Gene editing technology for precise gene correction. * **Novel CFTR Modulators:**  Development of new drugs targeting a wider range of mutations. * **Personalized Medicine:**  Tailored treatment based on specific CFTR mutations and individual needs.

13. Illustrative Images of New Treatments** * Images depicting:     * Gene therapy vectors.     * CRISPR-Cas9 system targeting CFTR gene.     * Novel CFTR modulators.     * Personalized medicine approach

14. Importance of Multidisciplinary Care** * **CF Care Team:**  Pulmonologists, gastroenterologists, CF specialists, dietitians, respiratory therapists, social workers, and nurses. * **Coordination of Care:**  Ensures comprehensive management of all aspects of CF.

15. Patient Education and Empowerment Understanding CF:** Knowledge is power.
* **Adherence to Treatment:** Compliance with medication regimens and therapies is crucial.
* **Self-Management Skills:** Airway clearance techniques, nutritional awareness, and lifestyle modifications.

16. Support Systems for People with CF** Cystic Fibrosis Foundation:** Provides research funding, patient support, and advocacy.
* **Local CF Centers :** Offer specialized care and support groups.
* **Online Communities:** Connect with other people living with CF.

17. Lifestyle Modifications Regular Exercise:** Improves lung function and overall health.
* **Smoking Cessation:** Smoking exacerbates lung disease.
* **Vaccinations:** Prevent infections.
* **Healthy Diet:** Ensures adequate nutrition and energy levels

18. Research and Advocacy** Ongoing Research:**  Advances in treatment and cure. * **Advocacy Efforts:**  Raise awareness, increase funding, and improve access to care. * **Patient Advocacy Groups:**  Support research and advocate for patient rights.

19. Conclusions * ** Pharmacotherapeutic advances in CF treatment have significantly improved quality of life and life expectancy.**
* **Continued research and development hold promise for a cure.**
* **Multidisciplinary care and patient empowerment are essential for optimal outcomes.**

20. Thank You