p.3
Treatment Options for Autoimmune PAP
What factors influence the choice of treatment options for autoimmune PAP?
The severity of the disease.
p.3
Overview of Pulmonary Alveolar Proteinosis (PAP)
What is the rate of spontaneous remission among patients with mild autoimmune PAP?
High rate of spontaneous remission.
p.5
Whole Lung Lavage (WLL) Procedure
How is the patient positioned during the WLL procedure?
In the lateral recumbent position with the lung to be lavaged nondependent.
p.2
Treatment Options for Autoimmune PAP
What was observed in nonresponders receiving subcutaneous GM-CSF?
GM-CSF antibody titers did not decline.
p.2
Treatment Options for Autoimmune PAP
What occurs to serum GM-CSF antibody titers in patients receiving nebulized GM-CSF?
Serum GM-CSF antibody titers increase despite clinical improvement.
p.5
Whole Lung Lavage (WLL) Procedure
What should be the maximum volume of unrecovered fluid in the lungs during WLL?
Not more than 200 to 300 mL.
p.2
Assessment of Disease Severity in PAP
How do initial BAL fluid levels of GM-CSF antibodies correlate with PAP severity?
They correlate better with the severity of PAP than serum titers.
p.7
Recombinant GM-CSF Therapy
When is recombinant GM-CSF therapy reserved for patients?
For patients who cannot undergo or have failed WLL.
p.1
Forms and Causes of PAP
What are the underlying causes of secondary PAP?
Certain hematologic/immunologic/metabolic diseases, inhalational exposures, and infections.
p.3
Assessment of Disease Severity in PAP
What characterizes moderate-to-severe symptoms in PAP patients?
Dyspnea with minimal exertion or at rest and requiring supplemental oxygen.
p.1
Forms and Causes of PAP
What causes primary PAP?
Disorders of GM-CSF signaling due to GM-CSF antibodies.
p.6
Whole Lung Lavage (WLL) Procedure
What monitoring is necessary during the WLL procedure?
Oxygenation, dynamic lung compliance, endotracheal tube position, and recovery of infused saline.
p.3
Management of Secondary and Congenital PAP
Why should patients with PAP be advised not to smoke cigarettes?
Smoking requires twice as many sessions of whole lung lavage compared to nonsmokers.
p.7
Recombinant GM-CSF Therapy
What improvement was observed in the largest randomized trial of GM-CSF therapy?
Significant improvement in AaO gradient and DLCO compared to placebo.
p.2
Assessment of Disease Severity in PAP
What is assessed to determine the appropriate treatment for PAP?
Severity of PAP using symptoms, serum LDH levels, GM-CSF antibody titers, pulmonary function tests, and HRCT imaging.
p.3
Assessment of Disease Severity in PAP
What are the proportions of stable, improved, and worsening disease among symptomatic patients?
About 45% stable, 30% improved, and 25% worsening.
p.1
Forms and Causes of PAP
What is secondary PAP?
PAP that occurs due to underlying disorders affecting alveolar macrophage function, such as certain hematologic, immunologic, or metabolic diseases.
p.6
Whole Lung Lavage (WLL) Procedure
What is obtained after the WLL procedure to check for complications?
An upright chest radiograph.
p.1
Overview of Pulmonary Alveolar Proteinosis (PAP)
What is pulmonary alveolar proteinosis (PAP)?
A diffuse lung disease characterized by the accumulation of amorphous, PAS-positive lipoproteinaceous material in the distal air spaces.
p.2
Assessment of Disease Severity in PAP
What pulmonary function tests are used in assessing PAP severity?
Lung volumes, diffusing capacity, and six-minute walk with oxygen saturation determination.
p.4
Assessment of Disease Severity in PAP
What is the management approach for asymptomatic or mild disease in patients with PAP?
They do not require immediate treatment and can be observed with periodic reassessment of symptoms, oxygen saturation, pulmonary function tests, and/or chest imaging.
p.6
Whole Lung Lavage (WLL) Procedure
What are some complications associated with WLL?
Malpositioning of the endotracheal tube, saline spillover, intraprocedural hypoxemia, pneumothorax, and hydropneumothorax.
p.7
Recombinant GM-CSF Therapy
What was the improvement in DLCO for the continuous GM-CSF group compared to placebo?
12.0 percent versus 4.2 percent.
p.9
Treatment Options for Autoimmune PAP
What investigational therapies are considered for refractory disease?
Rituximab, therapeutic plasma exchange, participation in clinical trials, or lung transplantation.
p.9
Rituximab and Other Investigational Therapies
What improvements were observed after rituximab treatment in the study?
Significant improvements in arterial oxygen tension (PaO2) and A-aO2 difference at three and six months.
p.10
Treatment Options for Autoimmune PAP
What is therapeutic plasma exchange (TPE) used for in patients with autoimmune PAP?
It is used for patients who have failed to improve with whole lung lavage (WLL).
p.14
Investigational Therapies
What therapies have been employed for refractory autoimmune PAP?
Rituximab and therapeutic plasma exchange.
p.12
Management of Secondary and Congenital PAP
How are patients with lysinuric protein intolerance managed?
With dietary modification, occasionally WLL, and rarely inhaled GM-CSF in refractory cases.
p.18
Overview of Pulmonary Alveolar Proteinosis (PAP)
What condition is associated with anti-GM-CSF antibodies in pediatric patients?
Pulmonary alveolar proteinosis.
p.5
Whole Lung Lavage (WLL) Procedure
What type of endotracheal tube is used during WLL?
A double-lumen endotracheal tube.
p.5
Whole Lung Lavage (WLL) Procedure
What technique is suggested to enhance the efficacy of WLL?
Repetitive manual hyperinflation and intermittent chest percussions.
p.11
Treatment Options for Autoimmune PAP
What is the optimal treatment for secondary, hereditary, and congenital PAP?
The optimal treatment has not been determined.
p.14
Whole Lung Lavage (WLL) Procedure
How is the whole lung lavage typically performed?
One lung is lavaged in the first procedure, followed by the opposite lung one to two weeks later.
p.13
Prognosis and Future Directions in PAP Treatment
What does the development of HRCT findings consistent with parenchymal fibrosis indicate?
It portends a poorer outcome.
p.8
Recombinant GM-CSF Therapy
What was the mean change in alveolar-arterial oxygen gradient for the GM-CSF group compared to placebo?
−4.50±9.03 mm Hg versus 0.17±10.50 mm Hg.
p.1
Assessment of Disease Severity in PAP
What does radiographic imaging typically reveal in PAP patients?
Bilateral symmetric alveolar opacities located centrally in mid and lower lung zones, often in a 'bat wing' distribution.
p.3
Assessment of Disease Severity in PAP
What should be monitored during the treatment or observation of PAP?
Symptoms, spirometry, diffusing capacity, ambulatory oximetry, and chest CT scan.
p.7
Recombinant GM-CSF Therapy
How is recombinant GM-CSF administered for inhalational therapy?
Reconstituted in 2 mL of normal saline and administered via nebulizer.
p.11
Management of Secondary and Congenital PAP
What is the most important intervention for secondary PAP due to inhalational exposures?
Completely discontinue further exposures.
p.13
Prognosis and Future Directions in PAP Treatment
What hypothesis has been proposed regarding some cases of autoimmune PAP?
They may result from an acquired clonal disorder of hematopoietic cells.
p.14
Management of Secondary and Congenital PAP
What is the treatment approach for secondary and hereditary PAP?
Treatment options depend on the specific cause and may include whole lung lavage for moderate-to-severe symptoms.
p.15
Overview of Pulmonary Alveolar Proteinosis (PAP)
What did the study by Kariman et al. in 1984 investigate?
Prospective clinical experience in 23 patients with pulmonary alveolar proteinosis over 15 years.
p.20
Rituximab and Other Investigational Therapies
What did the open-label trial of rituximab therapy in pulmonary alveolar proteinosis demonstrate?
It provided evidence for the efficacy of rituximab in treating the condition.
p.20
Rituximab and Other Investigational Therapies
How does rituximab therapy affect alveolar macrophage lipid homeostasis?
It improves lipid homeostasis in alveolar macrophages.
p.20
Treatment Options for Autoimmune PAP
What treatment options were discussed alongside plasmapheresis for alveolar proteinosis?
GM-CSF and other therapies.
p.21
Treatment Options for Autoimmune PAP
What was the outcome of statin therapy in a case report for pulmonary alveolar proteinosis?
Successful treatment without hypercholesterolemia.
p.21
Treatment Options for Autoimmune PAP
What was the outcome of corticosteroid administration in autoimmune pulmonary alveolar proteinosis?
Retrospective cohort study results.
p.17
Management of Secondary and Congenital PAP
What did the review by Vymazal and Krecmerova focus on?
Respiratory strategies and airway management in patients with pulmonary alveolar proteinosis.
p.19
Recombinant GM-CSF Therapy
What did Seymour et al. (1998) find regarding the hematopoietic response to GM-CSF?
They observed an attenuated response in patients with acquired pulmonary alveolar proteinosis.
p.6
Recombinant GM-CSF Therapy
What is the purpose of recombinant GM-CSF in the context of PAP?
To improve gas exchange and support patients during procedures like WLL.
p.1
Forms and Causes of PAP
What is congenital PAP?
Disorders of surfactant production or metabolism due to genetic variants in surfactant and other proteins.
p.2
Treatment Options for Autoimmune PAP
What happens to GM-CSF antibody titers in responders to subcutaneous GM-CSF treatment?
Serial GM-CSF antibody titers decline.
p.7
Recombinant GM-CSF Therapy
What is the advantage of nebulized GM-CSF over subcutaneous administration?
Greater ease of administration and possibly better response.
p.4
Treatment Options for Autoimmune PAP
What treatment options are available for patients with moderate-to-severe respiratory impairment?
Whole lung lavage (WLL) or a trial of experimental treatment such as subcutaneous or inhaled GM-CSF, or rituximab.
p.9
Recombinant GM-CSF Therapy
What adverse effects are associated with GM-CSF therapy?
Mild effects including fever, fatigue, headache, and injection site complications.
p.12
Prognosis and Future Directions in PAP Treatment
What potential treatment may reverse PAP due to GM-CSF receptor variants?
Hematopoietic stem cell transplantation (HSCT).
p.14
Management of Secondary and Congenital PAP
What is lung transplantation reserved for in the context of PAP?
Selected patients with severe, refractory PAP.
p.15
Overview of Pulmonary Alveolar Proteinosis (PAP)
What did the study by Inoue et al. in 2008 characterize?
A large cohort of patients with autoimmune pulmonary alveolar proteinosis in Japan.
p.15
Infectious Complications Associated with PAP
What infectious complications are associated with PAP?
Increased risk of superinfection with opportunistic organisms such as Nocardia, mycobacteria, and various fungi.
p.15
Management of Secondary and Congenital PAP
What specific therapies may respond in certain types of secondary PAP?
Treatment of underlying hematologic malignancy, hematopoietic stem cell transplantation, and avoidance of occupational dust exposures.
p.17
Whole Lung Lavage (WLL) Procedure
What comparison was made in the study by Hammon et al. regarding pulmonary alveolar proteinosis?
Comparison of manual to mechanical chest percussion for clearance of alveolar material.
p.17
Whole Lung Lavage (WLL) Procedure
What was the finding of Grutters et al. regarding whole lung lavage treatment?
Increased efficacy using a new modified lavage technique.
p.22
Infectious Complications Associated with PAP
What role do GM-CSF autoantibodies play in pulmonary alveolar proteinosis?
They are associated with neutrophil dysfunction.
p.19
Recombinant GM-CSF Therapy
What was the focus of the study by Tian et al. (2020)?
The effectiveness of inhaled GM-CSF for mild-to-moderate autoimmune pulmonary alveolar proteinosis over six months.
p.6
Whole Lung Lavage (WLL) Procedure
What is used to support patients with severely compromised gas exchange during WLL?
Extracorporeal membrane oxygenation.
p.3
Assessment of Disease Severity in PAP
What characterizes asymptomatic or mild symptoms in PAP patients?
Little or no physiologic impairment, normal to mildly reduced DLCO, and normal SpO2 at rest.
p.4
Whole Lung Lavage (WLL) Procedure
What are some indications for considering whole lung lavage?
Resting PaO2 <65 mmHg, A-aO2 difference ≥ 40 mmHg at rest, severe dyspnea and hypoxemia at rest or on exercise.
p.4
Whole Lung Lavage (WLL) Procedure
What are some contraindications for whole lung lavage?
Uncorrectable clotting disorders, anesthetic risks, and cardiopulmonary instability.
p.9
Treatment Options for Autoimmune PAP
What is the optimal therapy for patients with refractory disease despite WLL and GM-CSF therapy?
The optimal therapy is not known and must be decided on a case-by-case basis.
p.9
Rituximab and Other Investigational Therapies
What was the dosage of rituximab in the phase II study for autoimmune PAP?
1 g infused intravenously for two doses about two weeks apart.
p.10
Treatment Options for Autoimmune PAP
What was the outcome of one patient who underwent therapeutic plasma exchange?
One patient did not improve, while another responded to low intensity TPE over two months.
p.11
Whole Lung Lavage (WLL) Procedure
What were the results of WLL for patients with PAP following inhalational exposure?
Results have been mixed; some patients responded while others did not.
p.12
Prognosis and Future Directions in PAP Treatment
What does HSCT aim to prevent in patients with PAP?
The return of alveolar macrophages with aberrant GM-CSF receptors.
p.10
Management of Secondary and Congenital PAP
What has been noted about lung transplantation in patients with autoimmune PAP?
Some patients developed recurrent disease in the lung allograft.
p.20
Rituximab and Other Investigational Therapies
What is the therapeutic effectiveness of rituximab in autoimmune pulmonary alveolar proteinosis?
Rituximab has shown effectiveness in patients unresponsive to other treatments.
p.16
Overview of Pulmonary Alveolar Proteinosis (PAP)
What syndrome is discussed in the Clin Chest Med 2016 article by Suzuki and Trapnell?
Pulmonary Alveolar Proteinosis Syndrome.
p.18
Recombinant GM-CSF Therapy
What therapy was tested in the 2010 Am J Respir Crit Care Med publication for pulmonary alveolar proteinosis?
Inhaled granulocyte/macrophage-colony stimulating factor.
p.18
Recombinant GM-CSF Therapy
What was reported in the 2001 Thorax publication regarding GM-CSF therapy?
A complete response to GM-CSF therapy in pulmonary alveolar proteinosis.
p.18
Recombinant GM-CSF Therapy
What did the 2024 Eur Respir J study find about inhaled recombinant GM-CSF?
It reduces the need for whole lung lavage and improves gas exchange in autoimmune pulmonary alveolar proteinosis patients.
p.16
Whole Lung Lavage (WLL) Procedure
What procedure was discussed in the 2009 Chest article by Michaud et al.?
Whole-lung lavage for pulmonary alveolar proteinosis.
p.19
Recombinant GM-CSF Therapy
What is the role of aerosol granulocyte-macrophage colony-stimulating factor (GM-CSF) in pulmonary alveolar proteinosis?
It is used to improve lung clearance in patients with the condition.
p.19
Recombinant GM-CSF Therapy
What was the outcome of the study on inhaled Molgramostim therapy in autoimmune pulmonary alveolar proteinosis?
It showed effectiveness in treating the condition.
p.3
Management of Secondary and Congenital PAP
What vaccinations should patients with PAP obtain annually?
Influenza, COVID-19, pneumococcal pneumonia, and respiratory syncytial virus.
p.7
Recombinant GM-CSF Therapy
What was the outcome of sequential treatment with inhaled GM-CSF after WLL?
It greatly reduced the need for repeat WLL over 30 months of follow-up.
p.14
Treatment Options for Autoimmune PAP
Are glucocorticoids indicated for autoimmune PAP?
No, glucocorticoids are not indicated for autoimmune PAP.
p.10
Treatment Options for Autoimmune PAP
How might statins be beneficial for patients with autoimmune PAP?
They may improve surfactant uptake and clearance by alveolar macrophages.
p.12
Prognosis and Future Directions in PAP Treatment
What was observed in a patient with a CSF2RB mutation after lung transplantation?
Developed recurrent PAP after nine months.
p.15
Assessment of Disease Severity in PAP
What does the study by Sui et al. in 2017 assess?
Quantitative assessment of pulmonary alveolar proteinosis with ultra-dose CT and correlation with pulmonary function tests.
p.16
Overview of Pulmonary Alveolar Proteinosis (PAP)
What is the clinical significance of anti-GM-CSF antibodies in idiopathic pulmonary alveolar proteinosis according to Lin et al.?
It was discussed in their 2006 study published in Thorax.
p.16
Recombinant GM-CSF Therapy
What was the focus of the 2017 Ann Am Thorac Soc report by Ohkouchi et al.?
Sequential Granulocyte-Macrophage Colony-Stimulating Factor Inhalation after Whole-Lung Lavage.
p.16
Overview of Pulmonary Alveolar Proteinosis (PAP)
What did the 1965 Ann Intern Med report by Larson and Gordinier discuss?
Six cases of pulmonary alveolar proteinosis and a new theory.
p.16
Overview of Pulmonary Alveolar Proteinosis (PAP)
What insights were gained from the single-center cohort of 70 patients in the 2011 Respir Med article by Bonella et al.?
New insights into pulmonary alveolar proteinosis.
p.17
Prognosis and Future Directions in PAP Treatment
What progress is discussed in the study by Seymour and Presneill regarding pulmonary alveolar proteinosis?
Progress in the first 44 years of understanding and treatment.
p.19
Recombinant GM-CSF Therapy
What was the finding of Ohashi et al. (2012) regarding GM-CSF inhalation?
It provides direct evidence that GM-CSF inhalation improves lung clearance in pulmonary alveolar proteinosis.
p.19
Recombinant GM-CSF Therapy
What did Ohashi et al. (2012) report about GM-CSF autoantibodies?
They found reduced GM-CSF autoantibodies in improved lung conditions of autoimmune pulmonary alveolar proteinosis patients.
p.4
Whole Lung Lavage (WLL) Procedure
What is the most widely accepted and effective treatment for patients with moderate-to-severe symptoms and hypoxemia?
Whole lung lavage (WLL) under general anesthesia.
p.6
Treatment Options for Autoimmune PAP
How often do some patients require repeat lung lavages after WLL?
At intervals of 6 to 12 months.
p.6
Assessment of Disease Severity in PAP
What initial DLCO percentage is associated with a higher likelihood of requiring additional WLL?
42 percent predicted or less.
p.11
Management of Secondary and Congenital PAP
What vaccinations are advised for all patients with PAP?
Annual vaccinations against influenza and COVID-19, and age-appropriate vaccinations against pneumococcal pneumonia and respiratory syncytial virus.
p.9
Recombinant GM-CSF Therapy
What were the initial and subsequent dosages of subcutaneous GM-CSF in the trial?
Starting at 250 mcg/day for one month, increasing to 5 mcg/kg/day for the second month, and 9 mcg/kg/day for the third month.
p.14
Recombinant GM-CSF Therapy
What may recombinant GM-CSF help reduce in patients with autoimmune PAP?
The need for or frequency of recurrent whole lung lavage.
p.12
Infectious Complications Associated with PAP
Why do patients with PAP have an increased risk of infections?
Due to impaired macrophage and neutrophil function.
p.13
Management of Secondary and Congenital PAP
What vaccinations should all patients with PAP be counseled to obtain annually?
Influenza, COVID-19, respiratory syncytial virus, and pneumococcal pneumonia (if no contraindications).
p.10
Treatment Options for Autoimmune PAP
What has been observed in patients with autoimmune PAP treated with statins?
Improvements in lung function, oxygenation, and radiologic findings.
p.17
Whole Lung Lavage (WLL) Procedure
What aspect of whole lung lavage is discussed in the study by Tan et al.?
Anesthetic management for whole lung lavage in patients with pulmonary alveolar proteinosis.
p.17
Whole Lung Lavage (WLL) Procedure
What did Bonella et al. study in their 2012 research?
Wash-out kinetics and efficacy of a modified lavage technique for alveolar proteinosis.
p.17
Overview of Pulmonary Alveolar Proteinosis (PAP)
What condition is described in the study by Nimmatoori et al.?
Pulmonary alveolar proteinosis characterized by milky fluid from the lungs.
p.21
Forms and Causes of PAP
What occupational exposure was linked to pulmonary alveolar proteinosis in a 2010 study?
Indium processing facility.
p.4
Rituximab and Other Investigational Therapies
What is the role of rituximab in the treatment of PAP?
It may be tried if there is inadequate response to whole lung lavage and GM-CSF or if they are associated with unacceptable side effects.
p.9
Recombinant GM-CSF Therapy
What was the response rate to subcutaneous recombinant GM-CSF?
Slightly less than 50 percent.
p.11
Treatment Options for Autoimmune PAP
What treatment may improve secondary PAP due to hematologic dyscrasias?
Treatment of the underlying hematologic malignancy or myelodysplasia.
p.9
Recombinant GM-CSF Therapy
What correlation was observed with declining serum GM-CSF antibody levels?
They appear to correlate with a positive response to GM-CSF therapy.
p.10
Prognosis and Future Directions in PAP Treatment
What is a potential future direction for treating hereditary PAP?
Derivation of macrophages from gene-corrected pluripotent stem cells.
p.13
Management of Secondary and Congenital PAP
What is recommended for patients with minimal or mild disease of PAP?
Supportive care with supplemental oxygen, as needed.
p.12
Management of Secondary and Congenital PAP
What congenital disorders are associated with PAP?
Variants in genes for surfactant proteins (SP)-B or C, and others involved in surfactant metabolism.
p.15
Recombinant GM-CSF Therapy
What does the research by Bonfield et al. in 2002 suggest about anti-GM-CSF titer?
It predicts response to GM-CSF therapy in pulmonary alveolar proteinosis.
p.18
Recombinant GM-CSF Therapy
What is the focus of the open-label trial mentioned in the 2006 Chest publication?
Granulocyte macrophage colony stimulating factor therapy for moderate symptomatic pulmonary alveolar proteinosis.
p.16
Overview of Pulmonary Alveolar Proteinosis (PAP)
What did Claypool et al. update in their 1984 Chest article?
Clinical diagnosis, management, and pathogenesis of pulmonary alveolar proteinosis.
p.16
Whole Lung Lavage (WLL) Procedure
What long-term benefit was reported by Beccaria et al. in their 2004 study?
Durable benefit after whole lung lavage in pulmonary alveolar proteinosis.
p.21
Management of Secondary and Congenital PAP
What can secondary alveolar proteinosis cause in leukemic patients?
Reversible respiratory failure.
p.22
Prognosis and Future Directions in PAP Treatment
What mutation was identified in the patient who experienced recurrence of pulmonary alveolar proteinosis after bilateral lung transplantation?
A nonsense mutation in CSF2RB.
p.22
Infectious Complications Associated with PAP
What is the significance of nocardia in relation to pulmonary alveolar proteinosis?
It can induce granulocyte macrophage colony-stimulating factor, which is neutralized by autoantibodies.
p.4
Treatment Options for Autoimmune PAP
What is the suggested treatment approach for patients with moderate to severe symptoms?
A stepwise approach beginning with whole lung lavage, followed by inhaled GM-CSF if necessary.
p.12
Infectious Complications Associated with PAP
What is the increased risk for patients with PAP?
Superinfection with opportunistic organisms such as Nocardia, actinomyces, mycobacteria, and various fungi.
p.10
Treatment Options for Autoimmune PAP
What role do glucocorticoids play in the treatment of autoimmune PAP?
There is no role for glucocorticoids or other immunosuppressive agents as primary therapy.
p.20
Rituximab and Other Investigational Therapies
What type of study was conducted on rituximab for autoimmune alveolar proteinosis?
A real-life cohort study.
p.20
Treatment Options for Autoimmune PAP
What was the focus of the plasmapheresis protocol for pulmonary alveolar proteinosis?
It was designed for refractory cases.
p.20
Management of Secondary and Congenital PAP
What was reported in the case of living-donor lobar lung transplantation for pulmonary alveolar proteinosis?
Successful treatment in an adult patient.
p.20
Overview of Pulmonary Alveolar Proteinosis (PAP)
What advancement was made in gene correction for pulmonary alveolar proteinosis?
Human induced pluripotent stem cells were corrected to repair the cellular phenotype.
p.17
Recombinant GM-CSF Therapy
What treatment is discussed by Kavuru et al. for pulmonary alveolar proteinosis?
Exogenous granulocyte-macrophage colony-stimulating factor administration.
p.19
Recombinant GM-CSF Therapy
What did the systematic review by Munsif et al. (2023) focus on?
The use of nebulized GM-CSF in autoimmune pulmonary alveolar proteinosis.
p.19
Rituximab and Other Investigational Therapies
What is the significance of Rituximab therapy in autoimmune pulmonary alveolar proteinosis according to Borie et al. (2009)?
It is explored as a treatment option for the condition.
p.10
Treatment Options for Autoimmune PAP
What percentage of patients with refractory autoimmune PAP showed objective improvement after treatment with Rituximab?
30 percent (4 out of 13 patients).
p.14
Recombinant GM-CSF Therapy
Is recombinant GM-CSF approved for use in autoimmune PAP?
No, it is not approved for autoimmune PAP.
p.9
Recombinant GM-CSF Therapy
What challenge do many patients with autoimmune PAP face regarding GM-CSF therapy?
They may not manifest a normal hematologic response and may require very high doses for a modest elevation in white blood cell count.
p.8
Recombinant GM-CSF Therapy
What effect did GM-CSF inhalation have on GM-CSF autoantibodies?
It was associated with a decrease in GM-CSF autoantibodies.
p.15
Overview of Pulmonary Alveolar Proteinosis (PAP)
What is the focus of the study by Shah et al. in 2000?
Clinical aspects and current concepts on the pathogenesis of pulmonary alveolar proteinosis.
p.21
Forms and Causes of PAP
What defect is associated with human pulmonary alveolar proteinosis according to a 1997 study?
Defect in GM-CSF/IL-3/IL-5 receptor common beta chain expression.
p.21
Management of Secondary and Congenital PAP
What is secondary pulmonary alveolar proteinosis associated with?
Hematologic malignancies.
p.21
Forms and Causes of PAP
What event led to the development of pulmonary alveolar proteinosis due to dust exposure?
The Great East Japan Earthquake.
p.22
Infectious Complications Associated with PAP
What type of infections are patients with pulmonary alveolar proteinosis at risk for?
Opportunistic infections.
p.22
Overview of Pulmonary Alveolar Proteinosis (PAP)
What are the clinical features and outcomes of pulmonary alveolar proteinosis as discussed by Goldstein et al.?
The study provides insights into the clinical features and outcomes of the disease.
p.8
Recombinant GM-CSF Therapy
What was the outcome regarding adverse events between GM-CSF and placebo-treated patients?
No significant difference.
p.10
Prognosis and Future Directions in PAP Treatment
What is the significance of clinical trials for patients with PAP?
Patients may wish to participate in trials for treatments like rituximab and GM-CSF.
p.15
Overview of Pulmonary Alveolar Proteinosis (PAP)
What was the focus of the research by Kumar et al. in 2018?
Pathophysiology and clinical approach to pulmonary alveolar proteinosis in adults.
p.17
Whole Lung Lavage (WLL) Procedure
What is the focus of the study by Gay et al. published in Respiration in 2017?
Efficacy of Whole-Lung Lavage in Pulmonary Alveolar Proteinosis.
p.17
Whole Lung Lavage (WLL) Procedure
What type of survey did Campo et al. conduct in their 2016 study?
A global survey of current practices and procedures for whole lung lavage therapy.
p.20
Management of Secondary and Congenital PAP
What complication was noted following double lung transplantation?
Recurrent alveolar proteinosis.
p.20
Overview of Pulmonary Alveolar Proteinosis (PAP)
What innovative approach was used for modeling hereditary pulmonary alveolar proteinosis?
Murine iPSC-derived macrophages.
p.19
Recombinant GM-CSF Therapy
What did the study by Robinson et al. (2009) analyze?
The longitudinal response to aerosolized GM-CSF in adolescents with autoimmune pulmonary alveolar proteinosis.
p.22
Infectious Complications Associated with PAP
What was the focus of the study by Mabo et al. regarding pulmonary alveolar proteinosis?
Infections in autoimmune pulmonary alveolar proteinosis.
p.18
Recombinant GM-CSF Therapy
What was the outcome of GM-CSF therapy in acquired alveolar proteinosis according to the 1996 N Engl J Med study?
Efficacy of granulocyte-macrophage colony-stimulating factor.
p.18
Assessment of Disease Severity in PAP
What did the 2003 Thorax study investigate in relation to GM-CSF therapy?
The relationship of anti-GM-CSF antibody concentration, surfactant protein levels, and serum LDH to pulmonary parameters and response to GM-CSF therapy.
p.18
Recombinant GM-CSF Therapy
What was the focus of the meta-analysis conducted by Khan et al. in 2012?
Effectiveness of granulocyte-macrophage colony-stimulating factor therapy in autoimmune pulmonary alveolar proteinosis.
p.21
Management of Secondary and Congenital PAP
How does secondary pulmonary alveolar proteinosis complicate myelodysplastic syndrome?
It results in worsening of prognosis.
p.21
Treatment Options for Autoimmune PAP
What are the two transplant options discussed for pulmonary alveolar proteinosis?
Lung transplant or bone marrow transplant.
