OPTIMA: An Open-Label, Non-comparative Pilot Trial of Inhaled Molgramostim in Pulmonary Nontuberculous Mycobacterial Infection. Inhaled granulocyte-macrophage colony stimulating factor (GM-CSF) has been proposed as a potential immunomodulatory treatment for nontuberculous mycobacterial (NTM) infection. This open label, non-comparative pilot trial investigated the efficacy and safety of inhaled GM-CSF (molgramostim nebulizer solution) in patients with predominantly treatment-refractory pulmonary NTM (M. avium complex (MAC), M. abscessus (MABS)) infection, either in combination with ongoing guideline-based therapy (GBT), or as monotherapy in patients who had stopped GBT due to lack of efficacy or intolerability. 32 adult patients with refractory NTM (MAC 24, MABS 8) were recruited into two
Inhaled Molgramostim Therapy in Autoimmune Pulmonary Alveolar Proteinosis. Autoimmune pulmonary alveolar proteinosis (aPAP) is a rare disease characterized by progressive surfactant accumulation and hypoxemia. It is caused by disruption of granulocyte-macrophage colony-stimulating factor (GM-CSF) signaling, which pulmonary alveolar macrophages require to clear surfactant. Recently, inhaled GM -CSF was shown to improve the partial pressure of arterial oxygen in patients with aPAP. In a double-blind, placebo-controlled, three-group trial, we randomly assigned patients with aPAP to receive the recombinant GM-CSF molgramostim (300 μg once daily by inhalation), either continuously or intermittently (every other week), or matching placebo. The 24-week intervention period was followed by an open
Shorter Total Length of Stay After Intraperitoneal Fosfomycin, Metronidazole, and Molgramostim for Complicated Appendicitis: A Pivotal Quasi-Randomized Controlled Trial. We aimed to investigate the difference in the total length of hospital stay (LOS) after intraperitoneal vs. intravenous antibiotic treatment in patients with complicated appendicitis. We conducted a quasi-randomized
Autoimmune pulmonary alveolar proteinosis in an adolescent successfully treated with inhaled rhGM-CSF (molgramostim) Autoimmune pulmonary alveolar proteinosis (aPAP) is a rare parenchymal lung disease characterized by accumulation of surfactant in the airways with high levels of granulocyte-macrophage colony stimulating factor (GM-CSF) antibodies in blood. Disease leads to hypoxemic respiratory with molgramostim - new recombinant human GM-CSF (rhGM-CSF).
Molgramostim Nebulizer Solution Expanded Access Program Protocol Autoimmune pulmonary alveolar proteinosis (aPAP) is a rare disorder in which a material called surfactant builds up in the lungs and makes it hard to breathe. In addition to shortness of breath, people with aPAP can experience persistent cough, overwhelming fatigue, unintentional changes in weight, chest or back pain, suddenly is studying an investigational drug called molgramostim nebulizer solution to see if it activates the cells that help clear surfactant from the lungs, which improves oxygen transfer from the lungs to the bloodstream. Molgramostim nebulizer solution is administered by inhalation using a hand-held nebulizer. In clinical trials, molgramostim nebulizer solution has shown improvements in gas exchange and patient
Inhaled Molgramostim in Pediatric Participants With Autoimmune Pulmonary Alveolar Proteinosis (aPAP). The goal of this open-label study is to study molgramostim as a treatment for autoimmune pulmonary alveolar proteinosis (aPAP) in pediatric patients between age 6 and 18. The main questions it aims to answer are: The effect of molgramostim on breathing tests and activity in pediatric patients with aPAP and the safety of molgramostim in pediatric patients with aPAP. This is an open-label study: all participants will receive treatment with molgramostim. Patients will: Take molgramostim once daily via nebulizer every day for 12 months.Visit the clinic approximately every 12 weeks for checkups and tests.Keep a diary of any oxygen use. This is an interventional open-label, single arm, multi-center
Clinical Trial of Inhaled Molgramostim Nebulizer Solution in Autoimmune Pulmonary Alveolar Proteinosis (aPAP) 160 subjects with autoimmune pulmonary alveolar proteinosis (aPAP) will be randomized to receive once daily treatment with inhaled molgramostim or placebo for 48 weeks. Subjects completing the 48 week placebo-controlled period will receive open-label treatment with once daily inhaled molgramostim for 48 weeks. This is an interventional, randomized, double-blind, 2-arm, parallel groups, placebo-controlled, multi-center, phase 3 trial in adult subjects who are diagnosed with aPAP.An aPAP diagnosis should be confirmed by an anti-GM-CSF auto-antibody test result, and history of PAP based on either high resolution computed tomography, lung biopsy, or bronchoalveolar lavage cytology, should
GM-CSF Inhalation to Prevent ARDS in COVID-19 Pneumonia To assess the safety and tolerability of inhaled molgramostim nebuliser solution in patients with COVID-19 pneumonia. COVID-19 pneumonia is induced by the newly emerging pandemic Severe acute respiratory Syndrome (SARS) coronavirus 2 and results in progression to the acute respiratory distress syndrome (ARDS). Apart from protective
Topical effectiveness of molgramostim (GM-CSF) in sickle cell leg ulcers. Leg ulcers are a frequently neglected, severe complication of sickle cell disease (SCD), responsible for significant altering quality of life. The management of leg ulcers remains disappointing. Local application of recombinant human granulocyte-macrophage colony-stimulating factor has been shown to be effective in several types of chronic wound healing. We report 5 homozygous sickle cell patients with 14 leg ulcers lasting from 1 month to 6 years, treated with topical dripping of molgramostim solution. Healing was obtained for 9/14 ulcers, with no local or systemic adverse reactions. Molgramostim solution appears to be an adequate therapy for SCD leg ulcers but a more conclusive evaluation will depend on larger series
Priming and treatment with molgramostim (rhGM-CSF) in adult high-risk acute myeloid leukemia during induction chemotherapy: a prospective, randomized pilot study. In a randomized study of 18 adult patients with high-risk or advanced acute myeloid leukemia (AML) we investigated the effect of supplementing conventional induction chemotherapy with recombinant human granulocyte-macrophage colony
Phase I trial of etoposide, doxorubicin and cisplatin (EAP) in combination with GM-CSF. The aim of this study was to ameliorate the toxicity of the etoposide, doxorubicin and cisplatin (EAP) regimen and to investigate the feasibility of dose escalation, using the molgramostim form of granulocyte macrophage-colony stimulating factor (GM-CSF) 10 micrograms/kg/day s.c. into the regimen. The design
Unexpected hepatotoxicity after priming and treatment with molgramostim (rhGM-CSF) in acute myeloid leukemia during induction chemotherapy. The effect of supplementing induction chemotherapy with recombinant human granulocyte-macrophage colony-stimulating factor (rhGM-CSF) was studied in a randomized trial of 18 patients with acute myeloid leukemia (AML). Ten patients received rhGM-CSF, starting
Long-term safety of GM-CSF (molgramostim) administration after allogeneic bone marrow transplantation for hematologic malignancies: five-year follow-up of a double-blind randomized placebo-controlled study. In a double-blind, randomized study performed between 1988 and 1990, 40 patients undergoing allogeneic BMT from HLA-identical siblings for hematologic malignancies received 8 mg/kg/d rHuGM-CSF (molgramostim, n = 20) for 14 days. The median neutrophil count on day 14 was significantly higher in the GM-CSF group (1.90 vs 0.46 yen 10(9)/L, P < .0001). The incidence of acute GVHD and transplant-related mortality were comparable. Only two deaths occurred after 6 months; one due to pulmonary fibrosis in the GM-CSF group on day 1591, and one due to relapse on day 1590 in the placebo group. The Karnofsky
A randomized study comparing filgrastim versus lenograstim versus molgramostim plus chemotherapy for peripheral blood progenitor cell mobilization. We conducted a prospective randomized clinical trial to assess the mobilizing efficacy of filgrastim, lenograstim and molgramostim following a disease-specific chemotherapy regimen. Mobilization consisted of high-dose cyclophosphamide in 45 cases (44 %), and cisplatin/ifosfamide/etoposide or vinblastine in 22 (21%), followed by randomization to either filgrastim or lenograstim or molgramostim at 5 microg/kg/day. One hundred and three patients were randomized, and 82 (79%) performed apheresis. Forty-four (43%) patients were chemonaive, whereas 59 (57%) were pretreated. A median number of one apheresis per patient (range, 1-3) was performed. The median number
Low doses of GM-CSF (molgramostim) and G-CSF (filgrastim) after cyclophosphamide (4 g/m2) enhance the peripheral blood progenitor cell harvest: results of two randomized studies including 120 patients. The use of a combination of G-CSF and GM-CSF versus G-CSF alone, after cyclophosphamide (4 g/m2) was compared in two randomized phase III studies, including 120 patients. In study A, 60 patients
of an exclusively systemic application of GM-CSF in patients with coronary artery disease (CAD) and collateral artery promotion has not been studied so far. In 14 men (age 61 +/- 11 years) with chronic stable CAD, the effect of GM-CSF (molgramostim) on quantitatively assessed collateral flow was tested in a randomized, double-blind, placebo-controlled fashion. The study protocol consisted of an invasive
Molgramostim (GM-CSF) associated with antibiotic treatment in nontraumatic abdominal sepsis: a randomized, double-blind, placebo-controlled clinical trial. The addition of molgramostim (recombinant human granulocyte-macrophage colony-stimulating factor) to antibiotic therapy for nontraumatic and generalized abdominal sepsis is effective and has a significant impact on length of hospitalization , direct medical costs, and mortality. Randomized, double-blind, placebo-controlled clinical trial. Tertiary referral center. Fifty-eight patients with abdominal sepsis. Patients were allocated to receive, in addition to ceftriaxone sodium, amikacin sulfate, and metronidazole, molgramostim in a daily dosage of 3 microg/kg for 4 days (group 1) or placebo (group 2). Antibiotics were administered
Long-term survival of patients with advanced/recurrent carcinoma of cervix and vagina after neoadjuvant treatment with methotrexate, vinblastine, doxorubicin, and cisplatin with or without the addition of molgramostim, and review of the literature. A randomized phase III study was conducted to assess the addition of molgramostim (GM-CSF) to the combination of methotrexate, vinblastine