Clinical trial NCT01232504: Antifugal Effect of Recombinant Human Granulocyte-macrophage Stimulating Factor (rhGM-CSF) in Patients Post Allo-HSCT
Official title: Effect of Granulocyte-macrophage Stimulating Factor on Prevention and Treatment of Invasive Fungal Diseases in the Recipients of Allogeneic Stem Cell Transplantation: A Prospective Multicenter Randomized Phase 4 Trial
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE4
Conditions: Mycoses
Keywords: Allo-HSCT
rhGM-CSF
Systemic fungal infection (SFI)
Lead sponsor: Xiamen Amoytop Biotech Co., Ltd.
Sponsor class: INDUSTRY
Collaborators: name: Shanghai Jiao Tong University Affiliated First People's Hospital
First posted: 2010-11-02
Enrollment: 206
Sex: ALL
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: PREVENTION

Brief summary: We conducted a prospective, multicenter, open-label randomized trial to compare the antifungal effect of Recombinant Human Granulocyte-macrophage Stimulating Factor (rhGM-CSF), Recombinant Human Granulocyte Stimulating Factor (rhG-CSF) or a combination of rhGM-CSF and rhG-CSF for neutropenic patients undergoing allogeneic stem cell transplantation.

Detailed description: From Sept 2009 to Dec 2012, we recruited consecutive patients with hematological diseases undergoing allogeneic stem cell transplantation at 5 institutions in China. Recipients between ages of 14 to 60 years old were eligible. Eligible patients were randomized to receive once daily subcutaneous 5-7μg/kg/d GM-CSF (Molgramostim, TOPLEUCON®; Xiamen Amoytop Biotech Co., Ltd., China) (GM-CSF group), 5-7μg/kg/d G-CSF (G-CSF group), or a combination of 2-3μg/kg/d GM-CSF and 2-3μg/kg/d G-CSF each (G-CSF+GM-CSF group). Administration of CSFs was started on day 5 post-transplant and continued until recovery from neutropenia (absolute neutrophil count \[ANC\] \> 1.5×10(9)/L for 2 consecutive days). If absolute neutrophil count (ANC) decreased to \< 1.5×10(9)/L within 5 days after withdrawal of CSFs, the same CSF would be resumed until the absolute neutrophil count (ANC) reached 1.5×10(9)/L again. All patients received antimicrobial prophylaxis with oral levofloxacin 500 mg daily and antifungal prophylaxis with oral fluconazole 200 mg daily.

Eligibility criteria: Inclusion Criteria:

* Age 14\~60 years old
* Allogenic hematological stem cell transplantation(HSCT) patients.
* Cardiac ejection factor ≥ normal upper limit, Aspartate aminotransferase and/or Alanine aminotransferase \< 2 upper limit of normal, and/or total bilirubin \< 2.5 upper limit of normal, creatinine \< upper limit of normal.
* Informed consent.

Exclusion Criteria:

* Evidence of proven, probable or possible fungal infection at the time of enrollment.
* Patients were receiving anti-fungal treatment with proven SFI before transplantation.
* A history of hypersensitivity to G-CSF or GM-CSF.

Primary outcomes:
measure: Incidences of Invasive Fungal Diseases (IFD)
timeFrame: 100 day post transplant
Secondary outcomes:
measure: Hematological Engraftment
timeFrame: 100 days post transplant
measure: Transplant Related Mortality
timeFrame: 100 days post transplant
measure: Incidence of Ⅱ- Ⅳ Acute Graft Versus Host Disease (aGVHD)
timeFrame: 100 days post transplant
measure: IFD Related Mortality
timeFrame: 3-1099 days
measure: Relapse Related Mortality
timeFrame: 3～1099 days
measure: Graft Versus Host Disease (aGVHD) Related Mortality
timeFrame: 3-1099 days
measure: Hemorrhage Related Mortality
timeFrame: 3-1099 days
measure: Infection Related Mortality
timeFrame: 3～1099 days)
Other outcomes:
Locations:
city: Nanning
country: China
facility: The First Affiliated Hospital of Guangxi Medical University
state: Guangxi
city: Zhengzhou
country: China
facility: Henan Cancer Hospital
state: Henan
city: Wuhan
country: China
facility: Wuhan Tongji Hospital
state: Hubei
city: Shanghai
country: China
facility: Shanghai First People's Hospital
state: Shanghai
city: Urumqi
country: China
facility: The First Affiliated Hospital of Xinjiang Medical University
state: Xinjiang
Study officials:
affiliation: Shanghai General Hospital, Shanghai Jiao Tong University School of Medicine
name: Chun Wang, M.D.
role: PRINCIPAL_INVESTIGATOR
References:
citation: Wan L, Zhang Y, Lai Y, Jiang M, Song Y, Zhou J, Zhang Z, Duan X, Fu Y, Liao L, Wang C. Effect of Granulocyte-Macrophage Colony-Stimulating Factor on Prevention and Treatment of Invasive Fungal Disease in Recipients of Allogeneic Stem-Cell Transplantation: A Prospective Multicenter Randomized Phase IV Trial. J Clin Oncol. 2015 Dec 1;33(34):3999-4006. doi: 10.1200/JCO.2014.60.5121. Epub 2015 Sep 21.