Efficacy of Bone Marrow Mesenchymal Stem Cell in Pulmonary Hemosiderosis

Idiopathic Pulmonary Hemosiderosis Clinical Trial

Official title:

Sun Yat-sen Memorial Hospital

Clinical Trial Details — Status: Not yet recruiting

Administrative data

• NCT number: NCT02985346
• Other study ID #: WTan
• Status: Not yet recruiting
• Phase: Phase 0
• First received: December 3, 2016
• Last updated: December 6, 2016
• Start date: January 2017

Study information

• Verified date: December 2016
• Source: Sun Yat-sen University
• Contact: n/a
• Is FDA regulated: No
• Health authority: China: Health and Family Planning Commission of Guangdong Province
• Study type: Interventional

Clinical Trial Summary

Pulmonary hemosiderosis (PH) is a pulmonary hemosiderin deposition which caused by alveolar capillary hemorrhage. PH is easy to recurrent and can lead to pulmonary fibrosis and insufficiency if the disease was poor controlled. Steroid is the most common drug that was administered in acute phase of the disease. However, considered the side-effects, steroid is not suitable for long-time maintenance. Therefore, it is necessary to explore a new therapy. Bone marrow mesenchymal stem cells (BMSC) are a kind of adult stem cells with high self-renewal and multi-directional differentiation potential in bone marrow. It has become a hot topic in immunosuppressive and tissue repair therapy in recent years. To date, homing, colonization and differentiation of BMSCs in the lung have been observed in animal models of pulmonary hypertension, radiation pneumonitis and pulmonary fibrosis. It had been reported that BMSC transplantation in acute lung injury in mice, inflammation of lung injury can significantly improve. The aim of this study is to explore the effect of BMSC on PH and its mechanism, and to explore a new way to promote the repair of IPH. It is expected to improve the status of IPH therapy in children, especially improve the prognosis of refractory PH.

Recruitment information / eligibility

• Status: Not yet recruiting
• Enrollment: 100
• Est. primary completion date: December 2020
• Accepts healthy volunteers: Accepts Healthy Volunteers
• Gender: Both
• Age group: 1 Month to 18 Years

Eligibility

Inclusion Criteria:

• Patients diagnosed with Pulmonary hemosiderosis at an age less than 18 years.

Exclusion Criteria:

• Patients who cannot finish the established causes or die during the causes.

Study Design

Allocation: Non-Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Open Label, Primary Purpose: Treatment

Related Conditions & MeSH terms

• Hemosiderosis
• Idiopathic Pulmonary Hemosiderosis
• Lung Diseases

Intervention

Biological:
• Bone marrow mesenchymal stem cells in treatment of Idiopathic pulmonary hemosiderosis

Locations

Country	Name	City	State
n/a

Sponsors (1)

Lead Sponsor	Collaborator
Sun Yat-sen University

References & Publications (10)

Airaghi L, Ciceri L, Giannini S, Ferrero S, Meroni PL, Tedeschi A. Idiopathic pulmonary hemosiderosis in an adult. Favourable response to azathioprine. Monaldi Arch Chest Dis. 2001 Jun;56(3):211-3. — View Citation

Byrd RB, Gracey DR. Immunosuppressive treatment of idiopathic pulmonary hemosiderosis. JAMA. 1973 Oct 22;226(4):458-9. — View Citation

Calabrese F, Giacometti C, Rea F, Loy M, Sartori F, Di Vittorio G, Abudureheman A, Thiene G, Valente M. Recurrence of idiopathic pulmonary hemosiderosis in a young adult patient after bilateral single-lung transplantation. Transplantation. 2002 Dec 15;74(11):1643-5. — View Citation

Chen RL, Chuang SS. Silent idiopathic pulmonary hemosiderosis with iron-deficiency anemia but normal serum ferritin. J Pediatr Hematol Oncol. 2007 Jul;29(7):509-11. — View Citation

Hoffman AM, Paxson JA, Mazan MR, Davis AM, Tyagi S, Murthy S, Ingenito EP. Lung-derived mesenchymal stromal cell post-transplantation survival, persistence, paracrine expression, and repair of elastase-injured lung. Stem Cells Dev. 2011 Oct;20(10):1779-92 — View Citation

Knight DA, Rossi FM, Hackett TL. Mesenchymal stem cells for repair of the airway epithelium in asthma. Expert Rev Respir Med. 2010 Dec;4(6):747-58. doi: 10.1586/ers.10.72. Review. — View Citation

Lee JW, Fang X, Gupta N, Serikov V, Matthay MA. Allogeneic human mesenchymal stem cells for treatment of E. coli endotoxin-induced acute lung injury in the ex vivo perfused human lung. Proc Natl Acad Sci U S A. 2009 Sep 22;106(38):16357-62. doi: 10.1073/p — View Citation

Liu AR, Liu L, Chen S, Yang Y, Zhao HJ, Liu L, Guo FM, Lu XM, Qiu HB. Activation of canonical wnt pathway promotes differentiation of mouse bone marrow-derived MSCs into type II alveolar epithelial cells, confers resistance to oxidative stress, and promot — View Citation

Rojas M, Xu J, Woods CR, Mora AL, Spears W, Roman J, Brigham KL. Bone marrow-derived mesenchymal stem cells in repair of the injured lung. Am J Respir Cell Mol Biol. 2005 Aug;33(2):145-52. — View Citation

Wang G, Bunnell BA, Painter RG, Quiniones BC, Tom S, Lanson NA Jr, Spees JL, Bertucci D, Peister A, Weiss DJ, Valentine VG, Prockop DJ, Kolls JK. Adult stem cells from bone marrow stroma differentiate into airway epithelial cells: potential therapy for cy — View Citation

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Complete remission		3-6 months	Yes