Efficacy of Bone Marrow Mesenchymal Stem Cell in Pulmonary Hemosiderosis

NCT02985346 | Unknown Early Phase 1

• 1 other identifier: WTan
• Study Type: interventional
• Target: 100
• Locations: 0 countries
• Sites: N/A

Brief 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.

Conditions

Idiopathic Pulmonary Hemosiderosis

Keywords

Idiopathic pulmonary hemosiderosis; Bone marrow mesenchymal stem cells

Outcome Measures

Primary Outcomes (1)

• Complete remission

  3-6 months

Study Arms (2)

BMSC group

EXPERIMENTAL

Patients received Bone marrow mesenchymal stem cells (BMSC) plus standard treatment

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

Control group

ACTIVE COMPARATOR

Patients received standard treatment

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

Interventions

Bone marrow mesenchymal stem cells in treatment of Idiopathic pulmonary hemosiderosis BIOLOGICAL

BMSC group; Control group

Eligibility Criteria

• Age: 1 Month - 18 Years
• Sex: all
• Healthy Volunteers: Yes
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

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

You may not qualify if:

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

Sponsors & Collaborators

• Sun Yat-sen University: lead

Related Publications (10)

• Byrd RB, Gracey DR. Immunosuppressive treatment of idiopathic pulmonary hemosiderosis. JAMA. 1973 Oct 22;226(4):458-9. No abstract available.

  PMID: 4800237 BACKGROUND

• 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.

  PMID: 11665500 BACKGROUND

• 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. doi: 10.1097/00007890-200212150-00027.

  PMID: 12490803 BACKGROUND

• 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. doi: 10.1097/MPH.0b013e3180950372. No abstract available.

  PMID: 17609633 BACKGROUND

• 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. doi: 10.1165/rcmb.2004-0330OC. Epub 2005 May 12.

  PMID: 15891110 RESULT

• 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 cystic fibrosis. Proc Natl Acad Sci U S A. 2005 Jan 4;102(1):186-91. doi: 10.1073/pnas.0406266102. Epub 2004 Dec 22.

  PMID: 15615854 RESULT

• 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/pnas.0907996106. Epub 2009 Aug 31.

  PMID: 19721001 RESULT

• 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.

  PMID: 21128750 RESULT

• 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. doi: 10.1089/scd.2011.0105. Epub 2011 Jul 6.

  PMID: 21585237 RESULT

• 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 promotes their migration to injured lung tissue in vitro. J Cell Physiol. 2013 Jun;228(6):1270-83. doi: 10.1002/jcp.24282.

  PMID: 23154940 RESULT

MeSH Terms

Conditions

Hemosiderosis, Pulmonary

Condition Hierarchy (Ancestors)

Lung Diseases; Respiratory Tract Diseases; Hemosiderosis; Iron Overload; Iron Metabolism Disorders; Metabolic Diseases; Nutritional and Metabolic Diseases

Study Design

• Study Type: interventional
• Phase: early phase 1
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Sun Yat-sen Memorial Hospital

Study Record Dates

• First Submitted: December 3, 2016
• First Posted: December 7, 2016
• Study Start: January 1, 2017
• Primary Completion: December 1, 2020
• Last Updated: December 7, 2016

Record last verified: 2016-12