Genome-Wide Gene Expression Profiling of Patients With ITP Receiving Thrombopoietin Mimetics

NCT01727999 | Completed

• 1 other identifier: SPO 105489
• Study Type: observational
• Target: 75
• Locations: 1 country
• Sites: 2

Brief Summary

Introduction: Ineffective platelet production has been proven to play a role in the etiology of Immune Thrombocytopenia (ITP) in addition to increased platelet destruction. The second-generation thrombopoietin (TPO) mimetics have shown good efficacy in boosting platelet counts in the great majority of patients with chronic ITP in several clinical trials.1, 2 Nevertheless, about 20% of patients with ITP fail to respond to the TPO mimetic treatment. Those treatment-resistant patients are un-characterized and the reasons for the lack of response have not been studied. The identification of predictive blood biomarkers of patients' response to treatment will be useful in reducing both cost and potential side effects; and it will be of equal importance and interest to investigate the molecular mechanisms underlying the patients' heterogeneous responses to TPO mimetic treatment. Specific Aims:

1. 1.To identify blood classifier genes which correlate with patients' response to TPO mimetic treatment.
2. 2.To compare the blood gene expression changes in responders and non-responders after TPO mimetic treatment and explore the possible molecular mechanisms accounting for the non-responsiveness to the treatment.

Conditions

Immune Thrombocytopenia

Keywords

TPO response; ITP; immune thrombocytopenia

Outcome Measures

Primary Outcomes (1)

• 1. To identify blood classifier genes which correlate with patients' response to TPO mimetic treatment.

  2 years

Study Arms (2)

TPO responder

Patients with therapeutic response to TPO

TPO non-responder

Patients not responding to TPO agonists

Eligibility Criteria

• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

Patients with ITP receiving TPO agonists

You may qualify if:

• clinical diagnosis of ITP TPO treatment

You may not qualify if:

• thrombocytopenia not due to ITP

Sponsors & Collaborators

• Stanford University: lead
• Weill Medical College of Cornell University: collaborator

Study Sites (2)

Stanford University

Stanford, California, 94305, United States

Location

Weill Medical College, Cornell University

New York, New York, 10065, United States

Location

Related Publications (4)

• Bussel JB, Cheng G, Saleh MN, Psaila B, Kovaleva L, Meddeb B, Kloczko J, Hassani H, Mayer B, Stone NL, Arning M, Provan D, Jenkins JM. Eltrombopag for the treatment of chronic idiopathic thrombocytopenic purpura. N Engl J Med. 2007 Nov 29;357(22):2237-47. doi: 10.1056/NEJMoa073275.

  PMID: 18046028 BACKGROUND

• Zhang B, Lo C, Shen L, Sood R, Jones C, Cusmano-Ozog K, Park-Snyder S, Wong W, Jeng M, Cowan T, Engleman EG, Zehnder JL. The role of vanin-1 and oxidative stress-related pathways in distinguishing acute and chronic pediatric ITP. Blood. 2011 Apr 28;117(17):4569-79. doi: 10.1182/blood-2010-09-304931. Epub 2011 Feb 16.

  PMID: 21325602 BACKGROUND

• Rodeghiero F, Stasi R, Gernsheimer T, Michel M, Provan D, Arnold DM, Bussel JB, Cines DB, Chong BH, Cooper N, Godeau B, Lechner K, Mazzucconi MG, McMillan R, Sanz MA, Imbach P, Blanchette V, Kuhne T, Ruggeri M, George JN. Standardization of terminology, definitions and outcome criteria in immune thrombocytopenic purpura of adults and children: report from an international working group. Blood. 2009 Mar 12;113(11):2386-93. doi: 10.1182/blood-2008-07-162503. Epub 2008 Nov 12.

  PMID: 19005182 BACKGROUND

• Kuter DJ, Bussel JB, Lyons RM, Pullarkat V, Gernsheimer TB, Senecal FM, Aledort LM, George JN, Kessler CM, Sanz MA, Liebman HA, Slovick FT, de Wolf JT, Bourgeois E, Guthrie TH Jr, Newland A, Wasser JS, Hamburg SI, Grande C, Lefrere F, Lichtin AE, Tarantino MD, Terebelo HR, Viallard JF, Cuevas FJ, Go RS, Henry DH, Redner RL, Rice L, Schipperus MR, Guo DM, Nichol JL. Efficacy of romiplostim in patients with chronic immune thrombocytopenic purpura: a double-blind randomised controlled trial. Lancet. 2008 Feb 2;371(9610):395-403. doi: 10.1016/S0140-6736(08)60203-2.

  PMID: 18242413 BACKGROUND

Biospecimen

Retention: SAMPLES WITH DNA

RNA and DNA will be retained

MeSH Terms

Conditions

Purpura, Thrombocytopenic, Idiopathic

Condition Hierarchy (Ancestors)

Purpura, Thrombocytopenic; Purpura; Blood Coagulation Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Thrombotic Microangiopathies; Thrombocytopenia; Blood Platelet Disorders; Cytopenia; Hemorrhagic Disorders; Autoimmune Diseases; Immune System Diseases; Hemorrhage; Pathologic Processes; Pathological Conditions, Signs and Symptoms; Skin Manifestations; Signs and Symptoms

Study Officials

• James L Zehnder, MD

  Stanford University

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Professor

Study Record Dates

• First Submitted: November 12, 2012
• First Posted: November 16, 2012
• Study Start: July 1, 2012
• Primary Completion: February 1, 2017
• Study Completion: February 1, 2017
• Last Updated: April 26, 2017

Record last verified: 2017-04

Data Sharing

• IPD Sharing: Will not share

Locations

US (2)