Protocol Number: 97-H-0154

Title:

High-Dose Cyclophosphamide with CD34+ Selected Autologous Hematopoietic Cell Support for Treatment of Refractory Chronic Autoimmune Thrombocytopenia

Number:

97-H-0154

Summary:

Platelets are particles found along with red and white blood cells in the blood that play a role in the process of blood clotting. Disorders affecting the platelets can lower the amount of platelets in the blood and put patients at risk of bleeding. The condition of low platelets is referred to as thrombocytopenia.

Thrombocytopenia can be associated with a variety of diseases including cancer, leukemia, tuberculosis, or as a result of an autoimmune reaction. Autoimmune reactions are disorders in which the normal immune system begins attacking itself. Autoimmune thrombocytopenia (AITP) is a disorder of low blood platelet counts in which platelets are destroyed by antibodies produced by the immune system.

Unfortunately, many patients with AITP do not respond to standard treatments for thrombocytopenia. Cyclophosphamide is a drug that works to suppress the activity of the immune system. Researchers believe that combining this drug with transplanted rescued blood stem cells may provide effective treatment for AITP.

The purpose of this study is to explore the affordability and safety of this therapy for the treatment of AITP. The effectiveness of the therapy will be measured by the number of patients whose platelet levels rise greater than 100,000/m3.

If this treatment approach appears affordable, this study will form the basis for a larger study to compare alternate treatment approaches.

Sponsoring Institute:

National Heart, Lung and Blood Institute (NHLBI)

Recruitment Detail

Type: Participants currently recruited/enrolled

Gender: Male & Female

Referral Letter Required: No

Population Exclusion(s): None

Eligibility Criteria:

INCLUSION CRITERIA:

Male or female, ages 18-65 years old.

Refractory severe chronic autoimmune thrombocytopenia, with or without autoimmune hemolytic anemia (Evan's syndrome), with all the following:

a. Platelet count frequently below 20,000/mm(3) despite active

treatment for a period of greater than 6 months.

b. Normal or increased megakaryocytes on bone marrow

aspirate/bx.

c. No plausible alternative etiology such as drug-mediated

thrombocytopenia, marrow failure syndrome or thrombocytopenia

related to viral or bacterial infection.

d. Failure of treatment with:

i. conventional-dose steroids (e.g., prednisone or dosage of 40

mg/day or equivalent, followed by dosage taper) for at least 3

months.

ii. intravenous immunoglobulin.

iii. splenectomy.

e. Episodic bleeding requiring transfusions or ecchymoses interfering

with ordinary daily activities.

EXCLUSION CRITERIA:

ECOG performance status greater than 1.

Cardiopulmonary disease including:

a. History of coronary artery disease, angina pectoris or congestive heart failure.

b. LV ejection fraction less than 40 percent by 2D echocardiogram.

Renal disease, serum creatinine greater than 2.5 mg/dL or creatinine clearance less than 30 mL/min.

Significant hepatic dysfunction, bilirubin greater than 2 mg/dL or transaminases greater than 2 times UNL.

Uncorrected coagulopathy.

Bone marrow aplasia (cellularity less than 10 percent), single or multilineage hematopoietic failure, myelodysplastic syndrome, or extensive marrow fibrosis.

History or active diagnosis of malignancy (except treated non-melanoma skin cancer or cevical carcinoma in situ).

HIV positive.

Pregnancy or lactation, unwillingness to practice adequate birth control in the peritransplant period.

Psychiatric illness or mental incapacity to understand and give informed consent.

Other medical illness or condition which, in the opinion of the Investigators, may contraindicate participation in this study due to patients' risk or compromise of study integrity.

Special Instructions:

Currently Not Provided

Keywords:

Immunosuppression

Thrombocytopenia

Autoimmune Disease

Recruitment Keyword(s):

Evan's Syndrome

Autoimmune Hemolytic Anemia

Episodic Bleeding

Condition(s):

Autoimmune Disease

Autoimmune Hemolytic Anemia

Thrombocytopenia

Investigational Drug(s):

None

Investigational Device(s):

Isolex 300i

Intervention(s):

Device: Isolex 300i

Supporting Site:

National Heart, Lung and Blood Institute

Contact(s):

Patient Recruitment and Public Liaison Office
Building 61
10 Cloister Court
Bethesda, Maryland 20892-4754
Toll Free: 1-800-411-1222
TTY: 301-594-9774 (local),1-866-411-1010 (toll free)
Fax: 301-480-9793

Electronic Mail:prpl@mail.cc.nih.gov

Citation(s):

Semple JW, Freedman J. Abnormal cellular immune mechanisms associated with autoimmune thrombocytopenia. Transfus MedRev. 1995 Oct;9(4):327-38. Review. No abstract available.

Karpatkin S. Autoimmune (idiopathic) thrombocytopenic purpura. Lancet. 1997 May 24;349(9064):1531-6. Review. No abstract available.

George JN, et al., Chronic idiopathic thrombocytopenic purpura. N Engl J Med. 1994 Nov 3;331(18):1207-11. Review. No abstract available.

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