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Protocol Details

Non-Myeloablative Allogeneic Peripheral Blood Mobilized Hematopoietic Precursor Cell Transplantation for Hematologic Malignancies in High Risk Patients and in Patients with Debilitating Hematologic Diseases

This study is NOT currently recruiting participants.

Summary | Eligibility | Citations | Contacts




Sponsoring Institute

National Heart, Lung and Blood Institute (NHLBI)

Recruitment Detail

Type: Completed Study; data analyses ongoing
Gender: Male & Female
Min Age: 2 Years
Max Age: 80 Years

Referral Letter Required


Population Exclusion(s)

Pregnant Women


Graft vs. Host Disease;
Peripheral Blood Stem Cells;
Non-Myeloablative Bone Marrow Transplantation;
graft versus leukemia

Recruitment Keyword(s)



Myeloproliferative Disorders;
Acute Myelogenous Leukemia;
Chronic Myelogenous Leukemia;
Myelodysplastic Syndrome;
Acute Lymphoblastic Leukemia

Investigational Drug(s)


Investigational Device(s)



Drug: G-CSF
Procedure/Surgery: T-cell replete PBPC allograft
Drug: Methotrexate
Drug: Cyclosporine

Supporting Site

National Heart, Lung, and Blood Institute

The are a variety of cancerous diseases of the blood and bone marrow that can be potentially cured by bone marrow transplantation (BMT). Diseases like leukemia, lymphoma, and multiple myeloma are among the conditions that can be treated with BMT.

Some patients with these diseases can be treated with medical chemotherapy alone. However, patients who relapse following chemotherapy are usually not curable with additional chemotherapy treatments. The only option known to provide a potential cure if this occurs is BMT.

Allogenic transplants are cells collected from relatives of the patient. The transplant requires additional high intensity chemotherapy and radiation in order to destroy cancerous cells. In the process, many normal bone marrow cells are also destroyed. This is the reason for transplanting stem cells. The stem cells help to build new functioning bone marrow, red cells, white cells, and platelets. In addition, the immune cells from the donor are implanted into the recipient s body and help to fight off infection and kill remaining cancerous cells.

Unfortunately, the powerful doses of chemotherapy and radiation therapy associated with allogenic BMT have toxic side effects and often make BMTs too dangerous to attempt in many patients.

In order to reduce the complications of BMT, and make it a safer available option for patients with cancers of the blood and bone marrow, researchers have developed a new approach to the BMT.

In this study researchers plan to use stem cells collected from the blood stream of patient s relatives rather than from the bone marrow (blood progenitor/stem cell transplant). In addition, researchers plan to use low doses of chemotherapy and no radiation therapy to reduce side effects. The majority of the cancer killing effect will be the responsibility of the stem cell transplant rather than the chemotherapy.

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Group A: Subjects at high risk for transplant related complications and mortality as defined below:

Ages 10 to 75 (both inclusive) with a history of one of the following:

-Treatment with dose intensive chemotherapy and/or radiotherapy

-Previous history of allo/auto transplant

-History of multiple myeloma or extramedullary plasmacytoma

-Chronic disease or co-morbid medical condition including subjects with symptoms or signs of significant pulmonary disease, hepatic disease, kidney disease, cardiac disease or disease of other organ systems which would result in increased risk of morbidity or death from a standard myeloablative transplant.

Diseases to be included:

-CML chronic phase

-Acute lymphoblastic leukemia (ALL), all subjects in complete or partial remission.

-AML: AML in first complete or partial remission Exceptions: AML with good risk karyotypes: AML M3 t(15:17), AML M4Eo (inv. 16), AML t(8;21). All AML in second or subsequent complete remission.

-MDS: refractory anemia with excess blasts (RAEB), or chronic myelomonocyte leukemia (CMML).

-Myeloproliferative diseases associated with either cytopenia or uncontrolled proliferation.

-CLL or small lymphocytic lymphoma (SLL) with bulky or progressive disease despite prior treatment with chemotherapy which includes purine analogs.


A) Intermediate or high grade relapsed or progressive despite treatment with standard therapy ineligible for autologous PBSC transplant.

B) NHL intermediate or high grade relapsing despite prior autologous transplant.

C) Low grade follicular or small lymphocytic lymphoma (1) high risk patients who have relapsed following conventional chemotherapy, (2) relapsed following autologous marrow or PBSC transplant, or (3) chemo resistant disease.

D) Mantle cell lymphoma

E) NHL intermediate or high grade with concurrent BCL2 and MYC translocations who are at high risk for relapsed and who have low survival with conventional chemotherapy.

-HD, relapsed after prior autologous transplant or after 2 or more combination chemotherapy regimens and ineligible for autologous PBSC transplant.

-EBV driven lymphoproliferative disorders progressing despite standard therapies.

-MM: MM subjects must be between the ages of 8 and 65 (both inclusive)

-Mycosis fungoides, which has been shown to be amenable to allogeneic stem cell transplants.

Group B: (Closed to enrollment Oct 2010) Subjects with hematologic diseases associated with reasonable longevity, shown to be curable by allogeneic BMT but where concern for a high procedural mortality with conventional BMT may delay or prevent such treatment.

Ages 8 to 80 (both inclusive) with a history of one of the following

-PNH associated with either life-threatening thrombosis, cytopenia, transfusion dependence or recurrent and debilitating hemolytic crisis.

-Aplastic anemia or PRCA (acquired or congenital) in subjects associated with transfusion dependence and/or neutropenia who are not candidates for or who have failed immunosuppressive therapy

-RA or RARS MDS subjects who have associated transfusion dependence and/or neutropenia.

Ability to comprehend the investigational nature of the study and provide informed consent. The procedure will be explained to subjects age 8-17 years with formal consent being obtained from parents or legal guardian.

Availability of HLA identical or single HLA locus mismatched family donor


HLA identical or single HLA mismatched family donor

Age greater than or equal to 2 up to 80 years old

Weight greater than or equal to 18 kg

Ability of donor or guardian of donor to comprehend the investigational nature of the study and provide informed consent.

EXCLUSION CRITERIA - Recipient - any of the following:

Pregnant or lactating

Group A: age less than 10 or greater than 75 (multiple myeloma age less than 8 or greater than 65);

Group B: Age less than 8 or greater than 80 years.

ECOG performance status of 3 or more (See NIH Bone and Marrow Consortium Supportive Care Guidelines for Allogeneic Hematopoietic Stem Cell Transplant Recipients -

Psychiatric disorder or mental deficiency severe as to make compliance with the BMT treatment unlikely and making informed consent impossible

Major anticipated illness or organ failure incompatible with survival from PBSC transplant

Diffusion capacity of carbon monoxide (DLCO) less than 40% predicted.

Left ventricular ejection fraction: less than 30%.

Serum creatinine greater than 2.5 mg/dl or creatinine clearance less than 50 cc/min by 24 hr urine collection

Serum bilirubin greater than 4 mg/dl, transaminases greater than 5x upper limit of normal,

Other malignant diseases liable to relapse or progress within 5 years.

EXCLUSION CRITERIA - Donor - any of the following:

Pregnant or lactating

Donor unfit to receive G-CSF and undergo apheresis (uncontrolled hypertension, history of congestive heart failure or unstable angina, thrombocytopenia)

HIV positive donor. Donors who are positive for hepatitis B (HBV), hepatitis C (HCV) or human T-cell lymphotropic virus (HTLV I/II) will be used at the discretion of the investigator following counseling and approval from the recipient

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Not Provided

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Principal Investigator

Referral Contact

For more information:

Richard W. Childs, M.D.
National Heart, Lung and Blood Institute (NHLBI)
BG 10-CRC RM 3-5330
(301) 451-7128

Kristen Gunn E. Wood, R.N.
National Heart, Lung and Blood Institute (NHLBI)
National Institutes of Health
Building 10
Room 44350
10 Center Drive
Bethesda, Maryland 20892
(301) 827-2977

Office of Patient Recruitment
National Institutes of Health Clinical Center (CC)
Building 61, 10 Cloister Court
Bethesda, Maryland 20892
Toll Free: 1-800-411-1222
Local Phone: 301-451-4383
TTY: TTY Users Dial 7-1-1

Clinical Trials Number:


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