Phase 2 N=41 Treatment

NMA Allogeneic Hematopoietic Cell Transplant in Hematologic Cancer/Disorders

Chronic Myeloproliferative Disorders · Leukemia · Lymphoma · Multiple Myeloma and Plasma Cell Neoplasm · Myelodysplastic Syndromes

Bottom Line

View on ClinicalTrials.gov: NCT00053989 ↗

Enrolled (actual)

Serious AEs

7.3%

Results posted

Feb 2020

Primary outcome: Primary: Day 100 TRM — 4 Participants

Study Design & Population

Study type: Interventional
Phase: Phase 2
Interventions: anti-thymocyte globulin (Biological); graft-versus-tumor induction therapy (Biological); sargramostim (Biological); therapeutic allogeneic lymphocytes (Biological); cyclophosphamide (Drug); fludarabine phosphate (Drug); methylprednisolone (Drug); mycophenolate mofetil (Drug); tacrolimus (Drug); allogeneic bone marrow transplantation (Procedure); peripheral blood stem cell transplantation (Procedure); umbilical cord blood transplantation (Procedure)
Age: Pediatric, Adult, Older Adult · 4+ yrs
Sex: All
Sponsor: Roswell Park Cancer Institute
Primary completion: Jul 2018

Outcome Measures

Outcome	Result	p-value
PRIMARY Day 100 TRM	4	—
PRIMARY Day 100 Best Response	22; 5; 4; 2; 8	—
SECONDARY PFS	27	—
SECONDARY OS	44	—
SECONDARY Acute GvHD	16	—

Summary

RATIONALE: Giving low doses of chemotherapy before a donor peripheral blood stem cell transplant helps stop the growth of cancer cells. It also stops the patient's immune system from rejecting the donor's stem cells. The donated stem cells may replace the patient's immune system and help destroy any remaining cancer cells (graft-versus-tumor effect). Giving an infusion of the donor's T cells (donor lymphocyte infusion) after the transplant may help increase this effect. Sometimes the transplanted cells from a donor can also make an immune response against the body's normal cells. Giving immunosuppressive therapy before or after the transplant may stop this from happening. PURPOSE: This phase II trial is studying how well chemotherapy followed by donor peripheral stem cell transplant works in treating patients with hematologic cancer or aplastic anemia.

Eligibility Criteria

DISEASE CHARACTERISTICS:

Diagnosis of aplastic anemia
Severe disease
Failed at least 1 course of standard immunosuppressive regimen with cyclosporine and anti-thymocyte globulin OR
Histologically confirmed hematologic malignancy including the following:
Acute leukemia
Any of the following types:
Acute myeloid leukemia (AML) with antecedent myelodysplastic syndromes
Secondary AML
AML with high-risk cytogenetic abnormalities
Acute lymphoblastic leukemia with high-risk cytogenetic abnormalities
Resistant or recurrent disease after combination chemotherapy with at least 1 standard regimen OR
In first remission at high risk of relapse
Chronic myelogenous leukemia
Chronic phase meeting at least 1 of the following criteria:
Failed imatinib mesylate
Failed interferon after at least 6 months of treatment with minimum of 21 million units of interferon per week
Unable to tolerate interferon
Accelerated phase (blasts less than 20%)
Myeloproliferative and myelodysplastic syndromes
Myelofibrosis (after splenectomy)
Refractory anemia
Refractory anemia with excess blasts
Chronic myelomonocytic leukemia
Lymphoproliferative disease
Chronic lymphocytic leukemia
Symptomatic disease after first-line chemotherapy
Low-grade non-Hodgkin's lymphoma (recurrent or persistent)
Symptomatic disease after first-line chemotherapy
Multiple myeloma
Progressive disease after autologous stem cell transplantation
Waldenstrom's macroglobulinemia
Failed 1 standard regimen
Non-Hodgkin's lymphoma meeting the following criteria:
Intermediate or high grade
Controlled and chemosensitive disease
First remission lymphoblastic or small non-cleaved cell lymphoma at high risk of relapse
Hodgkin's lymphoma
Relapsed and chemosensitive disease
Not eligible for standard myeloablative allogeneic stem cell transplantation
Availability of any of the following donor types:
Related donor matched at 5 or 6 HLA antigens (A, B, DR)
Unrelated donor fully matched by molecular analysis at A, B, DRB1, and DQB1 loci
Single antigen mismatch at C allowed
Cord blood that is 4, 5, or 6 match with recipient HLA antigens (A, B, DR) NOTE: No syngeneic donors permitted
No uncontrolled CNS disease (for hematologic malignancies) NOTE: A new classification scheme for adult non-Hodgkin's lymphoma has been adopted by PDQ. The terminology of "indolent" or "aggressive" lymphoma will replace the former terminology of "low", "intermediate", or "high" grade lymphoma. However, this protocol uses the former terminology.

PATIENT CHARACTERISTICS:

Age

4 to 75 (if related or unrelated donor peripheral blood or marrow transplantation)
4 to 60 (if unrelated cord blood transplantation)

Performance status

Karnofsky > 50%

Life expectancy

Not specified

Hematopoietic

Not specified

Hepatic

Bilirubin less than 3 times normal
Alkaline phosphatase less than 3 times normal
AST/ALT less than 3 times normal
No Child's class B or C liver failure

Renal

Creatinine clearance greater than 40 mL/min

Cardiovascular

Cardiac ventricular ejection fraction at least 35% by MUGA
No cardiovascular disease

Pulmonary

DLCO at least 40% of predicted, corrected for hemoglobin and/or alveolar ventilation

Other

Not pregnant or nursing
Negative pregnancy test
Fertile patients must use effective contraception
HIV antibody negative
No uncontrolled diabetes mellitus
No active serious infection
No other disease that would preclude study therapy
No other concurrent malignancy except non-melanoma skin cancer
No concurrent serious psychiatric illness

PRIOR CONCURRENT THERAPY:

Biologic therapy

See Disease Characteristics
patients may have received a prior autologous blood or marrow transplantation (BMT)
At least 6 months since prior allogeneic BMT

Chemotherapy

See Disease Characteristics
At least 2 weeks since prior chemotherapy, radiation or surgery

Endocrine therapy

Not

View full record on ClinicalTrials.gov →

Data sourced from ClinicalTrials.gov (NCT00053989). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.