Targeted Therapies

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ABSTRACT HIGHLIGHTS OF THE 47TH ANNUAL MEETING OF THE AMERICAN SOCIETY OF HEMATOLOGY

DECEMBER 10–13, 2005

 

NON-HODGKIN'S LYMPHOMA (NHL)

Number of rituximab (R)-CHOP-14 cycles for elderly patients.  Pfreundschuh and colleagues conducted a multicenter study (RICOVER-60 study) in which 1,330 elderly patients (61–80 years of age) with CD20+ diffuse large B-cell lymphoma (DLBCL; stage I–IV) were randomized to receive 6 or 8 cycles of CHOP-14 (CHOP given every 2 weeks instead of every 3 weeks) with or without rituximab (R) (given on study days 1, 15, 29, 43, 57, 71, 85, and 99). The current data reported were from a planned interim analysis performed on 828 evaluable patients. The primary endpoint was freedom from treatment failure (FFTF), with events defined as disease progression or relapse, need for additional therapy, failure to achieve a complete response, or death. The FFTF rate was highly significantly better for R-CHOP-14 than for CHOP-14 alone, such that the preplanned criterion (O'Brien-Fleming boundary) for stopping the study was met. In addition, after a median follow-up of 26 months, no difference was observed between 6 or 8 cycles of R-CHOP-14 in the FFTF rate. These results suggest that 6 cycles of R-CHOP-14 should be a standard initial therapy for elderly patients with DLBCL.  (Abstract 13)

Rituximab-containing induction and maintenance therapies in follicular lymphoma (FL). Hochster and colleagues randomized 304 patients with stage III–IV FL (n=237) or small lymphocytic lymphoma who had responded to 6 to 8 cycles of CVP chemotherapy to receive 2 years’ maintenance R or observation. After a median follow-up of 3 years, progression-free survival (PFS) was longer in the R arm than in the observation arm. In the subgroup with FL, both PFS and overall survival (OS) were significantly longer in the R-treated patients than in the observed patients. ¨ Solal-Celigny and coworkers updated a phase III study (42 months of follow-up) in which 321 patients with previously untreated stage III–IV FL were randomized to receive induction therapy with 8 cycles of either CVP or R-CVP. Superior response rates and median time to progression (TTP) were achieved with R-CVP vs. CVP alone. The 3-year OS rate was 81% in the CVP arm and 89% in the R-CVP arm. ¨ Van Oers and associates reported the results of a second preplanned interim analysis of a phase III study in which data were available for 461 patients with relapsed/resistant FL. Patients were randomized to receive 6 cycles of CHOP or R-CHOP; responding patients were randomized to receive R maintenance or observation. Partial response (PR) rates were similar in both induction arms, but the percentage of CRs was greater in the R-CHOP arm (30.4% vs. 18.1%). While maintenance R had no impact on OS, the median PFS was greater in the R than in the observation arm (38 vs. 15 months). Thus, R maintenance may confer PFS and OS advantages.  However, questions remain as to which patient subsets benefit the most and which induction regimen is best.  (Abstracts 349, 350, 353)

Enzastaurin, a protein kinase (PK-) inhibitor.  Gene expression profiling has identified PK- to be a therapeutic target for DLBCL. These findings led Robertson and coworkers to investigate the use of enzastaurin, an orally administered potent inhibitor of PK-, in a phase II study of patients with relapsed DLBCL (N=55). The patients were given enzastaurin 500 to 525 mg once daily. Enzastaurin was well tolerated. One patient achieved a CR, and 8 patients had stable disease. These results suggest that enzastaurin is active in relapsed DLBCL. Further studies are warranted.  (Abstract 934)

HGS-ETR1.  HGS-ETR1 (TRM-1, mapatumumab) is a human monoclonal antibody that is agonistic to the death receptor TRAIL-R1 (D4) and has demonstrated preclinical evidence of antitumor activity. Younes and colleagues report preliminary data on the use of HGS-ETR1 in 40 patients with relapsed or refractory NHL. Patients received 3 mg/kg (n=8) or 10 mg/kg (n=32) every 21 days for up to 6 cycles. Three patients achieved a response (1 CR and 2 PRs), and 12 patients (30%) had stable disease. No patient discontinued treatment because of adverse events. These early data indicate that HGS-ETR1 is active in relapsed/refractory NHL, warranting studies in combination with agents active in lymphoma.  (Abstract 489)

Anti-CD40 immunotherapy.  SGN-40 (Seattle Genetics, Inc.) is a humanized anti-CD40 antibody that is currently undergoing phase I testing (Advani et al.) in patients with NHL. The rationale for evaluating SGN-40 in NHL is that CD40 can regulate apoptosis in transformed cells and is expressed by most B-cell malignancies. This novel agent is administered intravenously once per week for 4 weeks. The maximum tolerated dose (MTD) had not been reached, but preliminary evidence of antitumor activity was noted at the 2 mg/kg dose level. Several patients have experienced an increase in plasma TNF-alpha and IL-6 levels following the first infusion (but not after subsequent infusions). No grade 4 toxicities were reported, although 1 patient developed grade 3 unilateral conjunctivitis and loss of vision that resolved within 6 weeks. This trial is ongoing.  (Abstract 1504)

Phase II study of thrombospondin-mimetic peptide (ABT-510).  ABT-510 (Abbott) is a nonapeptide that mimics the anti-angiogenic activity of thrombospondin-1. Levine and coworkers are conducting a study in which 67 patients with relapsed or refractory lymphoma have been randomized to receive ABT-510 at a dose of 10 mg bid or 100 mg bid by subcutaneous injection.  Preliminary data were available for 56 patients. In the 100 mg bid group, 3 PRs have been achieved. No responses have been achieved in the 10 mg bid group; in fact, 15 of 15 patients in this group developed disease progression within 12 months. Thus, the 10 mg bid arm was discontinued. The most frequent adverse events have been asthenia (39%), injection site reaction (39%), diarrhea (24%), and anorexia (20%). Further study of this novel agent is warranted.  (Abstract 1493)

LEUKEMIA

New agents for imatinib-resistant BCR-ABL–expressing leukemias.  Multiple studies of experimental agents aimed at the treatment of imatinib-resistant BCR-ABL–expressing leukemias were reported. One of these agents, dasatinib (Bristol-Myers Squibb) is an oral inhibitor of BCR-ABL and SRC kinases. Current studies of dasatinib include 4 separate trials (the START studies) investigating its use in patients who have imatinib-resistant/intolerant chronic-phase chronic myeloid leukemia (CML), accelerated-phase CML, CML in blast crisis, and CML in lymphoid blast crisis or Philadelphia chromosome-positive (Ph+) acute lymphoblastic leukemia (ALL). Preliminary data obtained from the first 28 to 35 patients entered into each of these studies show that major hematologic responses and cytogenetic responses have been achieved in a substantial proportion of patients in each study. The most important dasatinib-associated toxicity has been grade 4 neutropenia and/or thrombocytopenia. Promising data were also presented on a second new oral agent, AMN107. This aminopyrimidine ATP-competitive inhibitor of BCR-ABL is 50- to 60-fold more potent than imatinib against BCR-ABL–expressing cell lines.  A phase I study of 119 patients with Ph+ leukemias demonstrated that AMN107 was active in patients both with and without BCR-ABL mutations. The dose selected for further study was 400 mg bid; neutropenia and hyperbilirubinemia were identified as dose-limiting toxicities.  (Abstracts 37, 39, 40, 41, 42)

 

Lenalidomide treatment of relapsed or refractory chronic lymphocytic leukemia (CLL).  Chanan-Khan and coworkers reported preliminary results from a phase II study evaluating single-agent lenalidomide (an oral immunomodulatory agent derived from thalidomide) as therapy for patients with relapsed or refractory CLL. Among 17 evaluable patients, 11 patients (64.6%) achieved a response (2 CRs and 9 PRs). Another 5 patients had stable disease. A total of 29 patients were evaluable for safety. A flare reaction, manifested by tender swelling of lymph nodes and/or rash, was observed in almost all patients. Grade 3 or 4 hematologic toxicity occurred in 7 patients, and febrile neutropenia developed in 3 patients. Tumor lysis syndrome was observed in 2 patients. These findings indicate that lenalidomide is an active agent for CLL treatment, and further studies are warranted.  (Abstract 447)

 

Phase I/II study of HuMax-CD20 in CLL.  HuMax-CD20 is a human monoclonal antibody that targets a novel epitope of CD20 on B cells. In a SCID mouse model, HuMax-CD20 has been shown to inhibit the proliferation of engrafted B-cell tumors more efficiently than rituximab. Coiffier and colleagues studied 4 weekly intravenous infusions of HuMax-CD20 in relapsed or refractory CLL patients. Doses up to 2000 mg have been evaluated, and the MTD has not been reached. The following 5 HuMax-CD20–related serious adverse events have been reported: hepatic cytolysis, herpes zoster, neutropenia (2), and death due to pneumonia.  Marked reductions in CD19+CD5+ cell counts and improvements in lymphadenopathy have been observed. These data suggest that HuMax-CD20 is a potentially active agent against relapsed or refractory CLL.  (Abstract 448)

 

MULTIPLE MYELOMA

Combination lenalidomide plus dexamethasone.  Dimopoulos and colleagues conducted a double-blind trial in 351 relapsed or refractory multiple myeloma patients randomized to oral lenalidomide (25 mg QD for 3 weeks every 4 weeks) plus high-dose dexamethasone or placebo plus high-dose dexamethasone. Response rates were higher in the combination arm than in the placebo/dexamethasone arm (58% vs. 22%). With study duration of 18 months, median TTP was 13.3 months in the combination group and 5.1 months in the placebo/dexamethasone group. TTP differences surpassed the prespecified O'Brien-Fleming boundary for superiority. Thromboembolic events occurred in 8.5% of lenalidomide/dexamethasone patients vs. 4.5% of control patients.  Grade 3 or 4 neutropenia occurred in 16.5% of the combination therapy arm and 1.2% of the control arm. Otherwise, the safety profiles of the 2 treatment groups were similar. Combination lenalidomide and dexamethasone is effective for relapsed/refractory multiple myeloma; however, prophylactic antithrombotic therapy should be considered.  (Abstract 6)

 

BREAST CANCER (reported from the 28th Annual San Antonio Breast Cancer Symposium, December 8–11, 2005)

Trastuzumab-based regimens.  A total of 3,222 patients with HER2-amplified breast cancer with lymph node involvement or high-risk lymph node–negative disease were enrolled into the BCIRG 006 study (Slamon et al) and randomized to receive (1) 3 months of doxorubicin (A) and cyclophosphamide (C) followed by 3 months of docetaxel (T) (AC-T); (2) 3 months of AC followed by 3 months of T with 1 year of trastuzumab (H) (AC-TH); or (3) 18 weeks of T plus carboplatin with 1 year of H (TCH). The report presented results from the first preplanned interim analysis conducted after 322 events (relapse, second primary breast cancer, or death) had occurred (median follow-up, 23 months). Disease-free survival (DFS) was significantly longer in both of the H-containing regimens than in the AC-T regimen. There was no difference in DFS between the 2 H-containing regimens. Symptomatic cardiac events occurred in 1.2%, 2.3%, and 1.2% of patients in the AC-T, AC-TH, and TCH treatment groups, respectively. LVEF declines of >15% occurred in 0.6%, 2.4%, and 0.4% of patients in the respective treatment groups. These results confirm the benefit of adding H to effective breast cancer chemotherapy. The data also demonstrate that fewer cardiac events occur when H is given without prior A therapy.  Further follow-up is needed to compare the efficacy of TCH with AC-TH. (Abstract 1)

 

Oral ibandronate reduces markers of bone turnover.  Ibandronate is a bisphosphonate that is available in both intravenous and oral formulations. Lichinitser et al conducted a study in 256 breast cancer patients with ≥1 osteolytic lesions who were randomized to receive 12 weeks of oral ibandronate 50 mg daily or intravenous zoledronic acid 4 mg every 4 weeks. Comparable reductions in markers of bone turnover occurred in the 2 treatment groups. Studies that assess skeletal-related events are needed to determine whether the clinical benefit of oral ibandronate therapy is comparable to that of intravenous zoledronic acid therapy. (Abstract 6033)

 

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