A comparative effectiveness study of lipegfilgrastim in multiple myeloma patients after high dose melphalan and autologous stem cell transplant.

G-CSF administration after high-dose chemotherapy and autologous stem cell transplantation (ASCT) has been shown to expedite neutrophil recovery. Several studies comparing filgrastim and pegfilgrastim in the post-ASCT setting concluded that the two are at least equally effective. Lipegfilgrastim (LIP) is a new long-acting, once-per-cycle G-CSF. This multicentric, prospective study aimed to describe the use of LIP in multiple myeloma patients receiving high-dose melphalan and autologous stem cell transplantation (ASCT) and compare LIP with historic controls of patients who received short-acting agent (filgrastim [FIL]). Overall, 125 patients with a median age of 60 years received G-CSF after ASCT (80 patients LIP on day 1 post-ASCT and 45 patients FIL on day 5 post-ASCT). The median duration of grade 4 neutropenia (absolute neutrophil count [ANC] < 0.5 × 10 [9]/L) was 5 days in both LIP and FIL groups, whereas the median number of days to reach ANC ≥ 0.5 × 10 [9]/L was 10% lower in the LIP than in the FIL group (10 vs 11 days), respectively. Male sex was significantly associated with a faster ANC ≥ 0.5 × 10 [9] L response (p = 0.015). The incidence of FN was significantly lower in the LIP than in the FIL group (29% vs 49%, respectively, p = 0.024). The days to discharge after ASCT infusion were greater in patients with FN (p < 0.001). The study indicates that LIP had a shorter time to ANC recovery and is more effective than FIL for the prevention of FN in the ASCT setting.

A Comparative Assessment of Quality of Life in Patients with Multiple Myeloma Undergoing Autologous Stem Cell Transplantation Through an Outpatient and Inpatient Model.

Outpatient autologous stem cell transplantation (ASCT) has proven to be feasible in terms of physical morbidity and mortality outcomes, but little data exist on the impact of this procedure on quality of life (QoL). The purpose of this prospective, observational, longitudinal cohort study was to compare the effects of inpatient (n = 76) and outpatient (n = 64) modes of care on QoL in patients with multiple myeloma who underwent ASCT. Patients were treated according to their preference for the inpatient or outpatient model. QoL was assessed using the Functional Assessment of Cancer Therapy-Bone Marrow Transplantation (FACT-BMT) at baseline (7 days before ASCT; T1) and at days +7 (T2) and +30 (T3) after ASCT. Overall, inpatients achieved higher mean values at each time point (86.05 ± 15.54 at T1, 89.23 ± 19.19 at T2, and 87.96 ± 13.6 at T3) compared with outpatients (85.62 ± 14.51 at T1, 87.42 ± 23.41 at T2, and 83.98 ± 20.2 at T3), although the differences did not reach statistical significance. Inpatients showed higher mean scores than outpatients in physical well-being (7.67 ± 5.7, 15.44 ± 6.34, and 12.96 ± 6.03, respectively, versus 5.89 ± 4.33, 13.92 ± 7.05, and 8.84 ± 6.33, respectively; P < .05). Mean scores on social/family well-being were significantly higher in the outpatient group compared with the inpatient group (22.93 ± 13.29, 21.14 ± 5.31, and 21.64 ± 4.58, respectively, versus 20.59 ± 3.79, 19.52 ± 5.12, and 20.01 ± 3.97, respectively; P = .003). There were no significant between-group differences with respect to functional well-being and emotional status. Among adults at a single institution undergoing ASCT for MM, the use of outpatient care compared with standard transplantation care did not result in improved QoL during transplantation. Further research is needed for replication and to assess longer-term outcomes and implications.

Mobilization of hematopoietic stem cells with lenograstim in healthy donors: efficacy and safety analysis according to donor age.

We reviewed and analyzed safety and efficacy data after mobilization with granulocyte colony-stimulating factor (G-CSF) according to healthy donor's (HDs) age as follows: <50 years (HDs-1, n = 161), aged 50 to 59 years (HDs-2, n = 62), and ≥60 years or over (HDs-3, n = 23). Two hundred forty-six HDs were evaluated, and their characteristics were well balanced among age groups: most were male, siblings, and HLA matched. According to age group, the median numbers of CD34(+) cells in the peripheral blood for HDs-1, HDs-2, and HDs-3 were, respectively, 44.5, 34.5, and 26 (HDs-1 versus HDs-2, P = .002; HDs-1 versus HDs-3, P = .036; HDs-2 versus HDs-3, P = n.s.) at day 4 and 65.5, 58, and 46 (HDs-1 versus HDs-2, P = .039; HDs-1 versus HDs-3, P = .002; HDs-2 versus HDs-3, P = n.s.) at day 5. With a median apheresis session of 1, the number of CD34(+) cells/kg recipient body weight collected was not significantly different (6.4 in HDs-1, 6.0 in HDs-2, and 5.7 in HDs-3, P = n.s.). Short- and long-term safety did not differ among age groups. Bone pain was reported as the most frequent short-term adverse event (76.5%). After a median follow-up of 7.8 years, the observed rate of solid tumors, hematological malignancies, and cardiovascular and autoimmune events was similar to the expected incidence for these diseases in Western countries. These results show that G-CSF is effective in the mobilization of older HDs. Moreover, our data contribute to the growing body of evidence in support of the long-term safety of G-CSF for allogeneic donor stem cell mobilization also for elderly HDs.

Tolerability and efficacy of busulfan and fludarabine as allogeneic pretransplant conditioning therapy in acute myeloid leukemia: comparison with busulfan and cyclophosphamide regimen.

BACKGROUND: The aim of this study was to compare safety and efficacy of the association of busulfan with cyclophosphamide (BuCy2) versus busulfan and fludarabine (BuFlu) as a conditioning regimen in allogeneic hematopoietic progenitor cell transplantation (allo-HPCT) in patients with acute myeloid leukemia (AML). PATIENTS AND METHODS: A total of 65 consecutive patients who received an allo-HPCT from Human Leucocyte Antigen-matched sibling donors were analyzed. The conditioning was BuCy2 in 48 patients and BuFlu in 17 patients. RESULTS: There were no significant differences between the 2 cohorts in hematological engraftment, incidence of extrahematological toxicities, and acute graft versus host disease (GVHD). The incidence of chronic GVHD was 34% in the BuCy2 group versus 57% in the BuFlu group (P = .03). Transplant-related mortality was 17% (8 patients) in the BuCy2 group versus 0 in the BuFlu arm. Disease-related mortality was similar in the whole study population; in high-risk AML patients it was 11% in the BuCy2 group and 19% in the BuFlu group (P = .015). The probability of disease-free and event-free survival at 2 years was, respectively, 70% and 60% in the BuCy2 group and 59% and 58% in the BuFlu group (P = .06 and P = not significant [ns]). The probability of overall survival at 2 years was 71% in the BuCy2 group and 63% in the BuFlu group (P = ns), and in the high-risk group it was 83% and 67% in the BuCy2 and BuFlu group, respectively (P = ns). CONCLUSION: BuFlu is well tolerated and is less toxic than BuCy2 and our results did not suggest that in high-risk AML, BuCy2 should be the favorite regimen in terms of efficacy.

Long-term results in multiple myeloma after high-dose melphalan and autologous transplantation according to response categories in the era of old drugs.

BACKGROUND: The aim of this study was to investigate the correlation between the long-term prognosis of multiple myeloma (MM) and the quality of response to therapy in a cohort of 173 patients treated with high-dose melphalan (HDM) and autologous transplantation in the era of old drugs. PATIENTS AND METHODS: A total of 173 patients with de novo MM who received a transplant between 1994 and 2010 were analyzed. VAD (vincristine, doxorubicin [Adriamycin], dexamethasone) was used as front-line regimen before auto-HPCT. The conditioning was HDM 200 mg/m(2). Patients were evaluated for clinical response using the criteria from the European Group for Blood and Marrow Transplantation, modified to include near complete remission (nCR) and very good partial remission (VGPR). RESULTS: The response distribution after transplantation in our series was complete remission (CR) in 33 cases (19%), nearly complete remission (nCR) in 38 cases (22%), VGPR in 30 cases (17%), partial remission (PR) in 65 cases (38%), and stable disease (SD) in 7 cases (4%). Patients were followed for 48 ± 36 months. Median overall survival (OS) was not reached for the CR group. Progression-free survival (PFS) was 122 months for CR, 55 months for nCR, 56 months for VGPR, 32 months for PR, and 22 months for SD. Significant differences in PFS and OS were found between the CR and nCR groups (P = .003 and P = .001, respectively), between the CR and VGPR groups (P = .002 and P = .001, respectively), and between the CR and PR groups (P = .000 and P = .001, respectively). Responses were clustered in 3 main categories, ie, CR, nCR + VGPR + PR, and SD. The respective 10-year PFS and OS values were 58% and 70% for CR, 15% and 18% for nCR + VGPR + PR, and 0% and 0% for SD. CONCLUSION: The achievement of depth and prolonged response represents the most important prognostic factor. The relapse rate is low for patients in CR after 10 years of follow-up, possibly signifying a cure.