Long Term Survival Analysis after High-Dose Melphalan Chemotherapy Followed by Autologous Stem Cell Transplantation for Treatment of AL Amyloidosis: A Single Centre Experience.

Blood ◽  
2005 ◽  
Vol 106 (11) ◽  
pp. 5503-5503
Author(s):  
Jolanta B. Perz ◽  
Amin Rahemtulla ◽  
Chrissy M. Giles ◽  
Richard Szydlo ◽  
Jane F. Apperley
Keyword(s):  
Stem Cells ◽  
Overall Survival ◽  
Stem Cell ◽  
Stem Cell Transplantation ◽  
Autologous Stem Cell Transplantation ◽  
High Dose ◽  
Al Amyloidosis ◽  
Term Survival ◽  
High Dose Melphalan

Abstract Introduction: AL amyloidosis (AL) is a clonal plasma cell disease with a median survival of 10–14 months without therapy. Phase II studies report remission rates in up to 50% of patients (pts) after high-dose melphalan (HDM) and autologous stem cell transplantation (ASCT). We retrospectively analysed AL pts who underwent HDM and ASCT at the Hammersmith Hospital in London since 1996. Patients and methods: 19 pts (12 m, 7 f), median age 51 y (40 – 63 y) with lambda (14) or kappa (5) AL (2 pts with multiple myeloma stage IA) had the following leading organ involvements: 10 kidney, 3 cardiac, 3 liver or gut, 3 other. 12 pts had more than 2 involved organs. According to risk criteria for HDM (Comenzo and Gertz, 2002) 12 pts were high, 2 pts intermediate and 5 pts low risk. Stem cells were mobilised with Cyclophosphamide (4 g/m2) in 3 pts, Etoposide (1.6 g/m2) in 1 patient, G-CSF alone in 14 pts, 1 bone marrow harvest was performed. Melphalan dose was: 8 pts 200 mg/m2, 5 pts 140 mg/m2, 6 pts 100 mg/m2. The median number of infused stem cells was 3.45 x 106 CD 34+ /kg bw (1.96 – 11.3 x 106 CD 34+ /kg bw). Results: Treatment related mortality (TRM) was 26%. In 14 patients who survived longer than 100 days from ASCT 6 pts achieved a haematological remission (5 a complete remission, 1 a partial remission) but in 8 patients no remission was achieved: 2 pts received a second ASCT, 2 pts further chemotherapy, 2 pts required haemodialysis. The mean survival from HDM and ASCT is 58.1 months (SD 9.64 months, 95% CI 39.21 – 76.99). The 1 - year and 2 - years overall survival from HDM and ASCT was 73% and 62%, respectively. The overall survival dropped to 42% after 6 years and the main cause of death was progressive or relapsed AL. Conclusions: HDM and ASCT is feasible for patients with AL, however, TRM was high at 26% in a group of maily high risk patients. 62% of patients survived longer than 2 years, but disease relapse and deaths from relapsed or progressive disease occurred after longer than 2 years from HDM. Thus, new treatment strategies have to be investigated to treat disease progress and relapse in order to improve the long-term survival after HDM and ASCT in patients with AL.

Hepatic Response after High-Dose Melphalan and Stem Cell Transplantation for AL Amyloidosis Associated Liver Disease.

Blood ◽  
2007 ◽  
Vol 110 (11) ◽  
pp. 2873-2873
Author(s):  
Saulius Girnius ◽  
David C. Seldin ◽  
Martha Skinner ◽  
Kathleen T. Finn ◽  
Vaishali Sanchorawala
Keyword(s):  
Stem Cells ◽  
Overall Survival ◽  
Stem Cell ◽  
Stem Cell Transplantation ◽  
High Dose ◽  
Al Amyloidosis ◽  
Hepatic Involvement ◽  
High Dose Melphalan ◽  
Treatment Related Mortality ◽  
Related Mortality

Abstract Immunoglobulin light chain (AL) amyloidosis is a plasma cell dyscrasia with extracellular protein deposition in various organ systems, including the liver. The natural history of AL amyloidosis with liver involvement has a poor prognosis, with median survival of only 8.5 to 9 months. High-dose melphalan chemotherapy and autologous peripheral blood stem cell transplantation (HDM/SCT) has been shown to result in durable hematologic response and prolonged overall survival in systemic AL amyloidosis. Patients were included who had hepatic involvement and received HDM/SCT from 1998 to 2006. Data was collected with the approval of the Institutional Review Board. Patients receiving HDM/SCT had to meet eligibility criteria to qualify for this aggressive treatment. Stem cells were mobilized using G-CSF alone. Intravenous melphalan was administered at 100–200 mg/m2 over 2 days in divided doses and stem cells were re-infused 24–72 hours after HDM. Liver response was determined by criteria set up by the Consensus Opinion from the 10th International Symposium on Amyloid and Amyloidosis. Sixty-nine patients qualified for this study, including 47 males and 22 females. The median age was 56 years (range, 37–75), median bilirubin was 0.5 mg/dL (range, 0.1 to 5.7), median alkaline phosphatase was 193 U/L (range, 59 to 1243) and the median liver size was 3 cm (range, 0–20cm) below the costal margin. Nine patients (13%) died from treatment-related mortality. The hematologic CR one year after treatment was achieved by 53% (31/58) of evaluable patients. The overall survival was 84% at 1 year and 49% at 5 years, by Kaplan-Meier estimates. Hepatic response occurred in 21% (10/47) at 1 year after HDM/SCT and 40% (12/30) at 2 years after HDM/SCT. In summary, hepatic involvement with AL amyloidosis does not increase treatment-related mortality from HDM/SCT. Hepatic response follows a hematological response and can occur as far as 2 years from HDM/SCT. Forty % of surviving evaluable patients showed remission of their hepatic disease from AL amyloidosis by 2 years.

Modified High-Dose Melphalan and Autologous Stem Cell Transplantation (mHDM/SCT) In the Treatment of AL Amyloidosis (AL) and/or High-Risk Myeloma (hM): Analysis of SWOG Trial S0115

Blood ◽  
2011 ◽  
Vol 118 (21) ◽  
pp. 2004-2004
Author(s):  
Vaishali Sanchorawala ◽  
Antje Hoering ◽  
David C Seldin ◽  
Kathleen T Finn ◽  
Salli A Fennessey ◽  
...  
Keyword(s):  
Overall Survival ◽  
Stem Cell ◽  
Stem Cell Transplantation ◽  
Cell Transplantation ◽  
Autologous Stem Cell Transplantation ◽  
Research Funding ◽  
High Dose ◽  
Al Amyloidosis ◽  
Advisory Committees ◽  
High Dose Melphalan

Abstract Abstract 2004 Treatment of AL amyloidosis (AL) and myeloma (M) with high dose melphalan and autologous stem cell transplantation (HDM/SCT) is effective in inducing hematologic remissions and improving survival. However, the benefit of HDM/SCT in AL and host-based high-risk myeloma (hM) has not been explored in a multi-center study. We designed a trial of two cycles of modified high dose melphalan at 100 mg/m2 and autologous stem cell transplantation (mHDM/SCT) through SWOG 0115 (ClinicalTrials.govIdentifier: NCT00064337). The primary objective was to evaluate overall survival and additional objectives were to determine hematologic responses and tolerability of two cycles of mHDM/SCT in AL and hM in a multicenter study. Eligibility for patients with AL required evidence of tissue diagnosis of amyloidosis, underlying associated plasma cell dyscrasia, and adequate measures of performance status (Zubrod 0–2) and cardiopulmonary function (LVEF >45%, DLCO >50%). Eligibility for hM patients required age >70 years and/or serum creatinine of >2 mg/dL or calculated creatinine clearance of <50 mL/kg/min. Peripheral blood stem cells were collected following G-CSF alone with minimum yield of 7.0×106 CD34+ cells/kg required for participation in the trial. From 1/2004 to 11/2010, 70 eligible patients with AL (61 with AL and 9 with myeloma associated AL) and 27 with hM were enrolled at 17 centers in the US. The median age was 64 years (range; 33–79) and M:F ratio was 1.6:1. The median number of organs involved was 2 (range, 1–8). There were 22 patients (31%) with cardiac involvement. The median serum creatinine level was 1.7 mg/dL (range, 0.6–10.0) for patients with hM. There are 68 patients with AL and 25 patients with hM eligible for survival and 67 patients with AL and 25 patients with hM eligible for adverse event analysis at this time. The treatment-related mortality (TRM), defined as deaths within 100 days of registration (even without protocol-directed treatment) was 9% (n=9/97). TRM was 10% (n=7/70) for AL and 7% (n=2/27) for hM. TRM was 14% (n=3/22) for patients with AL and cardiac involvement. Grade 3 and higher non-hematologic adverse events by CTCAEv3.0 were experienced by 75% (n=50/67) of AL patients and 80% (n=20/25) of hM patients. The median overall survival is 68 months for AL with a median follow up for surviving patients of 40.6 months (range; 1.2–79). The median survival for hM patients has not been reached yet with a median follow-up of 34 months. The 5-year survival is 55% for AL and 54% for hM patients; and the median progression-free-survival is 43 months for AL and 31 months for hM. Hematologic responses, defined by the standard consensus criteria, were achieved by 39% (n=11/28) evaluable patients for AL and 57% (n=4/7) for hM following SCT. Clinical and organ responses were also evident at 1 year following HDM/SCT. Thirty % (n=3/10) patients with AL experienced cardiac response. In conclusion, this experience demonstrates that with careful selection of patients, mHDM for SCT in patients with AL amyloidosis and hM, even in the setting of a multicenter study, can lead to prolonged overall survival with acceptable TRM and morbidity. Disclosures: Holmberg: Celgene: Research Funding; Millennium-Takeda: Research Funding; Otsuka: Research Funding; Seattle Genetics: Research Funding; Genzyme: Research Funding; Genzyme: Research Funding; Merck: Research Funding; Genzyme: Membership on an entity's Board of Directors or advisory committees; Seattle Genetics: Membership on an entity's Board of Directors or advisory committees.

scholarly journals Long-term outcome of patients with AL amyloidosis treated with high-dose melphalan and stem-cell transplantation

Blood ◽  
2007 ◽  
Vol 110 (10) ◽  
pp. 3561-3563 ◽  
Author(s):  
Vaishali Sanchorawala ◽  
Martha Skinner ◽  
Karen Quillen ◽  
Kathleen T. Finn ◽  
Gheorghe Doros ◽  
...  
Keyword(s):  
Stem Cell ◽  
Stem Cell Transplantation ◽  
Cell Transplantation ◽  
Median Survival ◽  
High Dose ◽  
Al Amyloidosis ◽  
Term Survival ◽  
Long Term Outcome ◽  
High Dose Melphalan

AbstractLong-term survival and outcome were determined for 80 patients with immunoglobulin light chain (AL) amyloidosis treated with high-dose melphalan and stem cell transplantation (HDM/SCT) more than 10 years ago. Seventeen (21%) patients died within the first year of treatment, of treatment-related complications (14%) or progressive disease (8%). Of the 63 surviving evaluable patients at one year, 32 (51%) achieved a complete hematologic response (CR). For all 80 patients, the median survival was 57 months (4.75 yrs). The median survival exceeds 10 years for patients achieving a CR after HDM/SCT, compared with 50 months for those not achieving a CR (P < .001). In conclusion, HDM/SCT leads to durable remissions and prolonged survival, particularly for those patients who achieve a hematologic CR.

Short and long-term outcome of treatment with high-dose melphalan and stem cell transplantation for multiple myeloma-associated AL amyloidosis

2009 ◽  
Vol 89 (6) ◽  
pp. 579-584 ◽  
Author(s):  
Saulius Girnius ◽  
David C. Seldin ◽  
Martha Skinner ◽  
Kathleen T. Finn ◽  
Karen Quillen ◽  
...  
Keyword(s):  
Multiple Myeloma ◽  
Stem Cell ◽  
Stem Cell Transplantation ◽  
High Dose ◽  
Al Amyloidosis ◽  
Term Outcome ◽  
Long Term Outcome ◽  
Short And Long Term ◽  
High Dose Melphalan

scholarly journals Long-term outcomes of high dose treatment and autologous stem cell transplantation in follicular and mantle cell lymphomas – a single centre experience

2016 ◽  
Vol 51 (1) ◽  
pp. 81-87 ◽  
Author(s):  
Lucka Boltezar ◽  
Karlo Pintaric ◽  
Jože Pretnar ◽  
Maja Pohar Perme ◽  
Barbara Jezersek Novakovic
Keyword(s):  
Overall Survival ◽  
Stem Cell ◽  
Stem Cell Transplantation ◽  
Mantle Cell Lymphoma ◽  
Autologous Stem Cell Transplantation ◽  
Cell Lymphoma ◽  
High Dose ◽  
High Dose Treatment ◽  
Mantle Cell

Abstract Background Advanced follicular lymphoma (FL) and mantle cell lymphoma (MCL) are incurable diseases with conventional treatment. The high dose treatment (HDT) with autologous stem cell transplantation (ASCT), however, offers a certain proportion of these patients the prospect of a prolonged disease-free and overall survival. The aim of this study was to investigate the event free survival (EFS) and overall survival (OS) in patients with FL and MCL treated with ASCT. Patients and methods Seventeen patients with FL and 29 patients with MCL were included, 15 of them were transplanted to consolidate the response to second line treatment and 24 to consolidate their first remission, respectively. All were conditioned with total body irradiation (TBI) and high dose cyclophosphamide between 2006 and 2014 and all were transplanted with peripheral blood stem cells. Results The estimated 5-year OS for FL was 87.8% (95% confidence interval [CI] 59.5%–96.8%) and for MCL 79.3% (95% CI 56.1%–91.1%), respectively. The estimated 5-year EFS for FL was 76.0% (95% CI 48.0%–90.3%) and for MCL 69.8% (95% CI 45.5%–84.8%), respectively. There were no secondary hematological malignancies observed in either group. Conclusions Based on above results, the ASCT with TBI is a good treatment option in terms of long-term survival for patients with follicular and mantle cell lymphoma demonstrating a relatively low rate of late toxicities and secondary malignancies.

Sign in / Sign up

Export Citation Format

Share Document