Long-Term Survival (10 Years or More) in 30 Patients With Primary Amyloidosis

Blood ◽  
1999 ◽  
Vol 93 (3) ◽  
pp. 1062-1066 ◽  
Author(s):  
Robert A. Kyle ◽  
Morie A. Gertz ◽  
Philip R. Greipp ◽  
Thomas E. Witzig ◽  
John A. Lust ◽  
...  
Keyword(s):  
Objective Response ◽  
Al Amyloidosis ◽  
Primary Amyloidosis ◽  
Secondary Amyloidosis ◽  
Actuarial Survival ◽  
Histologic Diagnosis ◽  
Term Survival ◽  
Prognostic Features ◽  
Bone Marrow Plasma

The median survival in primary systemic (AL) amyloidosis is less than 18 months. No published series of patients with AL amyloidosis have reported survival of more than 10 years. The records of all Mayo Clinic patients with a diagnosis of AL amyloidosis between January 1, 1966 and March 1, 1987 were reviewed. Patients with secondary amyloidosis, familial amyloidosis, senile systemic amyloidosis, and localized amyloidosis were excluded. During the 21 years of the study, 841 patients with AL amyloidosis were seen. Of these, 29 were excluded because the diagnosis was made at autopsy, and 2 others were excluded because no follow-up data were available. Actuarial survival for the 810 patients was 51% at 1 year, 16% at 5 years, and 4.7% at 10 years. Thirty patients survived for 10 years or more after the histologic diagnosis of AL amyloidosis; all received alkylating-agent therapy. In 14 patients, the monoclonal protein disappeared from the serum or urine. Of 10 patients with nephrotic syndrome, 4 had an objective response. Congestive heart failure, older age, creatinine value of 2 mg/dL or more, bone marrow plasma cell value of 20% or more, platelet count of 500 × 109/L or less, and the presence of peripheral neuropathy were underrepresented in the 10-year survivors and are unfavorable prognostic features. Five percent of patients with AL amyloidosis survived for 10 years or more.

Long-Term Survival (10 Years or More) in 30 Patients With Primary Amyloidosis

Blood ◽  
1999 ◽  
Vol 93 (3) ◽  
pp. 1062-1066 ◽  
Author(s):  
Robert A. Kyle ◽  
Morie A. Gertz ◽  
Philip R. Greipp ◽  
Thomas E. Witzig ◽  
John A. Lust ◽  
...  
Keyword(s):  
Objective Response ◽  
Al Amyloidosis ◽  
Primary Amyloidosis ◽  
Secondary Amyloidosis ◽  
Actuarial Survival ◽  
Histologic Diagnosis ◽  
Term Survival ◽  
Prognostic Features ◽  
Bone Marrow Plasma

Abstract The median survival in primary systemic (AL) amyloidosis is less than 18 months. No published series of patients with AL amyloidosis have reported survival of more than 10 years. The records of all Mayo Clinic patients with a diagnosis of AL amyloidosis between January 1, 1966 and March 1, 1987 were reviewed. Patients with secondary amyloidosis, familial amyloidosis, senile systemic amyloidosis, and localized amyloidosis were excluded. During the 21 years of the study, 841 patients with AL amyloidosis were seen. Of these, 29 were excluded because the diagnosis was made at autopsy, and 2 others were excluded because no follow-up data were available. Actuarial survival for the 810 patients was 51% at 1 year, 16% at 5 years, and 4.7% at 10 years. Thirty patients survived for 10 years or more after the histologic diagnosis of AL amyloidosis; all received alkylating-agent therapy. In 14 patients, the monoclonal protein disappeared from the serum or urine. Of 10 patients with nephrotic syndrome, 4 had an objective response. Congestive heart failure, older age, creatinine value of 2 mg/dL or more, bone marrow plasma cell value of 20% or more, platelet count of 500 × 109/L or less, and the presence of peripheral neuropathy were underrepresented in the 10-year survivors and are unfavorable prognostic features. Five percent of patients with AL amyloidosis survived for 10 years or more.

Rebuttal

2004 ◽  
Vol 14 (S1) ◽  
pp. 114-114 ◽  
Author(s):  
Leonard L. Bailey
Keyword(s):  
Coronary Disease ◽  
Reconstructive Surgery ◽  
Fontan Circulation ◽  
Actuarial Survival ◽  
Term Survival ◽  
Long Term Survival ◽  
Fontan Physiology ◽  
Failing Fontan

The portrayal of a beautiful youngster performing uninhibited acrobatics based on Fontan physiology, as presented by Marshall Jacobs, is a brilliant and beautiful thing for us to see. It is, perhaps, all about will over physiology. But it is, nevertheless, happening for that child. Marshall mentioned the need for re-transplantation, whether the beginning strategy was transplantation or reconstructive surgery. Indeed, a relatively small percentage of children transplanted will require re-transplantation because of severe graft coronary disease. Remarkably, in the Loma Linda experience, 10-year actuarial survival for 26 patients following elective re-transplantation is over 85%, exceeding overall actuarial survival at 10 years for children following primary transplantation. Many of the transplanted infants, however, seem to be realistically hopeful that one heart will last their entire lifetime. Of course, the hope is that their's will be a long and healthy lifetime. The requirement for late transplantation following Fontan procedures, however, seems almost inevitable. We'll simply have to keep these children with Fontan circulation under surveillance to see when, in the course of their lives, transplantation will become necessary. Unfortunately, operative and long-term survival among children who are transplanted for failing Fontan physiology have, as yet, been somewhat suboptimal.

scholarly journals Leiomyosarcoma of Prostate

2020 ◽  
Vol 1 (1) ◽  
Author(s):  
Abdeljalil Heddat ◽  
Younes Houry ◽  
Redouane Rabii
Keyword(s):  
Therapeutic Approach ◽  
Multidisciplinary Approach ◽  
Malignant Tumors ◽  
Survival Rates ◽  
Multicenter Trial ◽  
Advanced Stage ◽  
Rare Tumor ◽  
Actuarial Survival ◽  
Term Survival

Prostate leiomyosarcoma is an extremely rare and very aggressive neoplasm that represents less than 0.1% of primary malignant tumors of the prostate. We present a patient with primary leiomyosarcoma of  prostate and examine the cases reported in the literature to discuss the clinical, diagnostic and therapeutic aspects of this rare tumor. Median survival was estimated at 17 months (95% CI 20.7–43.7 months) and the actuarial survival rates at 1, 3 and 5 years were 68%, 34% and 26%, respectively. The only predictors of long-term survival were negative surgical margins and the absence of metastatic disease at presentation. A multidisciplinary approach is necessary for the proper management of this terrible entity. Surgery with or without chemotherapy seems to be the main therapeutic method for operable leimyosarcomas, but in general there is no consensus on the best therapeutic approach. Most cases are diagnosed at an advanced stage of the disease. A global multicenter trial is needed to find therapies that would improve the prognosis.

Improvement of long-term survival in extensive small-cell lung cancer.

1988 ◽  
Vol 6 (7) ◽  
pp. 1161-1169 ◽  
Author(s):  
D V Jackson ◽  
L D Case ◽  
P J Zekan ◽  
B L Powell ◽  
R D Caldwell ◽  
...  
Keyword(s):  
Lung Cancer ◽  
Small Cell Lung Cancer ◽  
Cell Lung Cancer ◽  
Objective Response ◽  
Complete Response ◽  
Treated Group ◽  
Small Cell ◽  
Small Cell Lung ◽  
Term Survival

The effect of adding the epipodophyllotoxin etoposide (VP-16-213) to a standard chemotherapy regimen for patients with extensive stage small-cell lung cancer was evaluated during a randomized trial. Chemotherapy consisted of vincristine, doxorubicin, and cyclophosphamide (VAC) alone or with etoposide (EVAC). Of 139 patients enrolled, 136 patients were eligible for study and all but five were evaluable for response. The overall objective response was 46% in the VAC group v 70% in the etoposide-treated group (P = .008) with complete response (CR) rates of 12% v 29%, respectively (P = .030). Although the time to the observation of disease progression was significantly longer in the group of patients receiving etoposide (9.6 v 6.5 months, P = .010), overall survival was similar; this was probably due to administration of other agents including etoposide at the time of VAC failure. However, there were noteworthy differences in long-term (greater than or equal to 2 year) survival. Whereas only four (6%) patients treated with VAC lived 2 years, 11 (16%) of the etoposide-treated group did so (P = .100). Two-year failure-free survival was attained in one (2%) of the VAC patients and eight (11%) of the patients treated with etoposide (P = .034). Long-term survivorship, heretofore usually reported in patients with limited stage disease after a variety of treatments, may be possible with this drug combination in the setting of extensive disease.

Role of Lung Transplantation in the Treatment of Bronchogenic Carcinomas for Patients With End-Stage Pulmonary Disease

2004 ◽  
Vol 22 (21) ◽  
pp. 4351-4356 ◽  
Author(s):  
Marc de Perrot ◽  
Susan Chernenko ◽  
Thomas K. Waddell ◽  
Yaron Shargall ◽  
Andrew F. Pierre ◽  
...  
Keyword(s):  
Lung Transplantation ◽  
Bronchogenic Carcinoma ◽  
Stage I ◽  
Bronchioloalveolar Carcinoma ◽  
Actuarial Survival ◽  
Term Survival ◽  
Primary Indication ◽  
End Stage

Purpose To determine the role of lung transplantation in the treatment of patients presenting with bronchogenic carcinoma and end-stage lung disease. Methods An international survey was conducted to determine the outcome of patients with bronchogenic carcinoma in the explanted lung at the time of transplantation. A group of 69 patients was collected from 33 centers. Results Twenty-six patients underwent 29 lung transplantations for advanced multifocal bronchioloalveolar carcinoma (BAC) as the primary indication for transplantation, and 13 developed a recurrence, with an overall 5-year actuarial survival of 39%. Incidental bronchogenic carcinomas classified as stage I (n = 22), II (n = 12), and III (n = 2), or as incidental multifocal BAC (n = 7), were found in the explanted lung of the remaining 43 patients. The 5-year actuarial survival was 51% in patients with stage I carcinomas, and was significantly better than for patients with stage II and III carcinomas (survival of 14%) or with incidental multifocal BAC (survival of 23%). Time from transplantation to recurrence and from recurrence to death was significantly longer in patients with multifocal BAC than in patients with other types of bronchogenic carcinoma. In addition, the site of recurrence was limited to the transplanted lung in 88% of the patients with multifocal BAC, whereas it was always widespread in patients with other types of bronchogenic carcinoma. Conclusion This study demonstrates that long-term survival can be achieved after lung transplantation in patients with stage I bronchogenic carcinoma or with advanced multifocal BAC.

Relapse Rate and Long-Term Survival of AL Amyloidosis Patients Treated with High-Dose Melphalan and Autologous Stem Cell Transplantation (HDM/SCT).

Blood ◽  
2006 ◽  
Vol 108 (11) ◽  
pp. 3094-3094
Author(s):  
David C. Seldin ◽  
Martha Skinner ◽  
Betul Oran ◽  
Karen Quillen ◽  
Kathleen T. Finn ◽  
...  
Keyword(s):  
Median Survival ◽  
Plasma Cell ◽  
Relapse Rate ◽  
Long Term Results ◽  
High Dose ◽  
Al Amyloidosis ◽  
Term Survival ◽  
Long Term Survival ◽  
Early Results

Abstract AL amyloidosis is a plasma cell dyscrasia in which clonal immunoglobulin light chains misfold and are deposited in tissues, leading to organ failure in untreated patients, with median survival of only ~1 year. Oral melphalan and prednisone is minimally effective for the disease, with an increase in median survival to ~1.5 years, and a low rate of hematologic complete responses (CRs). Twelve years ago, we began treating patients with AL amyloidosis with HDM/SCT. In the plasma cell malignancy multiple myeloma, this approach produces hematologic CRs and improves survival, but is not curative, as all patients eventually relapse. In AL amyloidosis, the relapse rate and long-term survival have not been studied, but early results are promising, with most centers reporting CR rates of ~40% and excellent survival in responding patients. To address the durability and long-term results of treatment with HDM/SCT, here we report on the outcome for AL amyloidosis patients treated at Boston Medical Center with HDM/SCT >10 years ago. The first autologous transplant took place on July 18, 1994 in two years, by July 18, 1996, 43 patients with AL amyloidosis without myeloma or other hematologic disease had been treated with HDM/SCT, receiving 100–200 mg/m2 melphalan depending upon age and protocol. Of the 43 patients treated in this 2 yr period, 76% of the patients were male, 81% had a lambda monoclonal disease, and their median age was 52 (range, 29–71). In these first 43 patients, the 100 day peri-transplant mortality rate was 16%. Nineteen of the 43 patients (44%) achieved a hematologic CR after treatment. In annual followup, 4 of 19 patients (21%) eventually relapsed. Fourteen of 43 patients (33%) are still alive; 12 of 19 patients who achieved a CR (63%) are still alive, while only 2 of 34 patients who did not (6%) are still alive. Although there were fewer (8) patients with kappa clonal disease, they had a better outcome, with an 87% CR rate vs. 34% for lambda (P=0.006); 75% of the kappa patients are still alive, vs. 23% of the lambda patients (P=0.005). The median survival of all 43 patients is 4.7 years. Thus, treatment of AL amyloidosis patients with HDM/SCT produces a high CR rate that is durable and is associated with excellent 10 year survival, particularly for those patients achieving a hematologic CR and for patients with kappa clonal disease. Ongoing clinical trials of HDM/SCT, along with strategies to reduce morbidity and mortality and to improve the CR rate, incorporating additional cycles of HDM/SCT or new anti-plasma cell agents, appear to be well-justified by these results.

A Profile of 44 Long Term Survivors (>10yrs) with AL Amyloidosis.

Blood ◽  
2007 ◽  
Vol 110 (11) ◽  
pp. 4765-4765
Author(s):  
Ashutosh D. Wechalekar ◽  
Helen J. Lachmann ◽  
Julian D. Gillmore ◽  
Philip N. Hawkins
Keyword(s):  
Soft Tissues ◽  
Performance Status ◽  
Organ Involvement ◽  
Whole Body ◽  
Al Amyloidosis ◽  
Term Survival ◽  
Long Term Outcome ◽  
Significant Difference ◽  
Amyloid Load

Abstract AL amyloidosis has a poor outcome. Survival of only about one year was frequently reported in studies performed in the 1990s, and the diagnosis of AL amyloidosis continues to be widely regarded as incompatible with long term survival. We report here the features of patients with AL amyloidosis followed up at the UK Amyloidosis centre (NAC) for more than 10 years, who encouragingly represented more than 10% of all cases. All patients with AL amyloidosis who first attended the NAC between 1979–1997 and subsequently survived for more than 10 years were included in this study. AL type amyloidosis was confirmed in all patients histologically with genetic studies to robustly exclude hereditary amyloidosis as indicated. Organ involvement and responses were defined as per the international consensus criteria (Gertz et al 2005). I123 serum amyloid P (SAP) scintigraphy was used to identify additional organ involvement and monitor amyloid load. 361 patients with AL amyloidosis were assessed at the NAC before 1st August 1997, with a median survival of 2.4 yrs. 132 (20%) patients survived >5 yrs, and 44 (12%) patients survived >10 years with 2 patients living ≥ 16 yrs and 7 for ≥ 14 yrs. Three patients died at 10.2, 11 and 13 years due to unrelated causes with no deaths due to progressive amyloidosis. Among the 44 ten year survivors, at presentation, median age was 51 yrs (29–70), median creatinine 86μmol/L (47–400), 24 hour proteinuria 2g (<0.1g–50g), bilirubin 8μmol/L (2–130), alkaline phosphatase 82.5 units/L (41–2645), and median NT-ProBNP 19 pMol/L (1–2158). Median ECOG performance status was 1 (0–2). Organ involvement (consensus criteria) was: renal 31 (70%); liver 6 (14%); cardiac 8 (18%); autonomic neuropathy 1(2%); peripheral neuropathy - none; gastrointestinal 2(4%) and lymph nodes or other soft tissues 4 (9%). SAP scintigraphy showed renal amyloid deposits in 30 (68%), liver 11(25%), adrenals 3(7%) and bone infiltration 7 (16%). 35 (79%) had one organ involved, 8 (18%) had 2 organs and 1 (3%) patient had three organs involved by consensus criteria; SAP scintigraphy detected additional organ involvement in 12 cases (27%). The whole body amyloid load on SAP scintigraphy was small in 25(57%), moderate 2 (4%) and large 9 (21%). Median plasma cell infiltrate in the marrow was 5% (1–65). Treatment received comprised an alkylator based regimen in 11(26%), VAD chemotherapy in 14 (32%), and stem cell transplantation (SCT) in 18 (42%). 29 (67%) patients had a complete clonal response, 13 (30%) had a partial response and 1 (2%) had no response. 25(57%) had evidence of organ improvement by conventional criteria and in 26 (59%) SAP scintigraphy showed regression of amyloid. The median time to next treatment (progression free survival - PFS) has not been reached at 10 yrs. There was no significant difference in the PFS between patients treated chemotherapy or SCT, or among the complete or partial responders. 13 patients (30%) developed end stage renal disease (ESRD) a median 5.8 yrs after diagnosis and this was not significantly more frequent among partial responders compared with complete hematologic responders. Substantial improvements in diagnosis, monitoring and treatment of AL amyloidosis have occured since 1997. It is all the more encouraging that we are able to report here that 12% of patients diagnosed before this time survived for more than 10 years and patients with AL amyloidosis diagnosed more recently are likely to have even better prospect of good long term outcome in cases who achieve good and sustained clonal responses to therapy.

scholarly journals Objective Response to Radiation Therapy and Long-Term Survival of Patients with WHO Grade II Astrocytic Gliomas with Known LOH 1p/19q Status

2007 ◽  
Vol 183 (9) ◽  
pp. 517-522 ◽  
Author(s):  
Ali-Reza Fathi ◽  
Erik Vassella ◽  
Marlene Arnold ◽  
Jürgen Curschmann ◽  
Michael Reinert ◽  
...  
Keyword(s):  
Radiation Therapy ◽  
Objective Response ◽  
Who Grade ◽  
Term Survival ◽  
Long Term Survival ◽  
Grade Ii ◽  
Astrocytic Gliomas

scholarly journals Overall Survival of Patients With Unresectable or Metastatic BRAF V600-Mutant Acral/Cutaneous Melanoma Administered Dabrafenib Plus Trametinib: Long-Term Follow-Up of a Multicenter, Single-Arm Phase IIa Trial

2021 ◽  
Vol 11 ◽  
Author(s):  
Lili Mao ◽  
Ya Ding ◽  
Xue Bai ◽  
Xinan Sheng ◽  
Jie Dai ◽  
...  
Keyword(s):  
Overall Survival ◽  
Cutaneous Melanoma ◽  
Objective Response ◽  
Chinese Patients ◽  
Term Survival ◽  
Long Term Survival ◽  
Acral Melanoma ◽  
Long Term Follow Up

ObjectivesTo examine the long-term survival outcome of dabrafenib in combination with trametinib in Chinese patients with unresectable or metastatic acral/cutaneous melanoma with BRAF-V600 mutation and to explore potential predictors of effectiveness.MethodsThis was a long-term follow-up of Chinese patients with unresectable or metastatic BRAF V600-mutant acral/cutaneous melanoma administered dabrafenib (150 mg twice daily) plus trametinib (2 mg once daily) in an open-label, multicenter, single-arm, phase IIa study (NCT02083354). Efficacy endpoints included objective response rate (ORR), duration of response (DOR), progression-free survival (PFS), and overall survival (OS). The impacts of baseline characteristics on PFS and OS were analyzed.ResultsA total of sixty patients were included. The median age was 48 years, and 24 patients (40.0%) were male. Totally 12 individuals (20.0%) had acral melanoma, and 45 (75.0%) had failed previous systemic therapy. Up to July 2020, the median duration of follow-up was 37.0 (95% confidence interval [CI] 29.1-44.9) months. The updated ORR was 71.7% (95%CI 60.3%-83.1%). The 3-year OS rate was 28.8% (95%CI 19.1-43.6%) in the overall population, and 35.7% (95%CI 15.5–82.4%) in acral melanoma patients. The median DOR was 7.5 months (95%CI 4.5 to 10.5). Baseline normal lactic dehydrogenase (LDH), metastatic organ sites&lt;3 and complete response to combination therapy with dabrafenib plus trametinib were associated with improved PFS and OS.ConclusionDabrafenib combined with trametinib confer long-term survival in Chinese patients with BRAF V600-mutant, unresectable or metastatic acral/cutaneous melanoma.Clinical Trial Registrationhttps://clinicaltrials.gov/ct2/show/NCT02083354, identifier NCT02083354.

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