Delayed Cerebrovascular Complications of Intrathecal Colloidal Gold

ABSTRACT OBJECTIVE Therapy with intrathecal colloidal gold has been used in the past as an adjunct in the treatment of childhood neoplasms, including medulloblastoma and leukemia. We describe the long-term follow-up period of a series of patients treated with intrathecal colloidal gold and emphasize the high incidence of delayed cerebrovascular complications and their management. METHODS Between 1967 and 1970, 14 children with posterior fossa medulloblastoma underwent treatment at the University of Minnesota. Treatment consisted of surgical resection, external beam radiotherapy, and intrathecal colloidal gold. All patients underwent long-term follow-up periods. RESULTS Of the 14 original patients, 6 died within 2 years of treatment; all experienced persistent or recurrent disease. The eight surviving patients developed significant neurovascular complications 5 to 20 years after treatment. Three patients died as a result of aneurysmal subarachnoid hemorrhage, and five developed ischemic symptoms from severe vasculopathy that resembled moyamoya disease. CONCLUSION Although therapy with colloidal gold resulted in long-term survival in a number of cases of childhood medulloblastoma, our experience suggests that the severe cerebrovascular side effects fail to justify its use. The unique complications associated with colloidal gold therapy, as well as the management of these complications, are presented. We recommend routine screening of any long-term survivors to exclude the presence of an intracranial aneurysm and to document the possibility of moyamoya syndrome.

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Carcinoid of the Extra-Hepatic Bile Duct: A Case Report with Long-Term Follow-up and Review of Literature

The American Surgeon ◽

10.1177/000313480807400121 ◽

2008 ◽

Vol 74 (1) ◽

pp. 87-90 ◽

Cited By ~ 3

Author(s):

Niraj J. Gusani ◽

J. Wallis Marsh ◽

Michael A. Nalesnik ◽

Mitchell E. Tublin ◽

T. Clark Gamblin

Keyword(s):

Bile Duct ◽

Carcinoid Tumor ◽

Extrahepatic Bile Duct ◽

Strong Support ◽

Complete Excision ◽

Term Survival ◽

Long Term Survival ◽

Long Term Follow Up

Extrahepatic bile duct tumors, 80 per cent of which are adenocarcinomas, are rare neoplasms accounting for less than two per cent of all cancers. Carcinoid tumor of the extrahepatic bile ducts is a reportable lesion, with only approximately 50 cases described in the literature since 1959. We present a case of a primary extrahepatic bile duct carcinoid tumor resected for cure with the longest reported follow-up time (11 years) after surgery. We also summarize the existing literature with regard to this rare tumor. Our case lends strong support to the notion that extrahepatic biliary carcinoids are generally indolent lesions that, if aggressively resected, can result in excellent long-term survival. Complete excision with clear margins seems to provide the best chance of obtaining long-term survival and cure.

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Outcomes of cardiovascular surgery for chronic dialysis patients in current Japan

Asian Cardiovascular and Thoracic Annals ◽

10.1177/0218492319859147 ◽

2019 ◽

Vol 27 (6) ◽

pp. 464-470

Author(s):

Hiroshi Kurazumi ◽

Masaya Takahashi ◽

Shigeru Ikenaga

Keyword(s):

Survival Rate ◽

Cardiovascular Surgery ◽

Hazard Ratio ◽

Dialysis Patients ◽

Term Survival ◽

Long Term Follow Up ◽

History Of ◽

Atherosclerosis Obliterans

Background The number of dialysis patients in Japan is rising, with an increasing number requiring cardiovascular surgery. Methods We investigated the short- and long-term outcomes in 70 dialysis patients among a total of 1124 who underwent cardiovascular surgery in our hospital between 2004 and 2016. We investigated outcomes following open surgery and identified factors that affected the prognosis. We also compared the long-term survival rate with the survival rate of the Japanese dialysis population. Results The long-term survival rate was 70.6%, 51.1%, and 19.2% after 3, 5, and 10 years, respectively. The causes of long-term death were heart disease in 8 patients, cerebrovascular disease in 7, cachexia in 3, infection in 2, and other causes in 3. The freedom from cardiac death was 88.7%, 77.9%, and 54.9% after 3, 5, and 10 years, respectively. Multivariate analysis using Cox’s proportional hazard model showed that a history of atherosclerosis obliterans (hazard ratio 5.4, p = 0.05) and mediastinitis (hazard ratio 10.2, p = 0.03) were risk factors for death in long-term follow-up, and a history of atherosclerosis obliterans was an independent risk factor for cardiac death in long-term follow-up (hazard ratio 5.3, p = 0.01). Five-year survival of the study subjects was comparable to that of the Japanese dialysis population. Conclusions The prognosis for dialysis patients after open surgery was equivalent to that of Japanese dialysis patients in general. A high proportion of late postoperative deaths were due to heart disease. Patients with atherosclerosis obliterans had a poor prognosis.

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Long Term Follow-up Results of External Beam Radiotherapy as Primary Treatment for Retinoblastoma

Journal of Korean Medical Science ◽

10.3346/jkms.2010.25.4.546 ◽

2010 ◽

Vol 25 (4) ◽

pp. 546 ◽

Cited By ~ 9

Author(s):

Sang Yul Choi ◽

Mi-Sook Kim ◽

SungYul Yoo ◽

ChulKoo Cho ◽

YoungHoon Ji ◽

...

Keyword(s):

External Beam Radiotherapy ◽

Primary Treatment ◽

External Beam ◽

Long Term Follow Up

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LTBK-12. EORTC 26951, RANDOMIZED STUDY OF ADJUVANT PCV AFTER 59.4 GY RADIOTHERAPY: VERY LONG TERM FOLLOW-UP

Neuro-Oncology ◽

10.1093/neuonc/noz219.1201 ◽

2019 ◽

Vol 21 (Supplement_6) ◽

pp. vi285-vi285

Author(s):

Martin van den Bent ◽

Khe Hoang-Xuan ◽

Alba Brandes ◽

Johan Kros ◽

M C M Kouwenhoven ◽

...

Keyword(s):

Randomized Study ◽

Progression Free Survival ◽

Phase Iii ◽

Term Survival ◽

Long Term Survival ◽

Kaplan Meier ◽

Tumor Group ◽

Long Term Follow Up

Abstract BACKGROUND Between 1995 and 2002 the EORTC Brain Tumor Group conducted a prospective phase III study on adjuvant procarbazine, CCNU and vincristine (PCV) chemotherapy in anaplastic oligodendroglioma (AOD). A mature follow-up presented in 2012 showed survival benefit of the addition of PCV, in particular in 1p/19q co-deleted tumors and tumors with MGMT promoter methylation. We now present very long term follow-up. MATERIALS AND METHODS Patients were eligible if locally diagnosed with a newly diagnosed AOD. They were randomized between radiotherapy (RT, 33 x 1.8 Gy) and the same RT followed by 6 cycles PCV (RT/PCV). Primary endpoints were overall survival (OS) and progression free survival (PFS). 1p/19q status (FISH) was determined in 300 patient. Kaplan- Meier technique and Cox modeling were used for long term survival analysis. Primary analyses were adjusted for known prognostic factors. For other analyses no adjustment was performed. RESULTS With 368 patients included, a median follow-up of 18.4 years and 307 (83%) survival events, median and 20-year survival after RT/PCV versus RT alone were 42.3 mo and 16.8% vs 30.6 months and 10.1% (HR 0.78; 95% CI (0.63, 0.98), adjusted p=0.06). Eighty patients were 1p/19q codel of which 26 (33%) were still alive, in this subgroup median and 20-year survival after RT/PCV versus RT alone were 14 years and 37.1% versus 9.3 years and 13.6% (HR 0.60, 95% CI (0.35, 1.03), unadjusted p=0.06). Twenty year PFS in 1p/19q codel was 31.3% in RT/PCV treated patients and 10.8% in RT only treated patients (HR 0.49, 95% CI (0.29, 0.83), unadjusted p=0.007). In the 1p/19q codel subgroup age, WHO PS and necrosis at pathology were identified to be of independent prognostic value for OS. CONCLUSION This long term analysis confirms the earlier conclusions and provides data on long term survival in this patient group. In 1p/19q codel patients treated with RT/PCV, the 20-year PFS and OS rates are 31% and 37% respectively.

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Long-Term Survival Is Comparable between Palifermin-Treated and Placebo-Treated Patients (Pts) with Hematologic Malignancies (HM) Undergoing High-Dose Chemotherapy and Total Body Irradiation Followed by Autologous Hematopoietic Stem Cell Transplantation (HSCT).

Blood ◽

10.1182/blood.v106.11.2925.2925 ◽

2005 ◽

Vol 106 (11) ◽

pp. 2925-2925 ◽

Cited By ~ 1

Author(s):

Ricardo Spielberger ◽

Christos Emmanouilides ◽

William Bensinger ◽

Alan Rong ◽

Alessandra Cesano ◽

...

Keyword(s):

Controlled Study ◽

Secondary Malignancies ◽

Double Blind ◽

Term Survival ◽

Hematopoietic Stem ◽

Double Blind Placebo ◽

Disease Outcomes ◽

Long Term Follow Up

Abstract Oral mucositis (OM) is a severely debilitating side effect of chemoradiotherapy that often causes significant pain, diminished quality of life, and increased risk of infections. Palifermin decreases the incidence and duration of severe OM in HM pts receiving myelotoxic therapy and HSCT. Palifermin safety and efficacy have not been established in the non-HM setting. Since the rHuKGF receptor is not expressed by HM, palifermin is not expected to interfere with long-term disease outcomes in this pt population. Aim: We assessed palifermin’s effects on the long-term disease outcomes (survival, disease progression, secondary malignancies) in pts with HM. Methods: Long-term follow-up data were pooled from a 1998 phase 2, double-blind, placebo-controlled study (n=86) and a 2000 double-blind, placebo-controlled phase 3 study (n=212). The analysis included 152 pts treated with palifermin and 146 pts treated with placebo. Pts were assessed at 6-month intervals during the first year and annually thereafter until death or loss to follow-up. The Kaplan-Meier (K–M) method provided estimates of the safety endpoints. Data reported here are as of August 2004. Results: There were 298 pts (152 palifermin: 146 placebo) monitored for long-term follow-up. The median follow-up period was 23.3 months for palifermin and 23.5 months for placebo. The overall survival and progression-free survival curves (p=0.474 and p=0.253, respectively) are similar between the palifermin and placebo groups. Secondary malignancies occurred in only 6 of 152 (3%) palifermin and 5 of 146 (4%) placebo pts. All secondary malignancies were myelodysplastic syndromes: 9 patients with diagnoses of Non-Hodgkin’s lymphoma and 2 patients of Hodgkin’s Disease. The number of deaths was similar between the groups (30% palifermin; 27% placebo); most deaths occurred within 12 months of randomization and were attributable to the underlying HM disease. Conclusion: Use of palifermin for the prevention of severe OM has shown no negative impact on long-term disease outcomes, including survival, in the HSCT setting for patients with HM.

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Cetuximab and chemotherapy in the treatment of patients with initially “nonresectable” colorectal (CRC) liver metastases: Long-term follow-up of the CELIM trial.

Journal of Clinical Oncology ◽

10.1200/jco.2013.31.15_suppl.3538 ◽

2013 ◽

Vol 31 (15_suppl) ◽

pp. 3538-3538 ◽

Cited By ~ 2

Author(s):

Gunnar Folprecht ◽

Thomas Gruenberger ◽

Wolf Bechstein ◽

Hans-Rudolf Raab ◽

Juergen Weitz ◽

...

Keyword(s):

Liver Metastases ◽

Tumor Response ◽

Progression Free Survival ◽

R0 Resection ◽

Term Survival ◽

Free Survival ◽

Long Term Follow Up ◽

Clinical Trial Information

3538 Background: CRC liver metastases can be resected after downsizing with intensive chemotherapy schedules, with a strong correlation between the response and resection rates. Cetuximab plus chemotherapy has been shown to increase the rates of tumor response and resection of liver metastases. (Van Cutsem et al, JCO 2011). Methods: Patients (pts) with technically non-resectable and/or with > 4 liver metastases were randomized to treatment with FOLFOX/cetuximab (arm A) or FOLFIRI/cetuximab (arm B) and evaluated regarding resectability every 2 months. Resection was offered to all patients who became resectable during the study. K-ras and b-raf status were retrospectively evaluated. Data on tumor response and resection were reported earlier (Folprecht et al, Lancet Oncol 2010). Overall and progression free survival were analyzed in December 2012. Results: Between Dec 2004 and March 2008, 56 pts were randomized to arm A, 55 to arm B. For the current analysis, 109 pts were evaluable for overall survival (OS), and 106 patients for PFS. The median OS was 35.7 [95% CI: 27.2-44.2] months (arm A: 35.8 [28.1-43.6], arm B: 29.0 [16.0-41.9], HR 1.03 [0.66-1.61], p=0.9). The median PFS was 10.8 [9.3-12.2] months (Arm A: 11.2 [7.2-15.3], Arm B: 10.5 [8.9-12.2], HR 1.18 [0.79-1.74], p=0.4). Patients with R0 resection had a better OS (median: 53.9 [35.9-71.9] mo) than patients without R0 resection (27.3 [21.1-33.4] mo, p=0.002) and a better PFS (median 15.4 [11.4-19.5] and 8.9 [6.7-11.1] mo in R0 resected and not R0 resected pts, p<0.001). The 5 year survival in R0 resected patients is 46.2% [29.5-62.9%]. Conclusions: This study confirmed a favourable long term survival of patients with initially “nonresectable” CRC liver metastases treated in a multidisciplinary approach of neoadjuvant chemotherapy with cetuximab and subsequent metastasectomy in pts who became resectable. Clinical trial information: NCT00153998. [Table: see text]

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Precision medicine: Clinical outcomes including long-term survival according to the pathway targeted and treatment period–The IMPACT study.

Journal of Clinical Oncology ◽

10.1200/jco.2018.36.18_suppl.lba2553 ◽

2018 ◽

Vol 36 (18_suppl) ◽

pp. LBA2553-LBA2553 ◽

Cited By ~ 9

Author(s):

Apostolia Maria Tsimberidou ◽

David S. Hong ◽

Jennifer J. Wheler ◽

Gerald Steven Falchook ◽

Aung Naing ◽

...

Keyword(s):

Liver Metastases ◽

Single Agent ◽

Pi3k Pathway ◽

Molecular Testing ◽

Term Survival ◽

Starting Point ◽

Long Term Follow Up ◽

The Impact

LBA2553 Background: We evaluated the impact of pathway targeted and long-term follow-up of patients (pts) with refractory cancers referred to phase I trials. Methods: Pts referred to our program (2007-2013) had CLIA molecular testing. Pts treated with matched targeted therapy (MTT) vs. non-matched therapy (NMT) were analyzed. Results: Of 3,743 pts who had testing, 1,307 had ≥1 alteration and received therapy (MTT 711, NMT 596): med. age 57 yrs, range 16-86; 39% men; med. no. of prior therapies 4, range 0-16. The most common tumors were gastrointestinal 24.2%, gynecologic 19.4%, breast 13.5%, melanoma 11.9%, and lung 8.7%. Targeting MEK/RAF and RET pathways correlated with higher rates of CR/PR/SD≥6 months (mos), PFS and OS compared to others (all P < .001) (Table). Plateau was noted in OS (start, 38 mos): 74 of 711 (10.4%) in the MTT (max 10.7+ yrs) vs. 24 of 596 (4%) in the NMT (max 6 yrs) group were alive (p < .0001). In the MTT group, factors predicting longer PFS were non-PI3K pathway MTT (p < .001), no liver metastases (p < .001), PS < 2 (p = .006), normal LDH (p < .001) and albumin (p = .01) levels, and non-single agent therapy (p = .02). Factors predicting longer OS were non-PI3K pathway MTT (p < .001), no liver metastases (p < .001), PS < 2 (p < .001), normal LDH (p < .001) and albumin (p = .001) levels, and normal PLT counts (p = .03). Conclusions: Outcomes were superior in pts matched to RET and MEK/RAF inhibitors. Factors predicting longer PFS and OS were identified. In the MTT group, 10.4% of patients had OS ≥ 38 mos, the plateau starting point. Clinical trial information: NCT00851032. [Table: see text]

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Jeune syndrome with renal failure

Srpski arhiv za celokupno lekarstvo ◽

10.2298/sarh160218027p ◽

2017 ◽

Vol 145 (3-4) ◽

pp. 147-152

Author(s):

Amira Peco-Antic ◽

Mirjana Kostic ◽

Brankica Spasojevic ◽

Gordana Milosevski-Lomic ◽

Dusan Paripovic ◽

...

Keyword(s):

Renal Failure ◽

Growth Failure ◽

Term Survival ◽

Jeune Syndrome ◽

Long Term Follow Up ◽

The University ◽

Age Range

Introduction/Objective. Jeune syndrome (JS) is a rare hereditary ciliopathy characterized by asphyxiating thoracic dystrophy, shortened limbs and brachydactyly. Extraskeletal anomalies such as chronic renal failure (CRF), hepatic fibrosis, and retinitis pigmentosa may be a part of the JATD phenotype. The aim of this study is to present long-term follow-up of JS patients with early progressive kidney disease. Methods. This is a retrospective study of pediatric patients with JS and CRF who were treated at the University Children?s Hospital between January 1980 and December 2014. The patients? data were retrospectively reviewed from the medical records. Results. There were thirteen patients from 11 families, five girls and eight boys mean aged 4.3 years at the time of diagnosis. All of the patients had characteristic skeletal findings, retinal degeneration and an early onset of CRF at age range from 1.5 to 7 years. Five patients had neonatal respiratory distress and congenital liver fibrosis was diagnosed in five patients. One patient died due to complications of CRF, while others survived during follow-up of mean 11 years. IFT140 mutations were found in four genetically tested patients. Conclusion. The average incidence rate of JS with renal phenotype in Serbia was about 0.2 per one million of child population. Long-term survival of JS patients depends on renal replacement therapy, while skeletal dysplasia, growth failure, respiratory and eyes problems have impact on the patients? quality of life.

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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

Frontiers in Oncology ◽

10.3389/fonc.2021.720044 ◽

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<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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Long-term survival and toxicity in patients with progressive advanced neuroendocrine tumors treated with lutetium peptide radiolabelled radiotherapy: A Western Australian long-term follow-up study.

Journal of Clinical Oncology ◽

10.1200/jco.2021.39.15_suppl.e16202 ◽

2021 ◽

Vol 39 (15_suppl) ◽

pp. e16202-e16202

Author(s):

Kim Robyn Kennedy ◽

Phillip Claringbold ◽

William Macdonald ◽

Glenn Boardman ◽

David Turner Ransom ◽

...

Keyword(s):

Overall Survival ◽

Neuroendocrine Tumours ◽

Progression Free Survival ◽

Somatostatin Analogue ◽

Term Survival ◽

Free Survival ◽

Follow Up Study ◽

Long Term Follow Up

e16202 Background: There are limited treatment options for advanced neuroendocrine tumours, and radiolabelled somatostatin analogues have shown favourable safety and efficacy over other existing treatments. Lutetium Octreotate has been shown to be the somatostatin analogue of choice in Peptide Radiolabelled Radiotherapy (PRRT) for advanced neuroendocrine tumours (NETs). Methods: We conducted a retrospective review of the long term safety and survival outcomes of 104 patients prospectively treated on the CLEMENT1, CLEMENT2, NETTLE, and NETT VALuE trials where patients with advanced progressive NETs were treated with Lutetium Octreotate PRRT in Perth, Western Australia. With a median follow-up time of 68 months, this is the longest follow-up study of advanced NETs treated with Lutetium PRRT in the literature to date. Results: Results showed comparable periods of disease stability as other studies, with median progression free survival of 43 months, and superior survival to other series, with a median survival of 71 months. There were patients who had very durable responses, with five year overall survival 61.5%, five year progression free survival 30.1%, 10 year overall survival 30.1%, and 10 year progression free survival of 29.3%, demonstrating Lu 177 can provide a very long duration of response in some patients. PRRT treatment was well tolerated with 1.9% of patients suffering long term renal impairment, and 1% with long term mild thrombocytopenia attributed to PRRT. Importantly, there was a higher rate of MDS and leukaemia in our series (6.7%), which is likely attributed to the longer period of follow-up with all except one case occurring 48 months after PRRT treatment, which is later than the median follow up in most other studies. Conclusions: Overall, this study showed that Lutetium PRRT remains an efficacious and well tolerated treatment in long term follow-up. For clinicians deciding on the timing of PRRT for individual patients the 6.7% long term risk of MDS/leukaemia needs to be balanced against the 29.3% PFS at 10 years. Clinical trial information: ACTRN12610000440022.

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