Prevalence and significance of clonal hematopoiesis of indeterminate prognosis (CHIP) in multiple myeloma.

2020 ◽  
Vol 38 (15_suppl) ◽  
pp. 8542-8542
Author(s):  
Leslie Jane Padrnos ◽  
Betsy LaPlant ◽  
Shaji Kumar ◽  
Kimbery Henderson ◽  
Angela Dispenzieri ◽  
...  
Keyword(s):  
Multiple Myeloma ◽  
Small Sample Size ◽  
Gene Mutations ◽  
Small Sample ◽  
Common Finding ◽  
Secondary Malignancy ◽  
Hematopoietic Stem ◽  
Clonal Hematopoiesis ◽  
Significant Difference

8542 Background: Clonal hematopoiesis of indeterminate potential (CHIP) is defined by the presence of leukemia-associated somatic mutations in clonally expanded hematopoietic stem cells with a VAF of ≥2%. CHIP has been associated with myeloid malignancies and increased all cause mortality largely from atherovascular disease. The prevalence and impact of CHIP in patients with myeloma (MM) is yet to be defined. Aims: Evaluate the prevalence and significance of CHIP in 101 lenalidomide exposed patients with multiple myeloma. Methods: 101 MM patients, the majority exposed to > 2 years of lenalidomide (Len), with stored mononuclear blood samples were identified for Next Generation Sequencing (NGS) using a panel encompassing 42 gene mutations associated with CHIP. Electronic medical records were reviewed and outcomes of secondary malignancy, macrocytosis, thrombosis and mortality were extracted. Results: Thirty of 101patients were found to have CHIP with the most frequent mutations: DNMT3A (12%), TET2 (5%), and TP53 (4%). One third of patients with CHIP had > 1 mutation (37%). At 68 months median follow up over a quarter of patients experienced thrombosis (31%) and 13% developed subsequent malignancy/premalignant condition including myelodysplastic syndrome (3%). There was no significant difference in age, gender, duration of Len prior to NGS testing, thrombosis complication, or survival in those with versus without a CHIP mutation. At last F/U 30% had died, 25% remained on therapy, 27% were on maintenance therapy and 11% were on observation alone. Conclusions: CHIP mutations are common in plasma cell neoplasms, with 30% of patients in this study retrospectively found to have CHIP mutations by NGS testing. CHIP mutations have been associated with cardiovascular events and malignancy development, however no associations were identified in this small study. Limitations to this study include small sample size, retrospective nature and duration of follow up. Further study of this very common finding in MM patients regarding timing of development and subsequent impact of these mutations could help risk stratify and inform therapy selection. [Table: see text]

Outcome of Patients with IgD and IgM Multiple Myeloma Undergoing Autologous Hematopoietic Stem Cell Transplants: A Retrospective CIBMTR Study.

Blood ◽  
2008 ◽  
Vol 112 (11) ◽  
pp. 3328-3328
Author(s):  
Donna E. Reece ◽  
David H. Vesole ◽  
Smriti Shrestha ◽  
Angela Dispenzieri ◽  
Gustavo Milone ◽  
...  
Keyword(s):  
Multiple Myeloma ◽  
Stem Cell ◽  
Hematopoietic Stem Cell ◽  
Small Sample Size ◽  
Conditioning Regimen ◽  
Progression Free Survival ◽  
Marrow Transplant ◽  
Small Sample ◽  
Hematopoietic Stem ◽  
Prior Therapy

Abstract Multiple Myeloma (MM) is the most common indication for autologous hematopoietic stem cell transplantation (auto HCT). However, little information is available regarding the outcome of patients (pts) with the rarer immunoglobulin subtypes IgD and IgM, which represent 2% and 0.5%, respectively, of all MM cases. In addition, IgD MM has been reported previously to have a poorer prognosis, at least after conventional therapy. The CIBMTR conducted a retrospective analysis of MM pts transplanted between 1995–2005 to describe the characteristics and results of auto HCT in IgD (n=36) and IgM (n=11) among 3578 MM patients with auto HCT during this period. Median follow-up of survivors was 41 (range 2–130) months for pts with IgD and 58 (range 5–101) months for those with IgM MM. Among pts with IgD subtype, median age was 52 years (yrs), 67% were male, 36% had a creatinine >2 mg/L, 61% had Durie-Salmon stage III disease at diagnosis and 33% had Kappa Serum Light chain; the corresponding values for IgM pts were 58 yrs, 36%, 73% and 55% respectively. Prior to auto HCT, 75% of IgD pts were chemosensitive and 25% had received >2 lines of chemotherapy, while all IgM pts were chemosensitive and none had received more than 2 lines of prior therapy. Median time from diagnosis to auto HCT was 9 months in both subtypes. The most common conditioning regimen was singleagent melphalan, and all but 1 pt with IgD disease were grafted with blood stem cells. The small sample size precluded multivariate analysis for potential prognostic factors for outcome. Below table summarizes the post-auto HCT results in these pts contrasted with a reference pool of IgG and IgA MM receiving auto HCT in the same time period. Outcomes, probability (95% CI) IgD IgM IgG/IgA (n=36) (n=11) (n=1475) 100-day mortality, % 0 9 ( 0 – 32) 7 (5 – 8) Non MM deaths, % @ 1 yr 0 9 (0 – 32) 5 (4 – 7) @ 3 yrs 3 (0 – 13) 21 (2 – 51) 16 (14 – 18) Relapse/ Progression, % @ 1 yr 21 (9 – 37) 20 (2 – 49) 18 (16 – 20) @ 3 yrs 59 (41 – 76) 32 (8 – 64) 36 (34 – 39) Progression-free survival, % @ 1 yr 79 (63 – 91) 71 (41 – 93) 77 (74 – 79) @ 3 yrs 38 (21 – 56) 47 (17 – 78) 47 (44 – 50) Overall survival, % @ 1 yr 87 (74 – 97) 91 (68 – 100) 79 (77 – 85) @ 3 yrs 69 (51 – 84) 68 (36 – 93) 53 (50 – 57) No striking differences are apparent in the post auto HCT outcomes of patients with IgD and IgM MM. These results are also consistent with published outcomes of pts with IgD/ IgM MM (Wechaleker et al Ann Hematol. 2005 Feb; 84(2):115–7; Maisner et al Bone Marrow Transplant. 2008 Jan; 41(1):51–4).

Differential Clinical Impact Of Gene Mutations and Their Combinations In Primary Cytogenetically Normal Acute Myeloid Leukemia (CN-AML)

Blood ◽  
2013 ◽  
Vol 122 (21) ◽  
pp. 2540-2540
Author(s):  
Dimitrios Papaioannou ◽  
Kati Maharry ◽  
Krzysztof Mrózek ◽  
Heiko Becker ◽  
Klaus H. Metzeler ◽  
...  
Keyword(s):  
Annual Meeting ◽  
Small Sample Size ◽  
Prognostic Significance ◽  
Age Groups ◽  
Gene Mutations ◽  
Small Sample ◽  
Clinical Impact ◽  
Cebpa Mutations ◽  
American Society

Abstract In CN-AML, mutations of specific genes have prognostic significance and are used for treatment guidance. However, these mutations are in general not mutually exclusive and CN-AML patients (pts) frequently harbor mutational combinations with unclear prognostic significance because of the concurrent presence of mutations with favorable (CEBPA, NPM1) and unfavorable (ASXL1, DNMT3A, IDH1, IDH2, FLT3-ITD, MLL-PTD, RUNX1, TET2, WT1) impact on outcome. Here, we report the frequency and clinical significance of isolated mutations and their combinations in 364 CN-AML pts. Younger [<60 years (y); n=163] and older (≥60 y; n=201) pts were intensively treated on frontline CALGB/Alliance protocols and, per protocol, did not undergo allogeneic stem cell transplantation in first complete remission (CR). Median follow-up for pts alive was 8.7 y. Pts were analyzed centrally for mutations in the ASXL1, CEBPA, DNMT3A, IDH1, IDH2, NPM1, RUNX1, TET2, and WT1 genes and the presence of FLT3-ITD, FLT3-TKD and MLL-PTD. We found that 99% of pts had ≥1 and 88% ≥2 of the 12 mutations. Specifically, 11% of pts had 1 gene mutated (n1), 40% had 2 (n2), 31% 3 (n3), and 18% harbored ≥4 mutated genes (n4). The distribution of the different n groups in younger and older pts was similar (Fig 1). The expected frequency of each mutated gene in every numerical (n) group was calculated as follows: [no. of pts with the mutated gene] x [total no. of mutations in the n group] / [total no. of mutations in all pts], and compared with the observed frequency. CEBPA, IDH2, NPM1, and RUNX1 mutations were overrepresented in the n1 and n2 groups (P<.001, P<.001, P=.002, P=.02, respectively) and thus likely represent “early” events in leukemogenesis. In contrast, DNMT3A, FLT3-ITD, and TET2 mutations were more frequent in the n3 and n4 groups (P=.008, P<.001, P=.02, respectively) suggesting that they are likely “late” events. The distribution among age groups and clinical impact of the “early” mutations (i.e., CEBPA, NPM1, IDH2) presenting as isolated mutations or in combination with other mutations were then investigated. Isolated RUNX1 mutations could not be evaluated due to small sample size. Younger pts were more likely to harbor an isolated “early” mutated gene than older pts (21% v 12%, P=.03). Specifically, isolated CEBPA and NPM1 mutations were more frequent in younger pts compared with the older (7% v 2%, P=.007; 7% v 3%, P=.07, respectively). In pts with isolated CEBPA mutations, these were always bi-allelic. Single “early” mutated genes varied in their impact on CR achievement (CEBPA: 100%, NPM1: 94%, IDH2: 67%), and overall survival (OS; 3-y OS: CEBPA: 87%, NPM1: 69%, IDH2: 22%). Acquisition of additional mutations adversely modified the prognostic significance of favorable “early” mutations in the CEBPA (Fig 2A) and NPM1 (Fig 2B) genes, but left unchanged the unfavorable effect of IDH2 mutations (Fig 2C). Two mutational combinations were exceptions to this: younger pts with both NPM1 and DNMT3A mutations (8 pts) had an excellent prognosis with 3-y OS of 100%; and older pts with concurrent NPM1 and IDH2 mutations (9 pts; all IDH2 mutations were in codon R140) had a high 3-y OS rate of 67%. We conclude that CEBPA and NPM1 mutations mainly define pts with favorable prognosis when they present as single markers, whereas mutated IDH2 predominantly associates with adverse outcome. The NPM1+DNMT3A and NPM1+IDH2 mutational patterns may define novel favorable molecular subtypes in younger and older CN-AML pts, respectively, but these results require corroboration in larger pt cohorts. Disclosures: Mrózek: AMERICAN SOCIETY OF HEMATOLOGY: I will review abstracls submitted for presentation at the 55th ASH Annual Meeting in the 611 category Other. Eisfeld:AMERICAN SOCIETY OF HEMATOLOGY: I will review abstracls submitted for presentation at the 55th ASH Annual Meeting in the 608 category Other.

scholarly journals Is stereotactic radiotherapy equivalent to metastasectomy in patients with pulmonary oligometastases?

2019 ◽  
Vol 29 (4) ◽  
pp. 544-550 ◽  
Author(s):  
Shuangjiang Li ◽  
Shihong Nie ◽  
Zhiping Li ◽  
Guowei Che
Keyword(s):  
Small Sample Size ◽  
Disease Free Survival ◽  
Small Sample ◽  
Treatment Groups ◽  
Evidence Strength ◽  
Significant Difference ◽  
Choice Of Treatment ◽  
Size Heterogeneity ◽  
Study Type

Summary A best evidence topic in thoracic surgery was written according to a structured protocol. The question addressed was whether stereotactic body radiotherapy (SBRT) was equivalent to metastasectomy in patients with pulmonary oligometastases arising from solid tumours. Altogether, 1612 papers were found using the reported search, of which 5 cohort studies derived from 4 patient populations represented the best evidence to answer the clinical question. The authors, journal, date and country of publication, patient group studied, study type, relevant outcomes and results of these papers are tabulated. All 5 studies demonstrated no significant difference in post-treatment overall survival, disease-free survival or local control between SBRT and metastasectomy for pulmonary oligometastases. One of the 5 studies showed a significantly decreased rate of severe complications among the patients treated with SBRT. The other papers reported higher rates of complications in the SBRT groups, invariably due to radiation, but with uncertain clinical significance. The evidence strength of these findings may be largely attenuated due to the small sample size, heterogeneity of SBRT protocols and incomparable follow-up periods between the 2 treatment groups. The selection criteria for the choice of treatment were not stated. We conclude, based on limited evidence, that SBRT has equivalent outcomes to metastasectomy in the treatment of patients with pulmonary oligometastases.

Abstract 19483: Assessment of Outcome in Patients With Acute Minor Ischemic Stroke Treated With Intravenous TPA

Circulation ◽  
2014 ◽  
Vol 130 (suppl_2) ◽  
Author(s):  
Sameer Sharma ◽  
Umair Afzal ◽  
Mubashir Pervez ◽  
Rochele Clark ◽  
Julius G Latorre ◽  
...  
Keyword(s):  
Acute Stroke ◽  
Small Sample Size ◽  
Intervention Group ◽  
Small Sample ◽  
Minor Stroke ◽  
Good Outcome ◽  
Minor Ischemic Stroke ◽  
Significant Difference ◽  
Iv Tpa

Introduction: Minor acute stroke (NIHSS≤4) within 4.5 hours from symptom onset is a common reason for withholding intravenous (iv) Thrombolysis (TPA), due to potential risk of major bleeding with such treatment and assumed good outcome without intervention. This subgroup of patients was excluded from the landmark NINDS iv tPA trial as per the prespecified protocol and also from various recent clinical trials involving acute stroke. In a recent study of patients with Rapid Improving symptoms and Minor stroke who did not receive IV tPA, 28.3% could not be discharged home and 28.5% could not ambulate independently at the time of discharge (Smith et al 2011). The efficacy of iv TPA in Minor stroke has not been previously studied. Method: Retrospective review of consecutive patients with Minor stroke (NIHSS ≤4) arriving within 4.5 hours between January 2009-July 2013 was done. Outcome in patients who received IV TPA was compared with patients who did not receive any IV tPA. Good outcome was defined as mRS ≤2. Results: 186 patients were identified out of which 20 received iv tPA. The baseline median NIHSS was 2 in the non-intervention group vs 3 in the intervention group (p =0.001), more cardioembolic, cryptogenic and lacunar stroke in tPA group (40% vs 35.53%, 20% vs 14.46% and 30% vs 22.89% respectively) there was no other statistically significant difference between the baseline characteristics of the two groups. Median change in NIHSS from admission to discharge was 1 for non-tPA vs 2.5 for tPA(p<0.001) and good outcome at discharge was seen in 80% patients in tPA vs 69.28% in non-tpa group (p =0.321). 8-12 week follow up data was available for 100 patients (12 tPA patients). Mean mRS was 1.34 in non-tPA vs 1 in tPA group (p=0.430) Conclusion: Acute intervention in Minor stroke appears to be safe. We did not find any statistically significant difference in clinical outcome between the two groups; this is likely due to small sample size, short follow-up period, and other confounding factors that we cannot fully account for in a retrospective study. A prospective randomized control study is warranted to clearly delineate the effect of iv TPA in patients with Minor stroke.

Elevated Neutrophil: Lymphocyte Ratio Does Not Predict Survival In Multiple Myeloma Patients

Blood ◽  
2013 ◽  
Vol 122 (21) ◽  
pp. 5336-5336
Author(s):  
Jean Paul Atallah ◽  
Basem Azab ◽  
Abhirami Vivekanandarajah ◽  
Ali Naboush ◽  
Houssein Abdul Sater ◽  
...  
Keyword(s):  
Multiple Myeloma ◽  
Conflicts Of Interest ◽  
Small Sample Size ◽  
Predictive Ability ◽  
Continuous Variable ◽  
Small Sample ◽  
University Hospital ◽  
Neutrophil Lymphocyte Ratio ◽  
Lymphocyte Ratio ◽  
Significant Difference

Abstract Background Cancer associated inflammation is one of the key determinants of outcome in patients with cancer. An elevated neutrophil: lymphocyte ratio (NLR) has been identified as a predictor of worse survival in patients with various solid tumors compared to hematologic malignancies but no reports yet examined its impact on multiple myeloma. The aim of this study was to examine the prognostic value of an elevated NLR in multiple myeloma. Methods We had approval by our institutional review board to collect the data on patients diagnosed with multiple myeloma at Staten Island University Hospital between year 2000 and 2012 identified from our local cancer database. Data on demographics, conventional prognostic markers, laboratory analyzes including blood count results, and histopathology were collected and analyzed. A cox proportional survival analysis was carried out to assess the relationship between NLR and mortality. NLR was assessed as a continuous variable as well as categorical variable (quartile 0.5-1.5, 1.6-2.2, 2.3-3.8, and 3.9-22.3). Results A total of 96 patients were identified with a median age at diagnosis of 70 (IQR of 61 to 79) years. The median neutrophil count was 3.5 (2.5—5.1) x 10-9/liters, median lymphocyte count 1.5 (1.05-2.4) × 10-9/liters, while the NLR was 2.28 (1.53-3.88). The median overall survival was 147.5 weeks, IQR (88.5-320). NLR did not prove to be a significant predictor of death as a continuous variable (0.95 (0.85-1.06), p =0.35). Furthermore, there was no significant difference in survival with any of the quartiles of NLR. Compare to lowest quartile of NLR, Hazards ratio for the consecutive quartiles were 1.25 (0.56-2.79, p 0.55), 1.36 (0.61-3.04, p=0.45) and 0.89 (0.36-2.22, p=0.80). Conclusion NLR does not appear to offer useful predictive ability for outcome and survival in multiple myeloma patients. Our study is limited with small sample size, further studies are needed to validate our results. Disclosures: No relevant conflicts of interest to declare.

scholarly journals Cervical Disc Replacement: Trends, Costs, and Complications

2020 ◽  
Vol 14 (5) ◽  
pp. 647-654 ◽  
Author(s):  
Nickul Saral Jain ◽  
Ailene Nguyen ◽  
Blake Formanek ◽  
Ram Alluri ◽  
Zorica Buser ◽  
...  
Keyword(s):  
Small Sample Size ◽  
National Level ◽  
The United States ◽  
Small Sample ◽  
Disc Replacement ◽  
Cervical Disc ◽  
Cervical Disc Replacement ◽  
Significant Difference ◽  
Insurance Database

Study Design: Retrospective review of insurance database.Purpose: To investigate national trends, complications, and costs after cervical disc replacement (CDR) using an administrative insurance database representative of the United States population.Overview of Literature: As CDR continues to be used to treat patients with cervical stenosis, it is important to gain a better understanding of its use on a national level, potential complications, and cost. This information will allow for optimal patient counseling, risk stratification, and healthcare cost assessments. Several prior studies have investigated complications associated with CDR, but they have been limited by small sample size, single institution experiences, limited follow-up, and potential conflicts of interest.Methods: Patients who underwent single or multilevel CDR between 2007 and 2015 were identified using an insurance database. We collected data on annual trends, reimbursement costs, patient demographic information, hospital information, and information on complications from the time of operation to 1 year postoperative.Results: Total of 293 patients underwent either single or multilevel CDR. The number of procedures increased nonlinearly over time at an average of 17% per year, with a greater increase seen in the outpatient setting. Less than 3.7% of patients had new onset pain within 1 year after CDR. Within 1 year, 12.3% of patients reported a mechanical and/or bone-related complication. There were no patients who indicated a new nerve injury within 6 months of follow-up. Less than 3.7% of patients presented with dysphagia or dysphonia within 6 months, infection within 3 months, or a revision or reoperation within 1 year. Average reimbursement for single-level inpatient versus outpatient CDR was US $33,696.28 and US $34,675.12, respectively (p =0.29).Conclusions: This study demonstrated that the use of CDR continued to increase. The most common complication was mechanical and/or bone-related, and cost analysis demonstrated no significant difference between inpatient and outpatient CDR.

scholarly journals P140 As we move to using short-acting drugs for the treatment of RA in the time of a pandemic, does baricitinib live up to its trial data in clinical practice?

Rheumatology ◽  
2021 ◽  
Vol 60 (Supplement_1) ◽  
Author(s):  
Henry J S Vardon ◽  
Karen M J Douglas
Keyword(s):  
Clinical Practice ◽  
Tyrosine Kinases ◽  
Disease Duration ◽  
Small Sample Size ◽  
Small Sample ◽  
Real World Data ◽  
Short Acting ◽  
Serious Infections ◽  
Significant Difference

Abstract Background/Aims  Baricitinib is an oral, reversible and selective inhibitor of JAK1 and JAK2 tyrosine kinases. It was approved for use in 2017 by NICE for the treatment of moderate to severe rheumatoid arthritis (RA). Considering the current risk of COVID-19, the BSR have advocated the use of short-acting drugs such as baricitinib when escalating treatment in RA. As real-world data is limited, we aimed to explore the efficacy of baricitinib in clinical practice. Methods  Observational data was collected retrospectively for patients at the Dudley Group NHSFT with RA (ACR/EULAR criteria) who had received at least one dose of baricitinib prior to 1st October 2019, with a follow up period to 1st October 2020. Patients were identified from a local biologics database. Further data was identified from patients’ medical records including, demographics, features of RA, previous RA therapy history and disease activity scores (DAS28) at 0, 6 and 12 months. Data was input into an Excel spreadsheet with subsequent analysis conducted using SPSS Version26. Results  We identified 26 RA patients (77% female) treated with baricitinib; mean age 61.6 (SD 14.6) years and median disease duration of 12.1 (IQR 5.8-18.4) years. Rheumatoid factor and anti-CCP antibody were positive in 73% and 65% respectively. 35% (n = 9) of patients were biologically naïve, in whom baricitinib was chosen due to needle-phobia (n = 7), or where anti-TNF drugs were considered inappropriate (bronchiectasis, ANA positivity). Mean DAS28 (SD) scores at baseline, 6 and 12 months were 5.9(0.8), 2.8(0.9) and 2.7(1.3) respectively, with significant reduction from baseline to both 6 and 12 months (P &lt; 0.001). A drop of ≥ 1.2 in DAS28 was recorded in 94% of patients with complete data at 6 months (n = 18, 4 missing, 4 discontinued). At 6 and 12 months, 85% and 81% of patients remained on Baricitinib. In total five patients discontinued Baricitinib due to side effects or tolerability issues. Reasons for discontinuation did not include thromboembolic events, zoster or serious infections. When comparing naïve and non-naïve groups, there was no significant difference in age, sex or disease duration. The number of previous biologics used by patients were 1(n = 6), 2(n = 3), ≥3(n = 8). Biologically naive compared to non-naïve patients had a higher DAS28 at baseline, (Mean [SD]) (6.2[0.9] versus 5.7[0.8] NS) but lower at 6 months (2.1[1.6] versus 3.1[1.1] P = 0.023) and greater DAS improvement at 6months (-4.4[1.2] versus -2.5[0.9] P &lt; 0.002). Conclusion  We observed that up to 94% of patients responded to baricitinib with a mean DAS improvement at 6 months of -3.1, biologic naïve patients doing best. Drug survival at 12 months was 81%. These trends are comparable to findings in clinical trials. However, due to our small sample size, the findings are vulnerable to type 1 and 2 errors and should be interpreted with caution. Disclosure  H.J.S. Vardon: None. K.M.J. Douglas: None.

Reference intervals for thyroid-stimulating hormone (TSH) and free thyroxine (FT4) in infants’ day 14–30 of life and a comparison with other studies

2020 ◽  
Vol 33 (9) ◽  
pp. 1125-1132
Author(s):  
Jeanne Sze Lyn Wong ◽  
Nalini M. Selveindran ◽  
Rashdan Zaki Mohamed ◽  
Fuziah M. Zain ◽  
Siti S. Anas ◽  
...  
Keyword(s):  
Thyroid Function ◽  
Small Sample Size ◽  
Age Groups ◽  
Reference Intervals ◽  
Thyroid Stimulating Hormone ◽  
Small Sample ◽  
Free Thyroxine ◽  
Significant Difference ◽  
Stimulating Hormone

AbstractObjectivesEstablished reference intervals of thyroid function in neonates are important; however, studies often consist of a small sample size or lack of clinical information. We aim to define reference intervals for thyroid-stimulating hormone (TSH) and free thyroxine (FT4) for infants aged 14–30 days. We also reviewed follow-up TSH for infants with initial values 10–20 mIU/L.MethodsVenous TSH and FT4 of term babies aged 14–30 days with breast milk jaundice that had thyroid function test performed as part of a prolonged jaundice workout from September 2016 to March 2017 were analyzed. Electronic medical records were reviewed to ensure only well babies with no pathological causes of jaundice or conditions that may affect thyroid function were included. TSH and FT4 were analyzed using immunoassay analyzer Dxl 800, Beckman Coulter.ResultsThere were no correlations between FT4 and TSH with gender, birth weight and ethnicity. Correlation coefficient between FT4 and total bilirubin was weak at 0.138 (p=0.001). No association was found between TSH and bilirubin levels. Mean FT4 was higher in the younger age group day 14–21 (p<0.01). There was no significant difference in TSH values between the age groups. Infants with mildly elevated TSH 10–20 mIU/L had normalized values on follow-up (mean, 11.41 vs. 4.42 mIU/L; p<0.01; 95%CI, 5.88–8.09). The following reference intervals (2.5–97.5th percentile) were derived: FT4 day 14–21 (n=513): 11.59–21.00 pmoL/L; FT4 day 22–30 (n=66): 10.14–19.60 pmoL/L; TSH day 14–30 (n=579): 1.90–10.34 mIU/L. Comparison between studies showed variations of reference intervals with different manufacturer assays, age and methodology.ConclusionsOur reference intervals would be useful in the clinical setting. Infants with mildly elevated TSH could be monitored first instead of immediate treatment.

scholarly journals The Long-Term Follow-Up of Patients with Cystine Stones: A Single-Center Experience for 13 Years

2021 ◽  
Vol 10 (7) ◽  
pp. 1336
Author(s):  
Toshifumi Takahashi ◽  
Shinya Somiya ◽  
Katsuhiro Ito ◽  
Toru Kanno ◽  
Yoshihito Higashi ◽  
...  
Keyword(s):  
Surgical Intervention ◽  
Median Number ◽  
Small Sample ◽  
Age At Diagnosis ◽  
Surgical Interventions ◽  
Significant Difference ◽  
Long Term Follow Up ◽  
Cystine Stones

Introduction: Cystine stone development is relatively uncommon among patients with urolithiasis, and most studies have reported only on small sample sizes and short follow-up periods. We evaluated clinical courses and treatment outcomes of patients with cystine stones with long-term follow-up at our center. Methods: We retrospectively analyzed 22 patients diagnosed with cystine stones between January 1989 and May 2019. Results: The median follow-up was 160 (range 6–340) months, and the median patient age at diagnosis was 46 (range 12–82) years. All patients underwent surgical interventions at the first visit (4 extracorporeal shockwave lithotripsy, 5 ureteroscopy, and 13 percutaneous nephrolithotripsy). The median number of stone events and surgical interventions per year was 0.45 (range 0–2.6) and 0.19 (range 0–1.3) after initial surgical intervention. The median time to stone events and surgical intervention was 2 years and 3.25 years, respectively. There was a significant difference in time to stone events and second surgical intervention when patients were divided at 50 years of age at diagnosis (p = 0.02, 0.04, respectively). Conclusions: Only age at a diagnosis under 50 was significantly associated with recurrent stone events and intervention. Adequate follow-up and treatment are needed to manage patients with cystine stones safely.

scholarly journals NI-19 Use of 11C-methionine PET for decision of discontinuation of adjuvant chemotherapy with temozolomide

2020 ◽  
Vol 2 (Supplement_3) ◽  
pp. ii14-ii14
Author(s):  
Takaaki Beppu ◽  
Yuichi Sato ◽  
Toshiaki Sasaki ◽  
Kazunori Terasaki ◽  
Kuniaki Ogasawara
Keyword(s):  
Adjuvant Chemotherapy ◽  
Small Sample Size ◽  
Standardized Uptake Value ◽  
Progression Free Survival ◽  
Residual Tumor ◽  
Small Sample ◽  
Tumor Type ◽  
Diffuse Astrocytoma ◽  
Significant Difference ◽  
Met Pet

Abstract Background: The aim was to clarify whether positron emission tomography with 11C-methyl-L-methionine (met-PET) is useful to decide on discontinuation of TMZ-adjuvant therapy in patients with residual diffuse astrocytic tumor. Methods: Subjects were 44 patients with residual tumor comprising 17 with IDH1-mutant diffuse astrocytoma (DA), 13 with IDH1-mutant anaplastic astrocytoma (AA), and 14 with IDH1-wild glioblastoma (GB). All patients received TMZ-adjuvant chemotherapy (median, 12 courses), and whether to discontinue or continue TMZ-adjuvant chemotherapy was decided on the basis of the tumor-to-normal ratio in standardized uptake value from met-PET (T/N); patients with T/N &lt; 1.6 immediately discontinued TMZ, and patients with T/N &gt; 1.6 were either to continued or discontinued TMZ. Progression-free survival (PFS) was compared between patients with T/N &gt; 1.6 and T/N &lt; 1.6 in each tumor type. Median observation period was 434 days after met-PET scanning. Results: The number of patient who underwent recurrence was 10 in DA, 7 in AA, and 11 in GB. All patients showing T/N &gt; 1.6 underwent tumor recurrence. PFS was significantly longer in patients with T/N &lt; 1.6 than T/N &gt; 1.6 in DA and AA (p &lt; 0.01 in both types), but was no significant difference between 2 groups in GB (p = 0.06). Sixteen of 17 patients (94%) in DA and AA showed recurrence from residual tumor, whereas 4 of 11 patients (36%) in GB showed recurrent tumor at remote regions which were different from residual tumor. Conclusions: The present study suggested that met-PET is beneficial to decide to discontinue adjuvant chemotherapy with TMZ in patients with residual tumors of DA and AA, but not useful for patients with GB. Reasons for unsuccessful results in GB might have been small sample size, failure of establishing the cut off value in T/N, recurrences at remote regions where not be assessed by met-PET.

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