scholarly journals Chronic nonbacterial osteomyelitis in children: a multicentre Belgian cohort of 30 children

2021 ◽  
Author(s):  
Sara Kaut ◽  
Ine Van den Wyngaert ◽  
Davy Christiaens ◽  
Carine Wouters ◽  
Nathalie Noppe ◽  
...  
Keyword(s):  
Tertiary Care ◽  
Age At Onset ◽  
Lower Limbs ◽  
Second Line ◽  
Imaging Data ◽  
Second Line Therapy ◽  
Paediatric Cohort ◽  
Leading Symptom ◽  
Mean Time

Abstract BackgroundTo evaluate clinical characteristics, imaging findings, therapeutic approach and outcome of paediatric patients with Chronic Non-Bacterial Osteomyelitis (CNO). MethodsRetrospective review of 30 children diagnosed with CNO at two tertiary care centres in Belgium. Imaging data were evaluated by blinded paediatric radiologists. ResultsMean age at onset was 10.3 years and mean age at diagnosis was 11.7 years. Bone pain was the leading symptom (29/30 patients). Out of 180 symptomatic lesions, 131 were confirmed on MRI as hyperintense geographic lesions on STIR images at the metaphysis and epiphysis adjacent to growth plates of tubular bones. The most common sites of involvement were the lower limbs, spine, sternoclavicular joint and humerus. For nearly half of the patients (14/30) monotherapy with NSAIDs was sufficient to obtain remission. The remaining 16 patients received second-line therapy: bisphosphonates (n=15/30), disease-modifying antirheumatic drugs (n=7/30), etanercept (n=4/30) and tocilizumab (n=1/30). 26/30 Patients reached remission after a mean time of 37.6 months. The prognosis was worse for patients with spinal involvement, resulting in more long-term sequelae. ConclusionsWe present a multicentre paediatric cohort of 30 CNO patients. A typical pattern of bone involvement could be found on MRI. NSAIDs were administered as first-line treatment. Second-line strategies included bisphosphonates, corticosteroids, methotrexate, etanercept and tocilizumab. Trial registrationRetrospectively registered.

Similar Efficacy of Dasatinib and Nilotinib As Second-Line Therapy in Patients with Chronic Phase Chronic Myeloid Leukemia Failing Imatinib: A Retrospective, Real-Life Study

Blood ◽  
2016 ◽  
Vol 128 (22) ◽  
pp. 5434-5434
Author(s):  
Mario Tiribelli ◽  
Massimiliano Bonifacio ◽  
Gianni Binotto ◽  
Alessandra Iurlo ◽  
Francesca Cibien ◽  
...  
Keyword(s):  
Chronic Phase ◽  
Cytogenetic Response ◽  
Similar Efficacy ◽  
Molecular Response ◽  
Second Line ◽  
Second Line Therapy ◽  
Secondary Resistance ◽  
Line Therapy ◽  
Mean Time

Abstract Background. Use of 2nd generation tyrosine kinase inhibitors (2G-TKIs) dasatinib (DAS) and nilotinib (NIL) in chronic phase (CP) chronic myeloid leukemia (CML) patients failing imatinib (IM) results in around 50% of sustained cytogenetic response, and around 40% major molecular response (MMR). However, these are historical data and it's unclear if there's a significant difference in efficacy of the two 2G-TKIs, especially in the long-term. Aims and methods. We retrospectively analysed 163 CP-CML patients resistant or intolerant to IM that received either DAS (n=95) or NIL (n=68) as second-line therapy. We compared the characteristics of the two groups at the time of CML diagnosis and at the time of IM failure, including the cause of switch to 2G-TKI, duration of IM therapy, IM dose escalation and Hammersmith score to predict the probability of response to 2G-TKIs. Cytogenetic and molecular responses were evaluated according to the ELN recommendations. Sustained deep molecular response (DMR) was defined as MR4 or better lasting ≥ 2 years, ongoing at the last contact, and with at least a Q-PCR test every 6 months. Time to treatment failure (TTF) was calculated from the start of 2G-TKI to any of the followings: progression to accelerated or blast phase (ABP), death for any cause at any time, treatment discontinuation for primary or secondary resistance or intolerance. Progression free survival (PFS) was calculated from the start of 2G-TKI to ABP or death. Overall survival (OS) was calculated from the start of 2G-TKI to death. Results. DAS and NIL cohorts were comparable for age, sex and risk score (Sokal and EUTOS) at diagnosis. Median duration of IM therapy was similar (DAS 19 months, NIL 14 months), but 27/95 patients (28%) had IM dose escalation before DAS compared to only 9/68 (13%) before NIL (p=0.03). There was a higher rate of switch to DAS than to NIL for secondary resistance (26/95, 27% vs 7/68, 10%; p=0.01) while more patients changed from IM to NIL due to intolerance (31/68, 46%, vs 21/95, 22% for DAS; p=0.002). Rates of primary resistance did not differ (47/95, 49% for DAS vs 28/68, 41% for NIL; p=0.37), as well as other causes of switch (1/95, 1% for DAS vs 2/68, 3% for NIL; p=0.77). Hammersmith score was almost identical in the two groups. Complete cytogenetic response (CCyR) was attained in 53/73 (73%) patients not in CCyR at the time of DAS start, and in 31/48 (65%) patients not in CCyR at the time of NIL start (p=0.46). Mean time to CCyR was similar (7.1 months for DAS and 5.3 months for NIL; p=0.30). MMR was achieved in 55/89 (65%) patients not in MMR at the time of DAS start and in 39/61 (65%) patients not in MMR at the time of NIL start (p=0.82). Again, mean time to MMR was not different in the DAS e NIL cohorts (12.4 vs. 8.5 months; p=0.14). DMR was obtained in 39/88 (44%) patients not in DMR at the time of DAS start and in 30/65 (46%) patients not in DMR at the time of NIL start (p=0.95). Sustained DMR was evaluable in 127 patients: 37 patients (29%) achieved sustained DMR, without difference between DAS (24/82, 29%) and NIL (13/45, 29%; p=1.00). With a median follow-up of 44 months (range 1-124), 5-year TTF was similar for DAS (65%, 95%CI 52-75%) and NIL (61%, 95%CI 43-74%; p=0.40) [Figure 1a]. Thirty-two of 95 patients (34%) stopped DAS due to toxicity (19/32, 59%), resistance (11/32, 31%) or other causes (3/32, 10%); 22/68 patients (32%) interrupted NIL for toxicity (11/22, 50%), resistance (8/22, 36%) or other causes (3/22, 14%). Probability of survival and progression were almost identical, with a 5-year PFS of 84% (95%CI 68-89%) for DAS and 92% (95%CI 79-97%) for NIL (p=0.27) [Figure 1b] and a 5-year OS of 89% (95%CI 78-95%) and 96% (95%CI 85-99%) (p=0.31), respectively. Conclusions. With the limits of a retrospective analysis, our data suggest similar efficacy of DAS and NIL after IM failure in CP-CML, with rates of cytogenetic and molecular responses higher than those previously reported and excellent long-term survival. Around 30% achieved sustained DMR with second-line therapy, thus being potentially candidate for TKI discontinuation. Disclosures Tiribelli: Bristol-Myers Squibb: Consultancy, Speakers Bureau; Novartis: Consultancy, Speakers Bureau; Ariad Pharmaceuticals: Consultancy, Speakers Bureau. Bonifacio:Ariad Pharmaceuticals: Consultancy; Pfizer: Consultancy; Bristol Myers Squibb: Consultancy; Novartis: Research Funding; Amgen: Consultancy. Fanin:Novartis: Speakers Bureau.

scholarly journals P1826ASSESSMENT OF GROWTH PARAMETERS OF CHILDREN IN LONG TERM FOLLOW UP WITH DIFFERENT RELAPSING COURSE OF NEPHROTIC SYNDROME

2020 ◽  
Vol 35 (Supplement_3) ◽  
Author(s):  
Sonia Sharma
Keyword(s):  
Nephrotic Syndrome ◽  
Growth Parameters ◽  
Tertiary Care ◽  
Age At Onset ◽  
Standard Deviation Score ◽  
Pediatric Nephrology ◽  
Group 2 ◽  
Nephrotic Children

Abstract Background and Aims Childhood Nephrotic syndrome has its peak onset in the age group 2-4 years, and that is also a period of significant height growth. Corticosteroids and chronic diseases are known to have long term effects on growth parameters of these children. Hence we assessed and compared the growth of children with Infrequent relapsing (IFRNS) and remission (R) as group 1, Frequent -relapsing (FR), and steroid - dependent (SD) as group 2 and steroid -resistant (SR) as group 3. Method This retrospective single center study collected data from medical records of children presented in a pediatric nephrology clinic in a tertiary care center in New Delhi. Nephrotic children, aged 1-18 years with regular follow up in the period of 2014 to 2019 were included. Basic demographic details including age at onset and age at last follow up in clinic, sex, anthropometry details at last follow up were recorded. We traced initial height at nephrotic syndrome onset to assess growth velocity but were unavailable at the moment so excluded from analysis. Also, children completed less than 1 year follow up, and compliance issues were excluded. Z scores (standard deviation score) for weight, height and BMI were calculated. Initial comparison of three groups as FR/SDNS, IFRNS/R and SRNS was done. But in the second comparison, we combined FR/SD and SR children in one group as Difficult nephrotic syndrome (DNS) group. Anova Kruskal-Wallis test was used to find significance in three groups in table 1. Subsequent analysis was done by the non-parametric statistic method Mann-Whitney Test to assess significance in subgroups of boys and girls. Results: 27 IFR/R nephrotic children, 36 FR/SD, and 15 SRNS were compared as in Table1. D-NS and IFRNS-R for boys and girls ratio (11; 16) & (18; 32) followed for a median period of 18 (max 46; 12), 24.6 (57.6; 12), and 32 (50;12) months respectively. Conclusion Linear growth (height) is the most affected parameters in children in SRNS and FR/SD nephrotic syndrome. Effect is more significant in girls then in boys as they showed improvement in . No difference in weight and BMI is seen on applied statistics in two groups.

scholarly journals Obeticholic acid—a new therapy in PBC and NASH

2020 ◽  
Vol 133 (1) ◽  
pp. 95-104 ◽  
Author(s):  
Roger W Chapman ◽  
Kate D Lynch
Keyword(s):  
Clinical Trials ◽  
Side Effects ◽  
Ursodeoxycholic Acid ◽  
Farnesoid X Receptor ◽  
Primary Biliary Cholangitis ◽  
Second Line ◽  
Obeticholic Acid ◽  
Second Line Therapy ◽  
Line Therapy

Abstract Introduction Obeticholic acid (OCA) is a semi-synthetic hydrophobic bile acid (BA) analogue that is highly selective agonist of farnesoid X receptor (FXR), a key nuclear BA receptor, which induces expression of gut-derived hormones, in particular fibroblast growth factor 19. The resulting beneficial effects of OCA on glucose and lipid metabolism and particularly hepatic inflammation make it a candidate for the treatment of a variety of conditions including primary biliary cholangitis (PBC) and nonalcoholic steatohepatitis (NASH). Sources of data In PBC patients who have not initially responded to ursodeoxycholic acid, OCA has been shown in double-blind controlled clinical trials to significantly reduce serum alkaline phosphatase. To date, OCA is the only therapy licensed by the FDA, EMA and endorsed by NICE as second line therapy for PBC. No medications are currently approved in Europe or the USA for the treatment of NASH. In recent clinical trials, OCA has been shown encouraging results by improving liver blood tests and reducing liver fibrosis with no worsening of NASH. Areas of agreement OCA is the established second line therapy for PBC in those patients who fail to adequately respond to ursodeoxycholic acid. Areas of controversy The main side effects of OCA treatment in both PBC and NASH is that of dose-dependent pruritis which can lead to treatment discontinuation in ~1–10% of patients. In addition, OCA-treated patients may also exhibit (reversible) alterations in serum lipid levels; most notably a small decrease in high density lipoprotein cholesterol. It is not yet known whether these changes carry a long-term cardiovascular risk in NASH. In addition, the relatively high cost of OCA may limit its use in cash-limited health systems. Growing Points Additional clinical trials are in progress to ascertain the long-term effects of OCA on survival in PBC and NASH. Areas timely for developing research New FXR agonists with a lower rate of side effects are being developed and trialed. Combination therapy with other agents may offer increased efficacy.

How I treat autoimmune hemolytic anemias in adults

Blood ◽  
2010 ◽  
Vol 116 (11) ◽  
pp. 1831-1838 ◽  
Author(s):  
Klaus Lechner ◽  
Ulrich Jäger
Keyword(s):  
Underlying Disease ◽  
Second Line ◽  
Limited Information ◽  
Second Line Therapy ◽  
Term Efficacy ◽  
Line Therapy ◽  
Steroid Dependent ◽  
Common Causes ◽  
Anti Cd20

Abstract Autoimmune hemolytic anemia is a heterogeneous disease with respect to the type of the antibody involved and the absence or presence of an underlying condition. Treatment decisions should be based on careful diagnostic evaluation. Primary warm antibody autoimmune hemolytic anemias respond well to steroids, but most patients remain steroid-dependent, and many require second-line treatment. Currently, splenectomy can be regarded as the most effective and best-evaluated second-line therapy, but there are still only limited data on long-term efficacy and adverse effects. The monoclonal anti-CD20 antibody rituximab is another second-line therapy with documented short-term efficacy, but there is limited information on long-term efficacy and side effects. The efficacy of immunosuppressants is poorly evaluated. Primary cold antibody autoimmune hemolytic anemias respond well to rituximab but are resistant to steroids and splenectomy. The most common causes of secondary autoimmune hemolytic anemias are malignancies, immune diseases, or drugs. They may be treated in a way similar to primary autoimmune hemolytic anemias, by immunosuppressants or by treatment of the underlying disease.

Allogeneic Myeloablative Hematopoietic Stem Cell Transplantation for Chronic Myelogenous Leukemia in the Imatinib Era.

Blood ◽  
2008 ◽  
Vol 112 (11) ◽  
pp. 970-970
Author(s):  
Jiri Pavlu ◽  
Matthias Klammer ◽  
Ian Gabriel ◽  
Richard Szydlo ◽  
Eduardo Olavarria ◽  
...  
Keyword(s):  
Risk Assessment ◽  
Scoring System ◽  
Chronic Phase ◽  
Myelogenous Leukemia ◽  
Prognostic Scores ◽  
Second Line ◽  
Second Line Therapy ◽  
Hematopoietic Stem ◽  
Line Therapy

Abstract Allogeneic hematopoietic stem cell transplantation (allo-HSCT) is no longer the first treatment option for patients with chronic myelogenous leukemia (CML) but there is a considerable debate about its use as a second line therapy. When used in this indication the second-generation tyrosine kinase inhibitors (2G-TKI) induce complete cytogenetic responses (CCyR) in 40–50% of patients in chronic phase but those without CCyR are unlikely to benefit in long term. It is therefore important to identify groups of patients with a good outcome after transplantation so that this may be offered as second line therapy where appropriate. The outcome of allo-SCT has improved over time so we restricted our analysis to the most recent 8 years to coincide with the introduction of imatinib into clinical practice. 131 patients received myeloablative transplants from January 2000 till December 2007. 67 patients were transplanted in chronic phase (14 in second and 2 in third chronic phase), 46 in accelerated phase and 2 in blastic phase. Forty-nine patients received imatinib at some point prior to transplantation and 30 of these experienced failure of imatinib therapy (as defined by European LeukemiaNet criteria). Conditioning consisted of cyclophosphamide and total body irradiation for 51 recipients of sibling stem cells. In addition in vivo T cell depletion with anti CD52 antibody (Campath 1H) was used for 80 unrelated donor transplants. The median age of the patients was 33.4 (15 to 56) years and the median disease duration at transplant was 13 (2 to 105) months. The probability of overall survival (OS) at 3 and 5 years was 64.8% and 62.6% respectively. We confirmed the prognostic value of the EBMT risk assessment score (Gratwohl) and pretransplant level of the C-reactive protein (CRP) and developed a combined additive pretransplant scoring system based on these predictive factors (EBMT risk assessment score plus 0 for CRP <2 mg/L, 1 for CRP from 2 to 10 mg/L, and 2 for CRP >10 mg/L). This identified 5 prognostic groups (Figure 1) with 3yr probabilities of survival of 92.6% (N= 27, score 0–1), 86.2% (N=29, score 2), 58.2% (N=29, score 3), 47.5% (N=20, score 4) and 30.8% (N=26, score 5 or more). The patients who failed imatinib (N=30) had significantly higher prognostic scores on the above described pre-transplant scoring system compared to the rest of patients transplanted (p=0.001). However, in a multivariate analysis adjusted for prognostic scores, their OS was significantly better (p=0.032). The OS in the best prognostic group is comparable with that of unselected patients treated with imatinib and it is possible that their long-term survival might be better. Allogeneic transplantation is unlikely to be preferred as the first line therapy even in selected patients due to its higher early mortality but our data support its use as second line therapy in patients in chronic phase who failed imatinib and have poor pre-2G-TKI predictive factors for CCyR as determined previously at our institution (namely Sokal risk score at diagnosis, the best cytogenetic response obtained on imatinib, G-CSF requirement during imatinib therapy and time from detection of imatinib failure to onset of 2G-TKI therapy) but achieved good score on the pre-transplant scoring system. It should also be used for those whose disease is more advanced where the 2G-TKI do not offer durable remissions. Figure 1 Figure 1.

What is the Long-Term Follow-Up of Nonoperatively Treated Patients with Appendicitis?

2019 ◽  
Vol 85 (5) ◽  
pp. 462-465
Author(s):  
Andrew Felber ◽  
Deven Catalano ◽  
Caitlin Stafford ◽  
Todd D. Francone ◽  
Patricia L. Roberts ◽  
...  
Keyword(s):  
Tertiary Care ◽  
Care Facility ◽  
Nonoperative Treatment ◽  
Interval Appendectomy ◽  
Tertiary Care Facility ◽  
Long Term Follow Up ◽  
Mean Time ◽  
Emergent Surgery

In this study, we determine outcomes after nonoperative treatment of appendicitis. First, we abstracted data for patients discharged with a diagnosis of appendicitis from a tertiary care facility from August 1, 2007, through June 30, 2017. For patients treated nonoperatively, we collected additional medical treatment for appendicitis, future surgical treatment, and date of last follow-up. In our study, we identified 487 patients treated for appendicitis. From this group, 66 patients were successfully treated nonoperatively. Eight patients (12%) had an interval appendectomy at a mean follow-up time of two months. Of the 58 remaining patients, 20 (34%) did not have any further appendicitis-related issues over a mean follow-up period of 25 months. A total of 38 (66%) had recurring or additional concerns requiring further treatment or emergent surgery within a mean time of four months. A large proportion, 76 per cent (n = 29), required unscheduled or emergent appendectomy. There were more patients diagnosed with an abscess (55%) in the group that had further appendicitis issues. In conclusion, nonoperative treatment of appendicitis is associated with significant likelihood of future appendicitis-related treatment or emergency surgery (66%). In addition, patients diagnosed with an abscess are at particularly high risk of future appendicitis-related issues.

scholarly journals Outcome Analysis of Lipomeningomyelocele Repair in Children in a Tertiary Care Center in Nepal

2019 ◽  
Vol 41 (2) ◽  
pp. 30-34
Author(s):  
Puspa R Koirala ◽  
Amit B Pradhanang ◽  
Gopal Sedain ◽  
Mohan R Sharma
Keyword(s):  
Urinary Incontinence ◽  
Low Income ◽  
Tertiary Care ◽  
Lower Limbs ◽  
Long Term Results ◽  
Filum Terminale ◽  
Total Excision ◽  
Satisfactory Outcome ◽  
Care Surgery

Introduction: Lipomeningomyelocele (LMM) is a common neural tube defect especially prevalent in low income countries. When they get appropriate care, long term result is generally good provided the patient receives good neurosurgical, pediatric and rehabilitation care. Surgery is the mainstay of treatment. Our aim was to analyze immediate and long-term results of lipomeningomyelocele repair in symptomatic patients presenting to TU Teaching Hospital, Kathmandu, Nepal. Methods: Thirteen patients admitted to Department of Neurosurgery from January 2017 to December 2018 were evaluated. All patients underwent MRI of whole spine before surgery. Surgical procedures involved total excision of lipoma and repair in 10 patients and subtotal excision and repair in 3 patients. Division of filum terminable could be donein 8 patients. Follow up varied from 6 months to 2 years. Results: This study included 8 (61.5 %) patients of lumbosacral LMM, 3 (23%) patients of sacral LMM and 2 (15.38%) patients of thoracolumbar LMM. About 2 (15.38%) were operated before 3 months of age, 2 (15.38%) were operated between 3-6 months of age, 5 (38.46%) were operated between 6-12 months of age and 4 (30.7%) were operated after 1 year. All children except one presented with lump on back since birth and four presented with urinary incontinence, one presented with bilateral club foot. Four (30.7%) patients had weakness of one or both lower limbs. Two (15.38%) patients had improvement in urinary incontinence, two unchanged and one had developed urinary incontinence postoperatively. Four children with had weakness of lower limbs; one patient improved whereas three patients did not improve postoperatively. There was no development of post operative hydrocephalus after LMM repair. Three (23%) patients developed wound infection who responded well with regular dressings and antibiotics. Conclusion: Lipomeningomyelocele repair can be done with satisfactory outcome with total excision of lipoma and division of filum terminale. Patients with residual diploma and undivided filum terminale should be observed closely for the development of progressive neurological deterioration.

Sign in / Sign up

Export Citation Format

Share Document