Paroxysmal Nocturnal Hemoglobinuria (PNH): Long Term Follow up of 48 Patients in a Single Institution.

Blood ◽  
2006 ◽  
Vol 108 (11) ◽  
pp. 3762-3762
Author(s):  
Jefferson Ruiz ◽  
Larissa A. Medeiros ◽  
Vaneuza A.M. Funke ◽  
Carmem M.S. Bonfim ◽  
Marco A. Bitencourt ◽  
...  

Abstract Background: PNH is a clonal acquired disease characterized by poor expression of CD55 and CD59, adding higher lytic activity to the complement pathway, ending up in hemolysis, pancytopenia and thrombosis. The clinical variability and the lack of effective therapeutics make the treatment of PNH a real challenge. Material and methods: A retrospective analysis of 48 patients diagnosed with PNH between feb/1997 and march/2006 admitted to our center was performed. Before 1999, diagnosis was made by HAM and SACAROSIS tests. From this point immunophenotyping analysis was used to establish diagnosis. Bone marrow aspirate and biopsy, cytogenetic and biochemistry analysis were performed for exclusion of comorbities. Patients were divided in two groups: 1) de novo PNH associated with marrow failure (MF) and 2) PNH as an evolution of severe aplastic anemia (SAA) or mielodysplastic syndrome (MDS). Response was defined as reduction at hemolytic episodes as well as improvement of CBC and transfusion requirements. Results: Median age was 24 years (range 9–75); male patients constituted 58% of the sample (male/female: 1,4:1). De novo PNH with associated marrow failure occurred in 22 patients (group 1) and evolutive PNH in 15, being 13 from previous SAA and 2 from previous hypoplastic MDS (group 2). Thirty seven patients were followed at our clinic during this time: 25 (67,5%) received immunesupression with cyclosporine and steroids. Overall response (partial or complete) after one year of therapy was 84% (31/37). The remaining 11 patients (32,5%) received steroids alone, androgens or only observation, four of them remained with stable disease. HLA match hematopoietic stem cell transplantation (HSCT) was performed in 18/48 (38%), being 11 from group 1 and 7 from group 2. Main indications were: pancytopenia (77%), thrombosis (17%) and AML (6%). Only nine patients died (19%): five after HSCT and 4 of those followed at our clinic (massive thrombosis and hemolysis). Overall survival after HSCT was 72% with median follow-up of 890 days. Overall survival among outpatient clinic patients was 86% with a median follow-up of 2340 days. Most frequent outpatient events were: hemolysis (48%), pancytopenia (13%), thrombosis (13 %), renal failure (9%) and infection (8%).Fourteen patients (37%) remained without symptoms. Conclusions:Immunosuppressive therapy with cyclosporine and corticosteroids is an excellent strategy in patients with PNH and associated marrow failure, leading to durable clinical responses.The present data supports previous published indications for HSCT: ASS and thrombosis.Overall survival of PNH patients who received HSCT achieved 72%.

2015 ◽  
Vol 2;18 (2;3) ◽  
pp. E147-E155
Author(s):  
Yasser M. Amr

Background: Chronic inguinal neuralgia has been reported after inguinal herniorrhaphy, caesarean section, appendectomy, and trauma to the lower quadrant of the abdomen or inguinal region. Objectives: This study was designed to evaluate the efficacy of pulsed radiofrequency in management of chronic inguinal neuralgia. Study Design: Randomized, double-blind controlled trial. Setting: Hospital outpatient setting. Methods: Twenty-one patients were allocated into 2 groups. Group 1 received 2 cycles of pulsed radiofrequency (PRF) for each nerve root. In Group 2, after stimulation, we spent the same time to mimic PRF. Both groups received bupivacaine 0.25% + 4 mg dexamethasone in 2 mL for each nerve root. Visual Analogue Scale (VAS) was assessed. Duration of the first block effective pain relief was reported. Repeated PRF blockade was allowed for any patient who reported a VAS > 30 mm in both groups during the one year follow-up period. The number and duration of blocks were reported and adverse effects were also reported. Results: Significantly longer duration of pain relief was noticed in Group 1 (P = 0.005) after the first block, while the durations of pain relief of the second block were comparable (P = 0.59). In Group 1 the second PRF produced pain relief from the twenty-fourth week until the tenth month while in Group 2, pain relief was reported from the sixteenth week until the eighth month after the use of PRF. All patients in Group 2 received 3 blocks (the first was a sham PRF) during the one year follow-up period. Meanwhile, 2 PRF blocks were sufficient to achieve pain relief for patients in Group 1 except 4 patients who needed a third PRF block. No adverse events were reported. Limitations: Small sample size. Conclusion: For intractable chronic inguinal pain, PRF for the dorsal root ganglion represents a promising treatment modality. Key words: Radiofrequency, chronic, inguinal neuralgia


Author(s):  
A.P. Voznyuk ◽  
◽  
S.I. Anisimov ◽  
S.Y. Anisimova ◽  
L.L. Arutyunyan ◽  
...  

Purpose. To evaluate the efficacy and safety of femtolaser-assisted phacoemulsification in glaucomatous eyes in the long-term follow-up. Materials and methods. A retrospective analysis of the results of the surgical treatment of patients with combined cataract and glaucoma pathology was analyzed. The patients were divided into groups depending on the method of surgical intervention: 1) phacoemulsification with femtolaser support (26 eyes, 23 patients); 2) phacoemulsification (36 eyes, 30 patients); Results. Before surgery, there were no statistically significant differences in IOP and corneal hysteresis (СН) between groups 1 and 2. The mean values of IOP cc, IOP g and СН of group 1 before surgery were 22.7±6.1 mm Hg, 20.9±6.9 mm Hg, 8.5±1.6 mm Hg; 2 group – 22.9±8.7 mm Hg, 21.6±8.9 mm Hg, 8.9±1.6 mm Hg respectively. Average values of IOP cc, IOP g and CН 5 years after the surgical treatment in group 1 were 15.3±1.2 mm Hg, 14.4±3.4 mm Hg, 9.6±4.2 mm Hg; in group 2 – 18.0±4.2 mm Hg, 16.1±4.2 mm Hg, 8.8±2.2 mm Hg respectively. In both groups, stabilization of IOP and CH indices was noted, which remained throughout the entire observation period, which shows the normalization of the biomechanical properties of the corneoscleral membrane of the eye in the long-term postoperative period. Conclusion. Femtolaser accompaniment of phacoemulsification is an effective and safe method of cataract surgery for combined pathology. Key words: femtolaser, cataract, glaucoma, phacoemulsification.


2003 ◽  
Vol 11 (2) ◽  
pp. 131-134 ◽  
Author(s):  
Jai Raman ◽  
Pallav Shah ◽  
Siven Seevanayagam ◽  
John Cheung ◽  
Brian Buxton

Mitral regurgitation due to bileaflet prolapse and ischemic causes can be difficult to repair. Midterm experience of the Alfieri edge-to-edge repair as an alternative to valve replacement is reported. Twenty-six patients with severe mitral regurgitation underwent the Alfieri repair between January 1998 and December 2000 (group 1); 15 cases were due to bileaflet prolapse and 7 were of ischemic origin. During the same period, valve replacement was performed in 36 patients (group 2), 20 of whom had similar indications. Follow-up was complete to a mean of 15 months (range, 1–28 months). There was no early death in either group. During follow-up, there was no reoperation in group 1, while 2 patients in group 2 required reoperations due to prosthetic valve endocarditis. There were 4 major thromboembolic or bleeding events in group 2, and none in group 1. All patients in group 1 had trivial to mild mitral regurgitation on follow-up echocardiography. The mean mitral valve gradient was significantly higher in group 2 compared to group 1 (7.2 versus 3.2 mm Hg, p = 0.001). The edge-to-edge repair is associated with good early and midterm results. Long-term follow-up is required to evaluate the durability of this technique.


Blood ◽  
2012 ◽  
Vol 120 (21) ◽  
pp. 4804-4804
Author(s):  
Mohamed Abdelfatah ◽  
Adel Al-Alwan ◽  
Zeyad Kanaan ◽  
Mark R Litzow ◽  
Alexandra Wolanskyj ◽  
...  

Abstract Abstract 4804 Background: Chronic myeloid leukemia (CML) is one of the classic myeloproliferative neoplasms characterized by a reciprocal translocation of BCR and ABL t(9;22)(q34;q11). Tyrosine kinase inhibitors (TKI) have revolutionized the management of CML in inducing rapid and prolonged responses. However; clonal evolution (CE) is considered a poor prognostic factor and a criterion for accelerated phase (AP) CML by the World Health Organization (WHO). Deletion of chromosome Y (−Y) is frequently considered an age-related abnormality and the exact prognostic value has not yet been determined. Aim: To determine if –Y carries an impact on the clinical outcome of male pts with CML. Methods: All male patients diagnosed with chronic phase CML in our institution between 1993 and 2011 were screened for -Y. Data were collected in a retrospective manner and compared (using t-test) to male patients with sole BCR-ABL translocation after excluding patients with advanced stages (accelerated phase, blast phase). Demographics, laboratory tests, cytogenetic analysis, molecular testing and survival data were abstracted. Kaplan-Meier estimates of overall survival were used via JMP software v9.0. Institutional Review Board (IRB) approval was obtained for this study in accordance with the Helsinki Declaration. Results: 20 of 162 (12%) males with CML were found to have –Y abnormality (group 1). CML male patients with sole Philadelphia chromosome abnormality were the control cohort (group 2). In group1; the median age was 57 years, BMI 27.7, hemoglobin 12.2 g/dL, white blood cell count (WBC) 32.8 x109/L, platelet 270 x109/L, and peripheral blood blasts 1%. Sokal risk was low in 30%, intermediate in 65% and high in 5% of pts. Nine pts (45%) were treated with interferon (IFN) prior to TKI. In group 2; the median age was 54 years, hemoglobin 12 g/dL, WBC 57 x109/L, and platelet 282 x109/L. Sokal risk was low in 37%, intermediate in 47%, and high in 16%. 46 of 142 patients (32%) had received previous interferon therapy. All patients in both groups had chronic phase CML at the time of diagnosis, with a median bone marrow cellularity of 95%. In group 1, 14 of 20 pts (70%) received imatinib, all of whom achieved a complete hematological response (CHR), 7 of 14 pts (50%) had partial cytogenetic response, 2 of 14 pts (14%) achieved a complete cytogenic response (CCyR);1 (7%) pt achieved CCyR within 12 months and an additional 1 (7%) by 18 months). Two pts of 12 (16%) achieved at least a major molecular response (MMR); one of whom (8%) achieved a complete molecular response (CMR). In comparison, 107of 142 pts (75%) in group 2 received imatinib, all of whom achieved CHR. Twenty-one pts (20%) achieved partial cytogenetic remission. CCyR was more frequently achieved than group 1 (48/107 pts (45%), p 0.026); 24 pts (22%) achieved CCyR within 12 months of therapy and an additional 10 pts by 18-months. MMR and/or CMR was higher in group 2 compared to group 1 (34 /101 (34%), p 0.18); 16 (16%) of which were CMR. In group 1; 6 (30%) pts had disease progression; 4 of 20 pts (20%) progressed to blast phase (BP) and 2 pts (10%) progressed to AP, compared to 32 (22%) pts in group 2 (p 0.17); 22 (15%) of whom progressed to BP and 10 (7%) patients progressed to AP. Median overall survival was 110 months in group 1 compared to 155 months in group 2 (log rank p=0.48). On multivariate analysis, CCyR was an independent factor for a better OS (p 0.03), but not –Y (p 0.7). Conclusion: Loss of the Y chromosome in chronic myelogenous leukemia is an infrequent phenomenon (12%). Although patients with –Y had a statistically significant less chance to achieve CCyR, loss of the Y chromosome did not affect the progression rate or overall survival. Larger scale studies are needed to confirm our observations Disclosures: No relevant conflicts of interest to declare.


2021 ◽  
Vol 8 (3) ◽  
pp. 181-186
Author(s):  
Didem Dereli Akdeniz ◽  
Gürkan Avcı

Objective: Multinodular goiter is a common surgical disease. There is no common consensus regarding the extent of thyroidectomy for multinodular goiter. This  study aims to present personal experience on treating patients with multinodular goiter and to compare complication rates and results of total and partial thyroidectomy for multinodular goiter. Material and Method: Three hundred fifty patients underwent thyroidectomy for multinodular goiter between May 2003 and October 2010. All patients were diagnosed as multinodular goiter and were referred to surgery by one endocrinologist. All operations were also performed by one surgeon using microsurgical techniques. Partial thyroidectomy (bilateral subtotal or unilateral total thyroidectomy and contralateral subtotal thyroidectomy) was performed in 65 patients (Group-1) and extracapsular total thyroidectomy was performed in 285 patients (Group-2). All patients are being followed followed from the day they were diagnosed until now by the same endocrinologist. Fisher exact test was used for statistical analysis. Results: In Group-1, one patient had transient vocal-cord palsy and but none had hypoparathyroidism. On the other hand, in Group-2, two patients had transient vocal-cord palsy, five had hypocalcemia (one was permanent), and one had a hematoma. Mortality and wound infection were absent in both groups. The histopathological studies showed that 40 incidental thyroid carcinomas occurred among Group-2 patients. During long-term follow-up, 13 patients had goiter recurrence (n = 65, 20%) in Group-1, whereas none had goiter recurrence in Group-2. Conclusion: There were no statistically significant differences in the complication rate between subtotal and total thyroidectomy groups (p>0.05). However, the recurrence rate was higher (statistically significant) after subtotal thyroidectomy than after total thyroidectomy (p<0.05). Total thyroidectomy eliminated future recurrence of the disease and is also curative in incidental thyroid carcinomas. In addition, it can be safely performed using microsurgical techniques.


2021 ◽  
Vol 27 (1) ◽  
Author(s):  
Osama Abdullah Bawazir ◽  
Obada Alladh Alhallaq ◽  
Bashair Albayhani ◽  
Abdullah Bawazir

Abstract Background Surgical correction of the webbed penis is challenging. We presented the safety and feasibility of webbed penis correction for neonates under local anesthesia using a simple principle of leaving equal cylindrical shaft skin. Methods This retrospective study included 530 patients who presented for circumcision to three pediatric surgery centers between May 2017 and January 2020. We included male patients aged less than four weeks old who had circumcision with a minimum of 6 months follow-up. We compared patients with normal penile anatomy (n = 451, Group 1) to a simple webbed penis (n = 79, Group 2). Results There were no differences in age and weight between groups. The procedure time was significantly longer in Group 2 (8.05 ± 3.11 vs. 7.48 ± 2.07 min; P = 0.04). There were no differences in bleeding (P = 0.38), redundant foreskin (P > 0.99), need for corrective surgery (P = 0.38), and re-suturing (P = 0.28) between groups. The procedure success was significantly higher in Group 1 (449 (99.56%) vs. 70 (88.6%); P < 0.001). Parents' satisfaction was measured at two weeks with no difference between both groups. Conclusion Simple penoscrotal web is not a contraindication for neonatal circumcision. Circumcision of the penoscrotal web had good esthetic results with comparable outcomes to those with a normal penis without a web.


2021 ◽  
Vol 10 (19) ◽  
pp. 4417
Author(s):  
Katarzyna Lewczuk ◽  
Joanna Konopińska ◽  
Joanna Jabłońska ◽  
Jacek Rudowicz ◽  
Patrycja Laszewicz ◽  
...  

This retrospective study analyzed the surgical and refractive outcomes of a XEN Gel Implant (Allergan, Abbvie Company, Irvine, CA, USA) in naïve patients versus those with previous glaucoma surgery. We evaluated the efficacy of XEN implantation in 86 glaucoma patients during a long-term follow-up period. Patients were divided into two groups: naïve patients (Group 1) and patients with previous glaucoma surgery (Group 2). Eyes that received a XEN Gel Stent placement from December 2014 to October 2019 were included. Intraocular pressure (IOP) change, corrected distance visual acuity (CDVA), change in glaucoma medications, frequency of slit lamp revision procedures, and frequency of secondary glaucoma surgeries were the primary outcomes. In Group 1, the mean IOP before surgery was decreased significantly from 25.00 ± 7.52 mmHg to 16.83 ± 5.12 mmHg by the end of the study. In Group 2, the mean IOP decreased significantly from 25.35 ± 7.81 mmHg to 17.54 ± 5.34 mmHg. The mean IOP decrease from baseline was 29% in Group 1 and 27% in Group 2 (p = 0.567). There were no significant differences between the groups in the IOP baseline level, the final level, or the change between preoperative and final levels. The qualified success rate for Group 2 was 68.7% versus 76.5% for Group 1 for the initial procedure and 15.4% vs. 20.2%, respectively, for complete success rate (p > 0.05). However, at the end of the follow-up, more patients achieved an IOP < 18 mmHg in Group 1 than in Group 2. Despite the need for more anti-glaucoma medications, repeat XEN Gel implantation appears to show promising results in patients with previously failed anti-glaucoma procedures, owing to its minimal invasiveness.


VASA ◽  
2011 ◽  
Vol 40 (3) ◽  
pp. 199-204 ◽  
Author(s):  
Xia ◽  
Yang ◽  
Qu ◽  
Cheng ◽  
Wang

Background: This study was designed to investigate the impact of carotid artery stenting (CAS) on plasma levels of P-selectin, von Willebrand (vWF) and endothelin-1. Patients and methods: Sixty-seven patients who received CAS were divided into group 1 (one stent for a simple lesion, n = 38) and group 2 (two stents for complex lesions, n = 29). The levels of P-selectin, vWF and endothelin-1 were measured before CAS, 1 h, 6h, 24 h and 2 weeks after the stenting. Results: Sixty-one patients completed one-year follow up. Restenosis was noted in 14 (23 %) patients, among these three (4.8 %) had a restenosis of > 50 % of the vascular lumen. In all patients, the levels of P-selectin, vWF and endothelin-1 increased immediately after CAS (P < 0.05 or < 0.01). The levels of vWF and endothelin-1 in group 2 were higher than in group 1 (P < 0.05 or 0.01). There was no significant difference in P-selectin and endothelin-1 between the restenosis and non-restenosis group (P > 0.05). The 24 h vWF in patients with restenosis were higher than in non-restenosis group (P < 0.05). Conclusions: CAS results in a significant increase in plasma P-selectin, vWF and endothelin-1. The post-CAS levels of P-selectin, vWF and endothelin-1 are related to the extent of endothelial injury. Whether they are associated with restenosis 12 months after the treatment requires further investigation.


Author(s):  
Paola Terlizzese ◽  
Miriam Albanese ◽  
Dario Grande ◽  
Giuseppe Parisi ◽  
Margherita Ilaria Gioia ◽  
...  

Background: Hypothyroidism is a frequently observed comorbidity in patients with chronic heart failure (CHF), possibly giving rise to unfavorable outcomes. Aim: The aim of the study was to evaluate the impact of TSH changes over time on cardiac function and prognosis of outpatients with CHF. Methods: Patients underwent clinical, electrocardiographic, and echocardiographic evaluations at baseline and after 12 months. Moreover, blood chemistry tests were performed to evaluate renal function, cardiac biomarkers, fT3, fT4, and TSH levels. Based on TSH serum levels, patients were retrospectively classified into four categories: Group 1, patients with improved thyroid function at one-year follow up vs. baseline; Group 2, patients with stable and mildly high TSH values (3.74 – 10 mUI/L); Group 3, patients with worsening thyroid function; Euthyroid patients Group, TSH levels within the normal range of reference at baseline as well as at 12 months follow-up. We considered as end-points: one-year changes of laboratory and echocardiographic parameters; hospitalizations due to worsening of HF (acute decompensated heart failure - ADHF); death for all causes. Results: Among 257 patients, 174 (67.7%) were euthyroid at baseline and after 12 months. Group 1 patients (n. 22, 8.6%) showed a significant improvement in systolic and diastolic function, filling pressures, NT-proBNP and Galectin-3. Group 2 patients (n. 34, 13.2%) did not exhibit significant modifications in studied parameters. Group 3 patients (n. 27, 10.5%) showed worsening of diastolic function and NT-proBNP and a greater risk of ADHF (HR: 2.12; 95%CI: 1.20-3.74; p: 0.009) and death (HR: 4.05; 95%CI: 2.01-8.15; p<0.001). Conclusion: In patients with CHF, changes in thyroid function over time influenced echocardiographic parameters and biomarkers reflecting modifications of cardiac function and prognosis, thus suggesting the clinical relevance of thyroid deficiency screening and correction.


Blood ◽  
2004 ◽  
Vol 104 (11) ◽  
pp. 5221-5221
Author(s):  
Mauricette Michallet ◽  
Quoc-Hung Le ◽  
Anne-Sophie Michallet ◽  
Anne Thiebaut ◽  
Emmanuelle Tavernier ◽  
...  

Abstract Multiple myeloma remains one of the best indication for intensive chemotherapy followed by autologous hematopoietic stem cell transplantation (autoT). Intensive therapy followed by autologous transplantation is superior to conventional chemotherapy and it was demonstrated that two autoT were superior to one except for patients in very good partial response or in complete response after the first autotransplant. Peripheral blood stem cells (PBSC) can be used as well as bone marrow as HSC source with the same efficacy but very few data have been reported regarding PBSC recruitment. The main goal of our work was to study the impact on overall and event-free survival (OS and EFS) of PBSC recruitment using either growth factors (GF) alone (steady state) or chemotherapy followed by GF. Secondly, we performed a multivariate analysis studying influence on OS and EFS of sex, age, lines of therapy, pretransplant status, TBI, PBSC recruitment and number of autoT. We have analyzed 193 PBSC autoT (1 autoT=160, 2 autoT=33) performed for 160 MM patients [81 males and 71 females, mean age: 55 years (39–71)]. At diagnosis, 88 patients presented a MM Ig G (70k and 18l), 28 Ig A (16k and 12l), 3 Ig D (1k and 2l), 21 light chains k and 13 light chains l, 3 non secreting and 4 with plasmocyte leukemias. According to Durie and Salmon classification 75% of patients were in stage III, 15% in stage II and 10% in progressive I. Before transplantation, patients have received 1 line of poly-chemotherapy (n=141), 2 lines (n=15) or 3 lines (n=4) and 154 were evaluated for the response with 11 complete remission, 113 partial remission and 30 stable or evolutive disease just before transplant. As HSC (n=189), patients received PBSC which were recruited by GF alone (n=105) or cyclophosphamide+GF (n= 84). Conditioning (n=189),consisted in melphalan and TBI (n=51), melphalan alone (n=132), melphalan associated to cyclophosphamide or busulfan (n=6). We divided the population into 4 groups : group 1 who received one autoT of PBSC recruited by GF (n=76), group 2 one autoT of PBSC recruited by chemotherapy+GF (n=50), group 3 two autoT of PBSC recruited by GF (n=16) and group 4 two autoT of PBSC recruited by chemotherapy+GF (n=17). The median follow-up (FU) of the 4 groups were different with shorter FU (group 3: 9.9 months, group 4: 13 months) for patients who received tandem autoT because of the recent character of this strategy as compared to a long term follow-up for patients who received only one transplant (group 1: 35months, group 2: 55.3 months). Probabilities of OS and EFS at 2 years were 76% (95%CI 67–87) and 60% (95%CI 49.5–73) for group 1, 77% (95%CI 65–90.5) and 70% (95%CI 57.5–85) for group 2, 87.5% (95%CI 73–100) and 72.9% (95%CI 49–100) for group 3, 100% and 66.7% (95%CI 36–100) for group 4. The difference was not significant because of follow-up differences between the 4 groups and small number of patients in groups 3 and 4. In addition, multivariate analysis did not show any significant influence of the different studied parameters on OS and EFS. Nevertheless, because of these interesting preliminary results, a longer follow-up is warranted for definitive conclusions.


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