单中心成人重型血友病A低/中剂量凝血因子Ⅷ预防治疗30例疗效分析北大核心CSCD

目的评估低/中剂量凝血因子Ⅷ(FⅧ)预防治疗在成人重型血友病A患者中的疗效。方法纳入在南京大学医学院附属南京鼓楼医院接受低/中剂量FⅧ预防治疗的30例成人重型血友病A患者(低剂量预防组20例、中剂量预防组10例),对预防治疗前、后的年出血次数(ABR)、年关节出血次数(AJBR)、靶关节数量、血友病功能独立性评分(FISH)、生活质量评分及健康状况评分(SF-36)进行回顾性分析、比较。结果中位随访48个月。与预防前按需治疗时相比,低、中剂量预防治疗组ABR、AJBR、靶关节数量均明显降低(P<0.05),且中剂量预防治疗组改善优于低剂量预防治疗组(P<0.05)。低、中剂量预防治疗后与按需治疗时相比,患者的FISH评分、生活质量评分及SF-36评分均明显改善(P>0.05),但低剂量预防组和中剂量预防组之间以上指标差异无统计学意义(P>0.05)。结论低、中剂量FⅧ预防治疗较按需治疗可明显降低重型血友病A患者出血频率,延缓关节病变的进展,提高患者生活质量。中剂量预防组临床出血改善方面优于低剂量预防组。     

单中心成人重型血友病A低/中剂量凝血因子Ⅷ预防治疗30例疗效分析

目的评估低/中剂量凝血因子Ⅷ(FⅧ)预防治疗在成人重型血友病A患者中的疗效。方法纳入在南京大学医学院附属南京鼓楼医院接受低/中剂量FⅧ预防治疗的30例成人重型血友病A患者(低剂量预防组20例、中剂量预防组10例), 对预防治疗前、后的年出血次数(ABR)、年关节出血次数(AJBR)、靶关节数量、血友病功能独立性评分(FISH)、生活质量评分及健康状况评分(SF-36)进行回顾性分析、比较。结果中位随访48个月。与预防前按需治疗时相比, 低、中剂量预防治疗组ABR、AJBR、靶关节数量均明显降低(P<0.05), 且中剂量预防治疗组改善优于低剂量预防治疗组(P<0.05)。低、中剂量预防治疗后与按需治疗时相比, 患者的FISH评分、生活质量评分及SF-36评分均明显改善(P>0.05), 但低剂量预防组和中剂量预防组之间以上指标差异无统计学意义(P>0.05)。结论低、中剂量FⅧ预防治疗较按需治疗可明显降低重型血友病A患者出血频率, 延缓关节病变的进展, 提高患者生活质量。中剂量预防组临床出血改善方面优于低剂量预防组。     

重组人凝血因子Ⅶ小剂量短疗程次级预防性治疗中重度血友病A患儿关节出血

目的 探讨重组人凝血因子Ⅶ制剂小剂量短程预防性输注能否有效减少中重度血友病A患儿关节出血问题.方法 对2008年11月-2009年4月期间就诊的13例年龄3~11岁的中重度血友病A患儿,均在为期2个月内接受重组人凝血因子Ⅶ2次/周、间隔3 d、每次7.5~10.0 U/㎏的静脉预防性输注,记录治疗前2个月与治疗2个月时...     

血友病关节出血中小剂量凝血因子输注的临床疗效观察

目的探讨血友病关节出血中小剂量凝血因子输注的临床疗效观察。方法选取某院收治的92例血友病急性关节出血患者,采用红蓝双色球法将其分成两组,每组各46例,对照组采用推荐剂量凝血因子输注治疗,观察组采用小剂量凝血因子输注治疗,比较治疗前后两组患者的关节出血程度和疼痛改善效果。结果治疗前两组患者的关节出血程度比较,差异无统计学意义(P>0.05);治疗后组间出血程度比较,差异无统计学意义(P>0.05);治疗后两组轻微出血者比例(54.35%、47.83%)高于本组治疗前(15.22%、17.39%),严重出血者比例(21.74%、21.74%)低于本组治疗前(52.17%、52.17%),差异有统计学意义(P<0.05),中等出血者比例本组治疗前后对比,差异无统计学意义(P>0.05)。治疗前、后,两组组间疼痛评分比较,差异无统计学意义(P>0.05);两组治疗后的疼痛评分均低于本组治疗前(P<0.05)。结论小剂量凝血因子输注与推荐剂量凝血因子输注治疗血友病急性关节出血均可有效改善关节出血情况和疼痛情况,在血友病急性关节出血治疗中在经济或药品应用条件受限情况下可首推小剂量凝血因子输注方案。     

运动功能的影响

目的观察肌力训练联合本体感觉训练对老年人群平衡及运动功能的改善作用。 方法采用随机数字表法将90例平衡能力低于同龄人水平的老年对象分为肌力训练组、本体感觉训练组及联合治疗组，每组30例。肌力训练组受试者给予肌力训练（包括仰卧位悬吊训练、俯卧位悬吊训练及行进间弓步转体训练等），本体感觉训练组受试者给予本体感觉训练，联合治疗组受试者则给予肌力训练及本体感觉训练，其训练方法同上。于入选时、锻炼9周后对各组受试者膝关节肌力、平衡能力及下肢运动功能进行检测对比。 结果经9周锻炼后，发现3组受试者膝关节肌力、平衡能力及下肢运动功能均较入选时有不同程度改善，并且上述指标均以观察组受试者的改善幅度较显著，与肌力训练组及本体感觉训练组间差异均具有统计学意义（P<0.05）。 结论肌力训练或本体感觉训练对老年人群平衡及运动功能均有不同程度改善作用，两者联用具有协同功效，能进一步提高老年人群肌力、平衡能力及肢体运动功能，该健身锻炼方法值得在老年人群中推广、应用。     

血友病患者全膝置换围手术期的凝血因子替代治疗

背景:世界血友病协会的治疗指南明确表示,关节置换应当在保证患者凝血因子活性的情况下进行。目的:观察血友病关节炎患者行人工膝关节置换围手术期凝血因子替代治疗的安全性和有效性。方法:1997/2006在血液内科的配合下,围手术期行凝血因子Ⅷ和Ⅸ活性水平监测,根据指南制定常规流程,进行因子Ⅷ(冻干人凝血因子Ⅷ)或因子Ⅸ(凝血酶原复合物)的替代治疗,对4例血友病关节炎患者共6个膝关节行人工膝关节置换治疗。结果与结论:围手术期应用凝血因子进行常规替代治疗,血友病患者围手术期出血量与类风湿关节炎和骨关节炎患者差异无显著性意义(P=0.885)。置换后早期3个膝关节出现关节内血肿或肌肉出血,其中1例患者因凝血因子抑制性抗体形成,导致1侧膝关节置换后伤口裂开,行扩创清理后,伤口愈合良好。4例患者6个膝关节置换后没有晚期感染、假体松动、移位和断裂等并发症发生。所有4例患者置换前膝关节KSS评分平均28.2分,功能评分平均35分。置换后KSS评分85.2分,功能评分87分,与普通类风湿关节炎和骨关节炎膝关节置换后评分相似,但平均住院天数延长约3倍,住院费用为普通膝关节置换的2.5~3倍。结果显示凝血因子的替代治疗和凝血因子水平监测是保证血友病关节炎行人工膝关节置换最后成功的关键。     

血友病患者慢性疼痛的自我管理与同伴教育

血友病是一种X染色体连锁的先天性出血性疾病,包括血友病A和血友病B,分别为凝血因子Ⅷ( FVIII)和凝血因子IX( FIX)缺乏所致。血友病常见的出血部位是关节和肌肉,出血产生的压力或局部组织炎症可引起急性或慢性的剧烈疼痛。而关节、肌肉的反复出血,最终导致永久性关节破坏、肌肉萎缩以致残废[1]。生活质量评估表明,71%的血友病患者伴有关节、肌肉的疼痛,其中4%的患者经历着非常严重的疼痛。多数血友病患者由于替代治疗受限,出血后不能得到及时治疗,造成慢性血友病性关节病,由此带来的慢性疼痛不仅损害了患者的生理功能,还限制了日常活动和社会交往,导致情绪低落、活动减少、自尊心下降以及痛感增强[2-4]。     

本体感觉训练结合针刺治疗对颈型颈椎病的疗效观察

目的：观察本体感觉训练结合针刺治疗对颈型颈椎病的临床疗效。方法：入组的90例颈型颈椎病患者,根据随机数字表法,随机分为：本体感觉训练组、针刺治疗组、本体感觉结合针刺治疗组。每周治疗5次,连续治疗4周。分别在治疗前和治疗4周后进行颈椎功能障碍指数（Neck Disability Index,NDI）调查问卷评分、简化疼痛量表评分（Mc Gill Pain Questionnaire,MPQ）、本体感觉功能评定。结果：经过为期4周的康复治疗后,3组颈型颈椎病患者的NDI评分、简化MPQ评分、本体感觉功能均较治疗前有所改善(P<0.05),差异具有显著性意义,组间比较显示结合治疗组较单纯本体感觉训练组和单纯针刺治疗组在NDI评分、简化MPQ评分降低幅度更大,本体感觉功能改善程度更为明显,组间比较差异具有显著性意义(P<0.05)。结论：本体感觉训练和针刺治疗均可促进颈型颈椎病患者的恢复,二者结合可更有效的改善颈型颈椎病患者的功能障碍状况,减轻患者颈肩部疼痛症状,促进患者颈椎本体感觉功能的恢复。     

血友病患者全膝置换围手术期的凝血因子替代治疗

背景：世界血友病协会的治疗指南明确表示,关节置换应当在保证患者凝血因子活性的情况下进行。目的：观察血友病关节炎患者行人工膝关节置换围手术期凝血因子替代治疗的安全性和有效性。方法：1997/2006在血液内科的配合下,围手术期行凝血因子Ⅷ和Ⅸ活性水平监测,根据指南制定常规流程,进行因子Ⅷ（冻干人凝血因子Ⅷ）或因子Ⅸ（凝血酶原复合物）的替代治疗,对4例血友病关节炎患者共6个膝关节行人工膝关节置换治疗。结果与结论：围手术期应用凝血因子进行常规替代治疗,血友病患者围手术期出血量与类风湿关节炎和骨关节炎患者差异无显著性意义（P=0.885）。置换后早期3个膝关节出现关节内血肿或肌肉出血,其中1例患者因凝血因子抑制性抗体形成,导致1侧膝关节置换后伤口裂开,行扩创清理后,伤口愈合良好。4例患者6个膝关节置换后没有晚期感染、假体松动、移位和断裂等并发症发生。所有4例患者置换前膝关节KSS评分平均28.2分,功能评分平均35分。置换后KSS评分85.2分,功能评分87分,与普通类风湿关节炎和骨关节炎膝关节置换后评分相似,但平均住院天数延长约3倍,住院费用为普通膝关节置换的2.5~3倍。结果显示凝血因子的替代治疗和凝血因子水平监测是保证血友病关节炎行人工膝关节置换最后成功的关键。     

血友病的治疗进展

血友病是一组X连锁隐形遗传性出血性疾病,主要表现为自发性出血或外伤、手术后出血,反复关节肌肉出血可以致残,危险部位出血甚至威胁生命。替代治疗是血友病唯一有效的治疗方法,同时需要康复、理疗、护理、心理关怀等综合治疗。治疗理念已从出血后按需治疗发展为预防治疗,未来的发展方向将是基因检测阻断患者出生,长效凝血因子的输注以及基因治疗等,从而使患者彻底摆脱疾病的困扰。     

Evidence-based use of recombinant FVIIa (NovoSeven, NiaStase) for the treatment of hemophilia with inhibitors in children and adolescents.

Children and adolescents comprise a significant proportion of the hemophilia population, including those patients who have developed inhibitors to factor VIII or FIX. We examine the use of rFVIIa for the treatment of bleeding episodes and the prevention of bleeding in children and adolescents with hemophilia A and B with inhibitors, focusing on registry data and recent clinical trial results. Based on this review of the literature, we conclude that recombinant FVIIa is safe and effective for use in controlling bleeding in these patient populations.     

家庭治疗和护理行为干预模式在血友病患儿中的应用

目的探讨适合地方血友病诊疗中心开展的儿童血友病家庭治疗和护理行为的干预模式。方法由血友病护士和2～18岁的血友病患儿及其父母组成护理干预小组,针对儿童血友病家庭治疗和护理中存在的问题进行讨论和归纳,通过文献回顾和专家咨询,制定综合性护理干预措施,包括个体化凝血因子注射培训指导、血友病知识讲座、血友病患儿家庭联谊会、家庭治疗护理行为随访等,并于干预前后对儿童血友病家庭治疗和护理行为进行测评比较,判断干预效果。结果干预后血友病患儿家庭的治疗和护理行为的测评显示,22个具体行为中,19个行为明显改善,干预前后比较,差异有统计学意义（χ2值分别为59．88,36．47,96．30,13．57,34．67,55．06,55．06,21．85,22．69,45．69,39．33,68．25,11．99,35．39,35．39,17．24,81．51,85．75,13．57;P〈0．01）;“准确记录每一次出血及处置经过”由干预前的2例（3．85％）上升至干预后的48例（92．31％）,“按月向血友病护士报告出血及治疗护理信息”由干预前的5例（20％）上升至干预后的52例（100％）,干预前后比较,差异均有统计学意义（χ2值分别为81．51,85．75;P〈0．01）。结论综合性护理干预措施可以在改善儿童血友病家庭治疗和护理的行为的同时实现血友病诊疗中心对血友病患儿病例信息的有效管理,适合在我国地方血友病诊疗中心开展。     

Factor concentrate usage in persons with hemophilia in New York State

BACKGROUND: Persons with a congenital deficiency of FVIII or F IX (hemophilia A and hemophilia B, respectively) receive factor concentrate to treat or prevent bleeding. STUDY DESIGN AND METHODS: A population-based study of all persons with hemophilia residing in New York State at any time during 1993 through 1998 was conducted. All available medical records for each patient were reviewed to determine type of therapy. RESULTS: Case finding yielded 1160 cases, for a prevalence of 63.9 per 1 million population in 1998. Recombinant factor concentrates were used by 56 percent of patients. Patients with severe disease used more (158,234 IU/patient) factor concentrate than did patients with moderate disease (46,315 IU) or mild disease (5794 IU). Over half (57%) of all factor concentrate was prescribed for patients with severe disease on prophylactic therapy. Patients undergoing immune tolerance therapy used the most per person-455,116 IU each. Hemophilia treatment centers provided factor concentrate for 62 percent of all patients who used factor and 73 percent of patients with severe disease. CONCLUSION: Hemophilia patients, especially patients with severe disease, use large amounts of expensive factor concentrates to prevent and to treat bleeding episodes. Specialized hemophilia treatment centers play a key role in the care of these patients.     

本体感觉训练联合肌内效贴对全膝关节置换术后患者膝关节功能与本体感觉的影响

目的:探讨本体感觉训练联合肌内效贴对全膝关节置换术后患者膝关节功能与本体感觉的影响.方法:将68例全膝关节置换术后患者随机分为本体感觉组和联合治疗组各34例.2组患者均进行基础治疗,并在治疗前及治疗4周后,分别采用膝关节评分(KSS)、"起立-行走"计时测试(TUGT)、主动关节角度重现偏差值进行评定,并评价术后6个月...     

Human parvovirus B19 in young male patients with hemophilia A: associations with treatment product exposure and joint range-of-motion limitation

BACKGROUND: To evaluate the risk of human parvovirus B19 (B19) transmission in recombinant antihemophilic factor, the seroprevalence among 798 two- to seven-year-old boys with hemophilia was compared. Also, data collected on joints were used to assess relations between B19 serostatus and joint range-of-motion (ROM) limitation. STUDY DESIGN AND METHODS: Staff at US hemophilia treatment centers collected data on product exposures and ROM of 10 joints and provided blood specimens as part of blood safety surveillance. Blood was tested for immunoglobulin G anti-B19. Associations between B19 seropositivity and treatment products and joint ROM limitations were examined in multivariate analyses. RESULTS: Compared to children who received no product, the odds of B19 seropositivity were 0.8 (p = 0.5), 1.9 (p = 0.05), and 7.6 (p < 0.001) for those children who received recombinant antihemophilic factor only, both recombinant antihemophilic factor and plasma-derived factor, and plasma-derived factor only, respectively. Children who were anti-B19 positive had an average 8 degrees less overall ROM (p = 0.002) than those who were B19 antibody negative after adjustment for other risk factors. CONCLUSION: The risk of B19 transmission by recombinant antihemophilic factor is low. Previous B19 infection is associated with ROM limitations in very young male patients with hemophilia. Virus inactivation techniques effective against B19 and other nonenveloped viruses are needed.     

Children's knowledge of illness and treatment experiences in hemophilia.

The purpose of this study was to describe what children with hemophilia understand about their unique illness and treatment experiences. Subjects included 20 children with hemophilia between the ages of 6 years and 13 years who had no evidence of cognitive problems. Following the principles of grounded theory, data about children's experiences of hemophilia and their understanding of these experiences were obtained through a semistructured interview using five pictorial stimuli representing illness and treatment domains. Data analysis was conducted using the constant comparative method. Children's knowledge was described in regard to (a) the nature of hemophilia and its origin, (b) the major illness experience: bleeding, (c) the nature of treatment and its purpose, and (d) the major treatment experience: poking. Findings indicated that although school-aged children with hemophilia were engaged actively in gaining knowledge about their disease and treatment, the overall level of understanding of children aged 6 years to 13 years did not follow a distinct path of development. Major areas of knowledge deficiency included knowledge about how one contracts hemophilia and the purpose of treatment.     

Tailored prophylaxis in children with severe hemophilia: A four-year Iranian study

BackgroundProphylaxis the current standard care for patients with severe hemophilia should be planned to optimize the replacement therapy and minimize bleeding. We report our single-center experience of tailored prophylaxis in children affected by hemophilia A (HA) and hemophilia B (HB).MethodsThis study was conducted on 55 patients, under 15 years, with HA (PWHA, n: 46) and HB (PWHB, n: 9) between 2015 and 2019. According to the phenotype, three prophylaxis regimens: 25−50 unit/kg once, twice, or three-times a week for PWHA, and two: 30−50 unit/kg once or twice a week for PWHB were administered. Following the occurrence of > 3 joint bleeding, or > 4 soft tissue bleeding, or one spontaneous major bleeding in the last 3 months, the prophylaxis regimen is changed. Annualized bleeding rate (ABR), annualized joint bleeding rate (AJBR), target joints development, inhibitor development, and hemophilia joint health score (HJHS) also were assessed.ResultsA mean ± SD of 2520 ± 1045 IU/kg/yr coagulation factor (F) VIII was used to reduce ABR and AJBR from 1.02 ± 1.11 and 0.8 ± 1.3 (in the first year of the study) to 0.27 ± 0.44 (P < 0.001) and 0.19 ± 0.38 (P = 0.004) (at the end of the study) in PWHA, respectively. Furthermore, in PWHB, in the first year of the study, with using 2168 ± 1216 IU/kg coagulation FIX, ABR and AJBR were 0.19 ± 0.39 and 0.06 ± 0.1. At the end of the study, ABR and AJBR were 0.02 ± 0.05 (p = 0.156) and 0.01 ± 0.03 (p = 0.361), respectively. During the study period, the mean number of the target joints and mean HJHS were 0.25 ± 0.57 and 7.6 ± 2.1 for PWHA and 0 and 6.3 ± 1.8 for PWHB, respectively.Finally, 5 PWHA (11 %) did not need dose-escalation in their prophylaxis regimen, whereas 31 (67 %) and 10 (21 %) PWHA needed two and three infusions a week, respectively. In PWHB, 7 (78 %) and 2 (22 %) were adjusted to receive a once and twice weekly regimen, respectively.ConclusionOur results suggest that tailored prophylaxis is an effective strategy to reduce the rate of bleeding and optimize the replacement therapy in children with hemophilia.     

Development of long-acting recombinant FVIII and FIX Fc fusion proteins for the management of hemophilia

Introduction: Prophylactic treatment with replacement clotting factor is the recommended regimen for patients with severe hemophilia to prevent bleeding episodes. However, currently available replacement clotting factors are limited by their relatively short half-lives and require intravenous injections up to three times weekly to maintain protective levels, which can impact compliance and, thus, patient outcomes.

Areas covered: The potential advantages of long-acting coagulation factors, including reduced injection frequency, increased treatment adherence, and improved clinical outcomes, are discussed. Fragment crystallizable (Fc) fusion technology is introduced and the development of long-acting recombinant factor VIII Fc (rFVIIIFc) and recombinant factor IX Fc (rFIXFc) fusion proteins for the treatment of hemophilia A and B, respectively, are described. Preclinical and clinical studies of rFVIIIFc and rFIXFc showing improved pharmacokinetics over currently available products are reviewed.

Expert opinion: Long-acting coagulation factors, including rFVIIIFc and rFIXFc, have the potential to change current paradigms of care for hemophilia A and B, respectively. Less frequent infusions may provide prolonged protection from bleeding and bleed resolution with fewer injections. In addition, long-acting coagulation factors provide an opportunity for improved individualized treatment for hemophilia.     

中西医结合预防高危人流术后宫腔粘连的临床观察

目的:观察中西医结合预防高危人流术后宫腔粘连的临床疗效。方法:选取30例普通人工流产术后患者为普通人流对照组(A组),90例高危人工流产术后患者随机分为高危人流对照组(B组)、西药对照组(C组)和中西药治疗组(D组),每组30例。4组均给予常规抗生素预防感染,C组术后给予补佳乐联合黄体酮人工周期药物治疗,D组在西药对照组的基础上给予自拟补肾活血方治疗。比较4组术后阴道流血时间、月经复潮时间以及月经复潮前子宫内膜厚度,比较4组术后第3个月的宫腔粘连情况。结果:C、D两组阴道流血时间、月经复潮时间均少于常规组和B组,D组阴道流血时间、月经复潮时间少于C组,差异均具有统计学意义(P<0.05);A组、B组和C组月经来潮前B超子宫内膜厚度均小于D组(P<0.05);A组、C组和D组术后宫腔粘连发生率均低于B组,D组术后宫腔粘连发生率低于C组,差异具有统计学意义(P<0.05)。结论:中西医结合治疗能增加子宫内膜厚度,显著缩短术后阴道流血时间、月经复潮时间,恢复月经周期,修复子宫创面,减少术后宫腔粘连发生率,促进子宫复旧。