PDF(Pubmed)
Abstract：
UNASSIGNED: Increased homogentisic acid (HGA) in alkaptonuria (AKU) causes severe arthritis. Nitisinone reduces the production of HGA, but whether it also decreases arthroplasty was examined in 237 AKU patients.
UNASSIGNED: Patients attending the United Kingdom National Alkaptonuria Centre (NAC) and the Suitability of Nitisinone in Alkaptonuria 2 (SONIA 2) study were studied. Assessments included questionnaires eliciting details of arthroplasty. Nitisinone was administered from baseline, 2 mg in the NAC and 10 mg in SONIA 2. In SONIA 2, subgroups consisted of those with baseline arthroplasty on and not on nitisinone (BR + N+, BR + N-), as well as those without baseline arthroplasty on and not on nitisinone (BR-N+, BR-N-).
UNASSIGNED: In the SONIA2 subgroups, new joint replacement (JR) probabilities after baseline were significantly different (BR + N+, BR + N-, BR-N+, BR-N-) (χ2 = 23.3, p < 0.001); mean (SD) was 3.8 (0.1) years in BR-N-, 3.7 (0.1) years in BR-N+, 3.4 (0.3) years in BR + N-, and 3.0 (0.3) years in BR + N+. Further, the BR + N- showed more JR than the BR-N- subgroup (p < 0.01), while BR + N+ similarly showed more JR than the BR-N+ subgroup (p < 0.001).In the NAC, the BR- group had a mean age of 51.6 (7.0) years at baseline but 57.7 (8.7) years at final follow up during nitisinone therapy and showed only 7 incident JR. The BR+ group had an age at baseline of 57.4 (8.5) years and had undergone 94 JRs at baseline.
UNASSIGNED: The incidence of arthroplasty was earlier and more frequent after the first JR and was not affected by nitisinone.
UNASSIGNED: Patients attending the United Kingdom National Alkaptonuria Centre (NAC) and the Suitability of Nitisinone in Alkaptonuria 2 (SONIA 2) study were studied. Assessments included questionnaires eliciting details of arthroplasty. Nitisinone was administered from baseline, 2 mg in the NAC and 10 mg in SONIA 2. In SONIA 2, subgroups consisted of those with baseline arthroplasty on and not on nitisinone (BR + N+, BR + N-), as well as those without baseline arthroplasty on and not on nitisinone (BR-N+, BR-N-).
UNASSIGNED: In the SONIA2 subgroups, new joint replacement (JR) probabilities after baseline were significantly different (BR + N+, BR + N-, BR-N+, BR-N-) (χ2 = 23.3, p < 0.001); mean (SD) was 3.8 (0.1) years in BR-N-, 3.7 (0.1) years in BR-N+, 3.4 (0.3) years in BR + N-, and 3.0 (0.3) years in BR + N+. Further, the BR + N- showed more JR than the BR-N- subgroup (p < 0.01), while BR + N+ similarly showed more JR than the BR-N+ subgroup (p < 0.001).In the NAC, the BR- group had a mean age of 51.6 (7.0) years at baseline but 57.7 (8.7) years at final follow up during nitisinone therapy and showed only 7 incident JR. The BR+ group had an age at baseline of 57.4 (8.5) years and had undergone 94 JRs at baseline.
UNASSIGNED: The incidence of arthroplasty was earlier and more frequent after the first JR and was not affected by nitisinone.
摘要：
■在碱尿（AKU）中增加的均质酸（HGA）会导致严重的关节炎。Nitisinone减少了HGA的产生，但是在237例AKU患者中检查了它是否也减少了关节成形术。
■研究了参加英国国家碱性尿症中心（NAC）的患者以及Nitisinone在碱性尿症2（SONIA2）研究中的适用性。评估包括引发关节成形术细节的问卷。Nitisinone从基线开始给药，2mg在NAC和10mg在SONIA2。在SONIA2中，亚组包括那些在基线上进行关节成形术而不是在nitisinone上进行关节成形术的亚组（BRN，BR+N-),以及那些没有基线关节成形术而没有Nitisinone(BR-N+，BR-N-）。
■在SONIA2子群中，基线后新关节置换(JR)概率有显著差异(BR+N+，BR+N-,BR-N+,BR-N-）（χ2=23.3，p<0.001）；BR-N-的平均值（SD）为3.8（0.1）年，以BR-N+为单位的3.7(0.1)年，3.4（0.3）年的BR+N-，以BR+N+计算3.0（0.3）年。Further,BR+N-比BR-N-亚组显示更多的JR（p<0.01），而BR+N+相似地显示出比BR-N+亚组更多的JR(p<0.001)。在NAC中,BR-组在基线时的平均年龄为51.6(7.0)岁,但在尼替辛酮治疗期间的最后一次随访时的平均年龄为57.7(8.7)岁,仅出现7例JR事件.BR+组的基线年龄为57.4（8.5）岁，基线时经历了94次JR。
■在第一次JR后，关节成形术的发生率更早，更频繁，并且不受尼替辛酮的影响。
■研究了参加英国国家碱性尿症中心（NAC）的患者以及Nitisinone在碱性尿症2（SONIA2）研究中的适用性。评估包括引发关节成形术细节的问卷。Nitisinone从基线开始给药，2mg在NAC和10mg在SONIA2。在SONIA2中，亚组包括那些在基线上进行关节成形术而不是在nitisinone上进行关节成形术的亚组（BRN，BR+N-),以及那些没有基线关节成形术而没有Nitisinone(BR-N+，BR-N-）。
■在SONIA2子群中，基线后新关节置换(JR)概率有显著差异(BR+N+，BR+N-,BR-N+,BR-N-）（χ2=23.3，p<0.001）；BR-N-的平均值（SD）为3.8（0.1）年，以BR-N+为单位的3.7(0.1)年，3.4（0.3）年的BR+N-，以BR+N+计算3.0（0.3）年。Further,BR+N-比BR-N-亚组显示更多的JR（p<0.01），而BR+N+相似地显示出比BR-N+亚组更多的JR(p<0.001)。在NAC中,BR-组在基线时的平均年龄为51.6(7.0)岁,但在尼替辛酮治疗期间的最后一次随访时的平均年龄为57.7(8.7)岁,仅出现7例JR事件.BR+组的基线年龄为57.4（8.5）岁，基线时经历了94次JR。
■在第一次JR后，关节成形术的发生率更早，更频繁，并且不受尼替辛酮的影响。
