Treatment for avascular necrosis of bone in people with sickle cell disease

 

Abstract

Background

Avascular necrosis of bone is a frequent and severe complication of sickle cell disease (SCD) and its treatment is not standardised.

Objectives

To determine the impact of any surgical procedure compared with other surgical interventions or non-surgical procedures, on avascular necrosis of bone in people with SCD in terms of efficacy and safety.

Search methods

We searched the Cochrane Cystic Fibrosis and Genetic Disorders Group Haemoglobinopathies Trials Register, comprising references identified from comprehensive electronic database searches and handsearches of relevant journals and abstract books of conference proceedings. Additional trials were sought from the reference lists of papers identified by the search strategy.

Most recent search: 27 November 2009.

Selection criteria

Randomised clinical trials comparing specific therapies for avascular necrosis of bone in people with SCD.

Data collection and analysis

Each author independently extracted data and assessed trial quality. Since only one trial was identified, meta-analysis was not possible.

Main results

One trial (46 participants) was eligible for inclusion. After randomisation eight participants were withdrawn, mainly because they declined to participate in the trial. Data were analysed for 38 participants at the end of the trial. After a mean follow up of three years, hip core decompression and physical therapy did not show clinical improvement when compared with physical therapy alone using the score from the original trial (an improvement of 18.1 points for those treated with intervention therapy versus an improvement of 15.7 points with control therapy). There was no significant statistical difference between groups regarding major complications (hip pain, relative risk (RR) 0.95 (95% confidence interval (CI) 0.56 to 1.60; vaso-occlusive crises, RR 1.14 (95% CI 0.72 to 1.80); and acute chest syndrome, RR 1.06 (95% CI 0.44 to 2.56)). This trial did not report results on mortality or quality of life.

Authors' conclusions

We found no evidence that adding hip core decompression to physical therapy achieves clinical improvement in people with SCD with avascular necrosis of bone compared to physical therapy alone. However, we highlight that our conclusion is based on one trial with high attrition rates. Further randomised controlled trials are necessary to evaluate the role of hip-core depression for this clinical condition. Endpoints should focus on participants' subjective experience (e.g. quality of life and pain) as well as more objective 'time-to-event' measures (e.g. mortality, survival, hip longevity). The availability of participants to allow adequate trial power will be a key consideration for endpoint choice.

 

Plain language summary

Treatments for people with sickle cell disease in whom poor blood supply to an area of bone leads to bone death

Many people with sickle cell disease experience bone death due to temporary or permanent loss of blood supply to parts of their bones. This can be very painful. The bones usually affected are the thigh bones at the hip joint and the arm bones at the shoulder joint. The aim of treatment is to stop the pain and maintain a mobile joint. Treatments include resting the joint, physiotherapy, the use of pain relief, joint replacements and bone grafts. However, complications from surgery may be more frequent in people with sickle cell disease. We found one eligible trial which analysed data from 38 people from 32 different treatment centres. The trial compared a treatment of surgery and physical therapy to physical therapy on its own. This trial did not show that the addition of surgery to a physical therapy regimen could improve the outcome for people with sickle cell disease and avascular necrosis. However, the strength of this trial’s findings were weakened by a number of participants who could not complete the treatment. Further trials are needed which will look at the long-term outcomes of different treatments for this condition. Trialists should consider including endpoints that focus on the subjective experience of participants (such as quality of life, and pain) as well as more objective such as mortality, survival, or hip longevity. The availability of participants to allow adequate trial power will be a key consideration during endpoint choice.

 

摘要

背景

鐮刀型貧血患者的骨頭缺血性壞死治療(avascular necrosis of bone with sickle cell disease)

骨頭缺血性壞死(avascular necrosis of bone)是鐮刀型貧血(sickle cell disease)常見的嚴重併發症，目前還沒有標準治療方法。

目標

對於患有缺血性壞死的鐮刀型貧血病人，評估其接受手術或非手術治療的效果與安全性。

搜尋策略

我們搜尋了Cochrane Cystic Fibrosis and Genetic Disorders Group Haemoglobinopathies Trials Register，其中包含電子資料庫、相關期刊與會議紀錄摘要。用新方法搜尋到的文獻參考資料中發現新的試驗。 最新的搜尋是：2009年3月27日。

選擇標準

比較在鐮刀型貧血患者使用各種骨頭缺血性壞死治療的臨床隨機對照試驗。

資料收集與分析

我們分別對每個作者的資料進行分析。因只找到一個試驗，無法進行統合分析。

主要結論

有一個有46名受試者的試驗符合收納標準。在隨機分組後有8位受試者不想繼續參與試驗而退出，試驗結果以最後留下的38名受試者資料來分析。 經過三年的追蹤，髖部減壓術(hip core decompression)加上物理治療(physical therapy)的臨床效果，在試驗當初設定的指標上沒有比單純使用物理治療更好。(前者增加了18.1分，對照組增加了15.7分) 在主要併發症上並沒有達到統計顯著差異。(髖部痛，相對風險0.95，信賴區間0.56−1.60)、(血管阻塞危機vasoocclusive crises，相對風險1.14，信賴區間0.72−1.80)、(急性胸痛症候群acute chest syndrome，相對風險1.06，信賴區間0.44−2.56) 本試驗並沒有死亡或是生活品質quality of life的報告。

作者結論

對於鐮刀型貧血骨頭缺血性壞死，沒有證據說支持髖部減壓術加上物理治療會比單純使用物理治療更好。不過我們的結論是建立在很薄弱的單一臨床試驗之上。需要有更多的隨機臨床對照試驗來評估髖部減壓術的成效。試驗終點(Endpoint)應該要聚焦在受試者主觀的感受(例如生活品質、疼痛)，以及客觀的測量(如死亡率、存活率、髖部壽命)。是否有足夠的受試者使試驗達到適當的檢力(power)將會是試驗終點選擇的考量。

翻譯人

本摘要由臺灣大學附設醫院陳敬軒翻譯。

此翻譯計畫由臺灣國家衛生研究院(National Health Research Institutes, Taiwan)統籌。

總結

許多鐮刀型貧血患者承受因暫時或永久血液供應不足造成的骨頭壞死。那是相當痛的感受。通常受到影響的是髖關節的大腿骨還有肩關節的上臂骨。 治療的目標是消除疼痛以及維持關節的活動性。治療的方式包含讓關節休息、生理治療physiotherapy、止痛藥、關節置換以及骨頭移植。然而在鐮刀型貧血患者身上手術併發症會比較多。 我們找到了一個分析來自32間醫院的38位患者的薄弱試驗，這個試驗比較單獨物理治療與物理治療加上手術治療的效果。這個試驗顯示手術治療對於鏈刀型貧血骨頭缺血性壞死患者的整體預後並沒有更多的改善。 但是這個試驗結果的強度因為一些受試者的退出而減弱，需要更進一步的試驗了解長期的預後。試驗設計者應該要將主觀的感受(如生活品質、疼痛)，以及客觀的測量(如死亡率、存活率、髖部壽命)納入試驗終點。是否有足夠的受試者使試驗達到適當的檢力將會是試驗終點選擇的考量。