- Email: [email protected]
China's first rare-disease registry is under development
Correspondence
Most events were low-grade and manageable. Further details are being assessed. The risk of osteonecrosis of the jaw is to be placed in context not only with the benefits to overall survival and progression-free survival, but also with the fact that zoledronic acid significantly lowered the risk of skeletal-related events versus clodronic acid throughout the course of the trial (proportion of patients with at least one skeletal-related event was 35% for clodronic acid and 27% for zoledronic acid, meaning that patients were more than twice as likely to have a skeletal-related event prevented than they were to develop osteonecrosis of the jaw with zoledronic acid versus clodronic acid).3,4 When Myeloma IX was started, there were limited data on the factors contributing to the risk of vascular thrombotic events (VTE) in myeloma patients. This is now a widely recognised issue, and effective thromboprophylactic regimens have been developed.5 Patients with multiple myeloma have an inherently increased risk of VTE, as reflected by the 4·7% rate in patients treated with melphalan and prednisolone (two agents not typically associated with VTE) irrespective of bisphosphonate therapy in Myeloma IX. Rates were higher with the thalidomidecontaining regimens. In the intensive pathway, cyclophosphamide, vincristine, doxorubicin, and dexamethasone (CVAD), which was delivered by continuous infusion via an indwelling central catheter, was also associated with increased VTE rates (15% with clodronic acid vs 21% with zoledronic acid) but the types of events were different from those associated with the thalidomidecontaining regimens, with 39 centralline-associated thromboses and 77 peripheral events. Use of a central indwelling catheter to administer chemotherapy to patients with multiple myeloma is no longer a standard of care in the www.thelancet.com Vol 378 August 27, 2011
MRC institutions. Nonetheless, on the basis of these VTE data, it would seem prudent to avoid using the central line to deliver bisphosphonate therapy in institutions where indwelling catheters and the CVAD regimen are still used. Moreover, these data collectively underscore the importance of proactive implementation of the newer thromboprophylaxis regimens in patients with multiple myeloma, especially when administering regimens containing thalidomide and related agents or when central lines are used for systemic therapy. Further analyses of the current Myeloma IX study database are underway to assess additional questions on the efficacy and safety of the agents studied. Additionally, follow-up continues, and longerterm data will mature later this year, providing the opportunity for more insight into long-term outcomes. I have participated in advisory boards for, received payment for lectures and development of educational presentations from, and received travel support from Celgene, Novartis, Merck, and Johnson and Johnson.
Gareth J Morgan [email protected] Section of Haemato-Oncology, Institute of Cancer Research, Brookes Lawley Building, Sutton SM2 5NG, UK 1
2
3
4
5
Kyrgidis A, Tzellos TG. Safety and efficacy of zoledronic acid in multiple myeloma. Lancet 2011; 377: 2177–78. Tanimoto T, Matayoshi T, Yagasaki F, Takeuchi K, Kami M. Safety and efficacy of zoledronic acid in multiple myeloma. Lancet 2011; 377: 2178. Morgan GJ, Davies FE, Gregory WM, et al. First-line treatment with zoledronic acid as compared with clodronic acid in multiple myeloma (MRC Myeloma IX): a randomised controlled trial. Lancet 2010; 376: 1989–99. Morgan GJ, Child JA, Gregory WM, et al. Effects of zoledronic acid versus clodronic acid on skeletal morbidity in patients with newly diagnosed multiple myeloma (MRC Myeloma IX): secondary outcomes from a randomised controlled trial. Lancet Oncol 2011; 12: 743–52. Palumbo A, Cavo M, Bringhen S, et al. Aspirin, warfarin, or enoxaparin thromboprophylaxis in patients with multiple myeloma treated with thalidomide: a phase III, open-label, randomized trial. J Clin Oncol 2011; 29: 986–93.
China’s first rare-disease registry is under development Christopher Forrest and colleagues (March 26, p 1057)1 propose a global rare-disease registry across disorders and international boundaries to facilitate research, education, and outcome improvement. Because there are no existing registries in some countries, such as China, it is important to establish a forum for sharing registry development experiences before the global registry’s creation and operation. The development of the first raredisease registry in China was started in May, 2010, by the China-Dolls Care and Support Association—a patients’ advocacy group mainly for those with osteogenesis imperfecta.2 The webbased registry is aimed at connecting physicians and patients, disseminating knowledge and information, and collecting data on patients. However, at a volunteers’ web conference, many challenges were identified, including the registry’s structure and operating techniques. For the registry to be compatible with Forrest and colleagues’ model,1 it needs international collaboration and, particularly, assistance from the USA and the European Union where such registries have already been developed and put into operation. China is working towards raredisease legislation for more than 10 million people living with rare diseases.2 The China-Dolls Association has regular contact with about 3000 patients with nearly 30 rare diseases in China, of whom more than 1000 have osteogenesis imperfecta. Just like the large contributions from China to drug research and development for common diseases,3,4 we hope the first rare-disease database under development in China will contribute to the recruitment of patients and global clinical research for orphan drugs. 769
Correspondence
YOW is president of the China-Dolls Care and Support Association. YJZ is a volunteer for the association. The other authors declare that they have no conflicts of interest. The rare-disease registry is a component of the rare-disease network hospital—an awarded project of the “Core World” Commonwealth Innovation Award by the Intel (China) Corporation.
*Yan-Jun Zhang, Yi-Ou Wang, Liang Li, Jeff J Guo, Jing-Bo Wang [email protected] College of Pharmacy, University of Cincinnati Academic Health Centre, Cincinnati, OH 45267, USA (YJZ, JJG); Health Economics and Outcomes Research, Bristol-Myers Squibb (China) Investment, Shanghai, China (YJZ); China-Dolls Care and Support Association, Beijing, China (YOW); Department of Medical Genetics, School of Basic Medical Sciences, Southern Medical University, Guangzhou, Guangdong, China (LL); and Department of Ophthalmology, People’s Liberation Army 309 Hospital, Beijing, China (JBW) 1
2
3 4
Forrest CB, Bartek RJ, Rubinstein Y, Groft SC. The case for a global rare-diseases registry. Lancet 2011; 377: 1057–59. Wang JB, Guo JJ, Yang L, Zhang YD, Sun ZQ, Zhang YJ. Rare diseases and legislation in China. Lancet 2010; 375: 708–09. Hughes B. Evolving R&D for emerging markets. Nat Rev Drug Discov 2010; 9: 417–20. Qi J, Wang Q, Yu Z, Chen X, Wang F. Innovative drug R&D in China. Nat Rev Drug Discov 2011; 10: 333–34.
UK Royal Colleges and health service reform Richard Gardner/Rex Features
In a press release,1 the UK’s Royal College of Paediatrics and Child Health (RCPCH) welcomed the report by the National Health Service (NHS) Future Forum,2 which it considers “passes the test for children”. But does it actually pass the test for anyone? The Future Forum report was commissioned as part of a “listening exercise” prompted by deep scepticism from the UK public about the government’s plans for the NHS. These plans for “reform” involve the fostering of market competition on the unproven basis that this increases cost-efficiency. If you were fortunate enough to hear about a local meeting of the Future Forum you could apply to attend, but unless you were from the voluntary sector it seems you had little chance of gaining entry. The consultation then seemed to 770
be along the lines of “how can we present privatisation in an acceptable way?” In fact, what the Secretary of State for Health meant by “listening” was amply demonstrated after the vote of no confidence from the members of the Royal College of Nursing: he apologised that his poor communication skills had not allowed them to understand what was being proposed, and promised to try harder in future. It is all well and good that concerns about children expressed by the President of the RCPCH have been acknowledged, but the fundamental problem remains: the aim of the Health and Social Care Bill is to reduce spending on health care from taxation and to promote privatisation.3 Raising of new forms of funding will inevitably follow, through facilitating the introduction of private insurance and top-up fees for patients. This will be bad for children’s health simply because, other than for a rich minority who can afford to pay, it will be bad for everyone’s health. A look at the USA gives us a pretty good idea of things to come.4 Recent experience in the Netherlands also highlights the failings of private insurance competition while serving as a reminder to all (including “independent experts”) that, in health care, “policy intentions should not be confused with outcomes”.5 Is it not time that Royal Colleges abandoned their relationship of cosy intimacy with government and began fighting on behalf of patients? I declare that I have no conflicts of interest.
J W L Puntis [email protected] Paediatric Offices, General Infirmary at Leeds, Great George Street, Leeds LS1 3EX, UK 1
2
Royal College of Paediatrics and Child Health. Future Forum Report passes the test for children—now Government must do the same. http://www.rcpch.ac.uk/sites/default/files/ RCPCH%20press%20release.pdf (accessed July 8, 2011). NHS Future Forum. NHS Future Forum recommendations to Government. http:// www.dh.gov.uk/en/Publicationsandstatistics/ Publications/PublicationsPolicyAndGuidance/ DH_127443 (accessed Aug 10, 2011).
3
4
Peedell C. Further privatisation is inevitable under the proposed NHS reforms. BMJ 2011; 342: d2926. Sommers BD, Epstein AM. Why states are so miffed about Medicaid—economics, politics and the “woodwork effect”. N Engl J Med 2011;
365: 100–02. 5
Okma KGH, Marmor TR, Oberlander J. Managed competition for Medicare? Sobering lessons from the Netherlands. N Engl J Med 2011; 365: 287–89.
Department of Error Saraiya M, Hariri S. HPV vaccine effect: is the glass half full or half empty? Lancet 2011; 377: 2057–58—In this Comment (June 18), the second sentence should have read: “The study reports a 0·38% absolute decrease in the rates of histologically confirmed high-grade cervical lesions in these girls during the 2 years after HPV vaccine introduction compared with the 3 years before vaccine introduction.” The seventh sentence should have read: “With the 47·5% relative decrease in the incidence of high-grade cervical abnormalities...”. These corrections have been made to the online version as of Aug 26, 2011. Syed-Abdul S, Lin CW, Scholl J, et al. Facebook use leads to health-care reform in Taiwan. Lancet 2011; 377: 2083–84—In this Correspondence (June 18), the final affiliation in the address box should have appeared as follows: “Graduate Institute of Biomedical Informatics, Taipei Medical University, Taipei 110, Taiwan (SSA, YCL).” This correction has been made to the online version as of Aug 26, 2011. Mansfield SJ, Bonning MA, Morrison SG, et al. Social networking and health. Lancet 2011; 377: 2083—In this Correspondence (June 18), the fifth author’s name should have been spelled “Sheng-Hui Wang”. This correction has been made to the online version as of Aug 26, 2011. Loewenberg S. Humanitarian response inadequate in Horn of Africa crisis. Lancet 2011; 378: 555–58—In this Special Report (Aug 13), the image caption on p 558 should have read: “Mathias Wamoto says that many children are malnourished in Kursin, Wajir”. The fourth paragraph on p 558 should have read: “Five children a month are dying of malnutrition at Wajir hospital, said Mohamed Hassan, and that does not count the many more who never get care at all.” The third sentence of the eighth paragraph on p 558 should have read: “Those most affected are pastoral communities, overwhelmingly poor people who have no buffer for the two seasons of drought, inflation of 12%, and skyrocketing food prices of more than 200%.” These corrections have been made to the online version as of Aug 26, 2011.
www.thelancet.com Vol 378 August 27, 2011
Most events were low-grade and manageable. Further details are being assessed. The risk of osteonecrosis of the jaw is to be placed in context not only with the benefits to overall survival and progression-free survival, but also with the fact that zoledronic acid significantly lowered the risk of skeletal-related events versus clodronic acid throughout the course of the trial (proportion of patients with at least one skeletal-related event was 35% for clodronic acid and 27% for zoledronic acid, meaning that patients were more than twice as likely to have a skeletal-related event prevented than they were to develop osteonecrosis of the jaw with zoledronic acid versus clodronic acid).3,4 When Myeloma IX was started, there were limited data on the factors contributing to the risk of vascular thrombotic events (VTE) in myeloma patients. This is now a widely recognised issue, and effective thromboprophylactic regimens have been developed.5 Patients with multiple myeloma have an inherently increased risk of VTE, as reflected by the 4·7% rate in patients treated with melphalan and prednisolone (two agents not typically associated with VTE) irrespective of bisphosphonate therapy in Myeloma IX. Rates were higher with the thalidomidecontaining regimens. In the intensive pathway, cyclophosphamide, vincristine, doxorubicin, and dexamethasone (CVAD), which was delivered by continuous infusion via an indwelling central catheter, was also associated with increased VTE rates (15% with clodronic acid vs 21% with zoledronic acid) but the types of events were different from those associated with the thalidomidecontaining regimens, with 39 centralline-associated thromboses and 77 peripheral events. Use of a central indwelling catheter to administer chemotherapy to patients with multiple myeloma is no longer a standard of care in the www.thelancet.com Vol 378 August 27, 2011
MRC institutions. Nonetheless, on the basis of these VTE data, it would seem prudent to avoid using the central line to deliver bisphosphonate therapy in institutions where indwelling catheters and the CVAD regimen are still used. Moreover, these data collectively underscore the importance of proactive implementation of the newer thromboprophylaxis regimens in patients with multiple myeloma, especially when administering regimens containing thalidomide and related agents or when central lines are used for systemic therapy. Further analyses of the current Myeloma IX study database are underway to assess additional questions on the efficacy and safety of the agents studied. Additionally, follow-up continues, and longerterm data will mature later this year, providing the opportunity for more insight into long-term outcomes. I have participated in advisory boards for, received payment for lectures and development of educational presentations from, and received travel support from Celgene, Novartis, Merck, and Johnson and Johnson.
Gareth J Morgan [email protected] Section of Haemato-Oncology, Institute of Cancer Research, Brookes Lawley Building, Sutton SM2 5NG, UK 1
2
3
4
5
Kyrgidis A, Tzellos TG. Safety and efficacy of zoledronic acid in multiple myeloma. Lancet 2011; 377: 2177–78. Tanimoto T, Matayoshi T, Yagasaki F, Takeuchi K, Kami M. Safety and efficacy of zoledronic acid in multiple myeloma. Lancet 2011; 377: 2178. Morgan GJ, Davies FE, Gregory WM, et al. First-line treatment with zoledronic acid as compared with clodronic acid in multiple myeloma (MRC Myeloma IX): a randomised controlled trial. Lancet 2010; 376: 1989–99. Morgan GJ, Child JA, Gregory WM, et al. Effects of zoledronic acid versus clodronic acid on skeletal morbidity in patients with newly diagnosed multiple myeloma (MRC Myeloma IX): secondary outcomes from a randomised controlled trial. Lancet Oncol 2011; 12: 743–52. Palumbo A, Cavo M, Bringhen S, et al. Aspirin, warfarin, or enoxaparin thromboprophylaxis in patients with multiple myeloma treated with thalidomide: a phase III, open-label, randomized trial. J Clin Oncol 2011; 29: 986–93.
China’s first rare-disease registry is under development Christopher Forrest and colleagues (March 26, p 1057)1 propose a global rare-disease registry across disorders and international boundaries to facilitate research, education, and outcome improvement. Because there are no existing registries in some countries, such as China, it is important to establish a forum for sharing registry development experiences before the global registry’s creation and operation. The development of the first raredisease registry in China was started in May, 2010, by the China-Dolls Care and Support Association—a patients’ advocacy group mainly for those with osteogenesis imperfecta.2 The webbased registry is aimed at connecting physicians and patients, disseminating knowledge and information, and collecting data on patients. However, at a volunteers’ web conference, many challenges were identified, including the registry’s structure and operating techniques. For the registry to be compatible with Forrest and colleagues’ model,1 it needs international collaboration and, particularly, assistance from the USA and the European Union where such registries have already been developed and put into operation. China is working towards raredisease legislation for more than 10 million people living with rare diseases.2 The China-Dolls Association has regular contact with about 3000 patients with nearly 30 rare diseases in China, of whom more than 1000 have osteogenesis imperfecta. Just like the large contributions from China to drug research and development for common diseases,3,4 we hope the first rare-disease database under development in China will contribute to the recruitment of patients and global clinical research for orphan drugs. 769
Correspondence
YOW is president of the China-Dolls Care and Support Association. YJZ is a volunteer for the association. The other authors declare that they have no conflicts of interest. The rare-disease registry is a component of the rare-disease network hospital—an awarded project of the “Core World” Commonwealth Innovation Award by the Intel (China) Corporation.
*Yan-Jun Zhang, Yi-Ou Wang, Liang Li, Jeff J Guo, Jing-Bo Wang [email protected] College of Pharmacy, University of Cincinnati Academic Health Centre, Cincinnati, OH 45267, USA (YJZ, JJG); Health Economics and Outcomes Research, Bristol-Myers Squibb (China) Investment, Shanghai, China (YJZ); China-Dolls Care and Support Association, Beijing, China (YOW); Department of Medical Genetics, School of Basic Medical Sciences, Southern Medical University, Guangzhou, Guangdong, China (LL); and Department of Ophthalmology, People’s Liberation Army 309 Hospital, Beijing, China (JBW) 1
2
3 4
Forrest CB, Bartek RJ, Rubinstein Y, Groft SC. The case for a global rare-diseases registry. Lancet 2011; 377: 1057–59. Wang JB, Guo JJ, Yang L, Zhang YD, Sun ZQ, Zhang YJ. Rare diseases and legislation in China. Lancet 2010; 375: 708–09. Hughes B. Evolving R&D for emerging markets. Nat Rev Drug Discov 2010; 9: 417–20. Qi J, Wang Q, Yu Z, Chen X, Wang F. Innovative drug R&D in China. Nat Rev Drug Discov 2011; 10: 333–34.
UK Royal Colleges and health service reform Richard Gardner/Rex Features
In a press release,1 the UK’s Royal College of Paediatrics and Child Health (RCPCH) welcomed the report by the National Health Service (NHS) Future Forum,2 which it considers “passes the test for children”. But does it actually pass the test for anyone? The Future Forum report was commissioned as part of a “listening exercise” prompted by deep scepticism from the UK public about the government’s plans for the NHS. These plans for “reform” involve the fostering of market competition on the unproven basis that this increases cost-efficiency. If you were fortunate enough to hear about a local meeting of the Future Forum you could apply to attend, but unless you were from the voluntary sector it seems you had little chance of gaining entry. The consultation then seemed to 770
be along the lines of “how can we present privatisation in an acceptable way?” In fact, what the Secretary of State for Health meant by “listening” was amply demonstrated after the vote of no confidence from the members of the Royal College of Nursing: he apologised that his poor communication skills had not allowed them to understand what was being proposed, and promised to try harder in future. It is all well and good that concerns about children expressed by the President of the RCPCH have been acknowledged, but the fundamental problem remains: the aim of the Health and Social Care Bill is to reduce spending on health care from taxation and to promote privatisation.3 Raising of new forms of funding will inevitably follow, through facilitating the introduction of private insurance and top-up fees for patients. This will be bad for children’s health simply because, other than for a rich minority who can afford to pay, it will be bad for everyone’s health. A look at the USA gives us a pretty good idea of things to come.4 Recent experience in the Netherlands also highlights the failings of private insurance competition while serving as a reminder to all (including “independent experts”) that, in health care, “policy intentions should not be confused with outcomes”.5 Is it not time that Royal Colleges abandoned their relationship of cosy intimacy with government and began fighting on behalf of patients? I declare that I have no conflicts of interest.
J W L Puntis [email protected] Paediatric Offices, General Infirmary at Leeds, Great George Street, Leeds LS1 3EX, UK 1
2
Royal College of Paediatrics and Child Health. Future Forum Report passes the test for children—now Government must do the same. http://www.rcpch.ac.uk/sites/default/files/ RCPCH%20press%20release.pdf (accessed July 8, 2011). NHS Future Forum. NHS Future Forum recommendations to Government. http:// www.dh.gov.uk/en/Publicationsandstatistics/ Publications/PublicationsPolicyAndGuidance/ DH_127443 (accessed Aug 10, 2011).
3
4
Peedell C. Further privatisation is inevitable under the proposed NHS reforms. BMJ 2011; 342: d2926. Sommers BD, Epstein AM. Why states are so miffed about Medicaid—economics, politics and the “woodwork effect”. N Engl J Med 2011;
365: 100–02. 5
Okma KGH, Marmor TR, Oberlander J. Managed competition for Medicare? Sobering lessons from the Netherlands. N Engl J Med 2011; 365: 287–89.
Department of Error Saraiya M, Hariri S. HPV vaccine effect: is the glass half full or half empty? Lancet 2011; 377: 2057–58—In this Comment (June 18), the second sentence should have read: “The study reports a 0·38% absolute decrease in the rates of histologically confirmed high-grade cervical lesions in these girls during the 2 years after HPV vaccine introduction compared with the 3 years before vaccine introduction.” The seventh sentence should have read: “With the 47·5% relative decrease in the incidence of high-grade cervical abnormalities...”. These corrections have been made to the online version as of Aug 26, 2011. Syed-Abdul S, Lin CW, Scholl J, et al. Facebook use leads to health-care reform in Taiwan. Lancet 2011; 377: 2083–84—In this Correspondence (June 18), the final affiliation in the address box should have appeared as follows: “Graduate Institute of Biomedical Informatics, Taipei Medical University, Taipei 110, Taiwan (SSA, YCL).” This correction has been made to the online version as of Aug 26, 2011. Mansfield SJ, Bonning MA, Morrison SG, et al. Social networking and health. Lancet 2011; 377: 2083—In this Correspondence (June 18), the fifth author’s name should have been spelled “Sheng-Hui Wang”. This correction has been made to the online version as of Aug 26, 2011. Loewenberg S. Humanitarian response inadequate in Horn of Africa crisis. Lancet 2011; 378: 555–58—In this Special Report (Aug 13), the image caption on p 558 should have read: “Mathias Wamoto says that many children are malnourished in Kursin, Wajir”. The fourth paragraph on p 558 should have read: “Five children a month are dying of malnutrition at Wajir hospital, said Mohamed Hassan, and that does not count the many more who never get care at all.” The third sentence of the eighth paragraph on p 558 should have read: “Those most affected are pastoral communities, overwhelmingly poor people who have no buffer for the two seasons of drought, inflation of 12%, and skyrocketing food prices of more than 200%.” These corrections have been made to the online version as of Aug 26, 2011.
www.thelancet.com Vol 378 August 27, 2011