From Pharma Times
Three new drugs put forward for EU approval
The CHMP is also recommending approval of Jinarc (tolvaptan) to slow the progression of cyst development and renal insufficiency in certain patients with autosomal dominant polycystic kidney disease (ADPKD).
Because of the potential for liver toxicity, a pharmacovigilance plan for Jinarc will be implemented on approval, and it is also proposed that treatment be initiated and monitored under the supervision of physicians with expertise in managing ADPKD and a full understanding of the risks of this therapy.
From FDA.gov
FDA authorizes use of first device to treat patients with dialysis-related amyloidosis
The U.S. Food and Drug Administration today authorized use of Lixelle Beta 2-microglobulin Apheresis Column, the first device to treat dialysis-related amyloidosis (DRA).
Dialysis-related amyloidosis is a chronic, progressive condition caused by the buildup in the body of a protein called beta 2-microglobulin. Dialysis-related amyloidosis is a complication of kidney failure. As beta 2-microglobulin builds up in the blood, deposits of the protein can form in the bones, joints and tendons causing painful and stiff joints, bone cysts that can lead to bone fractures, and torn tendons and ligaments. Beta 2-microglobulin deposits can also affect the digestive tract and organs, such as the heart and lungs.
Dialysis-related amyloidosis most often occurs in patients with kidney failure, especially adults older than 60, who have been on hemodialysis for more than five years.
The Lixelle Column works by removing beta 2-microglobin from the blood. It contains porous cellulose beads specifically designed to bind to beta 2-microglobulin as the patient’s blood passes over the beads. The device is used in conjunction with hemodialysis, a treatment where blood circulates outside the body through a special filter that removes waste products and extra fluid. The clean blood is returned to the body. When the Lixelle Column is used, the blood passes through the Lixelle Column before it enters the dialysis filter.
The device may help patients who have developed symptoms related to DRA and may be especially useful for those patients who may not have access to extended dialysis therapies or who may not be eligible for a kidney transplant. [Read more]
From Santa Monica Daily Press, California, By Enrique Rivero
The event will focus on integrative medicine. It will feature interactive stations for kidney education, diet and healthy cooking, exercise, meditation and yoga, upcoming clinical trials and research, polycystic kidney disease, high blood pressure, Fabry disease, dialysis, and much more. Transplant coordinators, nephrologists and living kidney donors will be available to address questions and concerns related to transplantation and encourage organ donation and its benefit to society.
Two booths will be staffed by Spanish-speaking physicians and health care workers, who will provide BMI testing and other health resources. Children’s games and crafts, music and children’s dance performances will accentuate this festive event that is expected to draw nearly 1,000 guests.
The event will be held from 9 a.m. to noon at Santa Monica Beach Park #1 (Ocean Park Boulevard and Barnard Way).
Kidney Policies
From Roll Call, By Rep. Tom Marino
Celebrate National Kidney Month by Supporting Bipartisan Kidney Care Legislation
Approximately 50 years ago, kidney failure was a death sentence. Even considering that fact is unnerving. Today, more than 600,000 Americans are living with kidney failure — and a large majority of those lives are sustained by life-saving dialysis treatments. That’s hundreds of thousands of lives that would have been lost and hundreds of thousands of families that would have been broken apart before the invention and expansion of dialysis treatment.
In 1972, Congress developed the Medicare End-Stage Renal Disease benefit. In doing so, Congress ensured that regardless of age or income, any American would have access to life-saving dialysis care. That was the turning point in kidney care.
Now it’s time for this Congress to take the next step for those living with kidney disease by modernizing policies, improving care coordination, expanding patient choice and intensifying research. I am confident the Chronic Kidney Disease Improvement in Research and Treatment Act (HR 1130) can be instrumental in accomplishing these goals, and I am very proud to be the bill’s lead sponsor — but even more honored to have my friend and colleague Democrat John Lewis of Georgia standing by me in this effort as well.
The legislation is built on three primary tenets. First, for individuals living with chronic diseases, especially when those diseases are complicated by multiple co-morbid conditions, coordinated care is key to improving outcomes and lowering health care costs. Second, increased research can lead to a deeper understanding of kidney disease prevention and ultimately to significant innovations in treatment. Lastly, stability in the Medicare program is central to an ESRD program that ensures quality and produces optimal results.
Studies show promoting collaboration between primary physicians and specialists treating the same patients through coordinated care improves patient outcomes and reduces costs across the health delivery system. The coordinated care model is especially important for kidney dialysis patients — many of whom are living with multiple chronic conditions and have to work with multiple health care providers and health care settings — to improve the care experience for the patient, improve outcomes, and capture savings and efficiencies.
To spur the creation of a workable coordinated care program for dialysis patients, HR 1130 would establish a voluntary program to incentivize nephrologists and dialysis facilities to better align medical treatment. [Read more]
From Virtual Press Office
Complementary Senate and House bills introduced would improve lives of individuals with kidney disease
Kidney Care Partners (KCP), the nation's largest kidney advocacy coalition, today praised Senator Mike Crapo (R-ID) for co-sponsoring the "The Chronic Kidney Disease Improvement in Research and Treatment Act" (S. 598), a bipartisan Senate bill that improves care coordination, expands access, and promotes research to benefit more than 636,000 Americans living with kidney failure, which is known as end-stage renal disease (ESRD). A complementary bill by the same name (H.R. 1130) was simultaneously introduced in the House.
Currently, 31 million Americans have some form of kidney disease and are at risk of developing kidney failure absent some form of disease management education or preventive care. Each year, more than 100,000 Americans are diagnosed with ESRD – the final stage of CKD – and therefore require a kidney transplant or dialysis. Due to the limited number of kidneys available for transplantation, 430,000 Americans now rely on life-sustaining dialysis care to survive. In general, patients must undergo dialysis three times a week for several hours per treatment. Under current law, dialysis treatments are covered by the Medicare program, regardless of the individual's age.
Advocates have long stressed that federal policies are needed to provide patient choice and to ensure access to life-sustaining dialysis, to increase research into CKD, and to create stability in Medicare's crucial ESRD program. Ultimately, the legislation introduced by Representatives Tom Marino (R-PA), John Lewis (D-GA) and Peter Roskam (R-IL) in the House and Senators Ben Cardin (D-MD), Mike Crapo (R-ID) and Bill Nelson (D-FL) in the Senate would improve patient outcomes through care coordination, expand access to traditionally underserved patient populations, and set the U.S. on the path towards a cure through efficiently managed and coordinated biomedical research.
"This bill is absolutely vital as it provides a clear blueprint for the future of the ESRD program," said Dr. Ed Jones, Chairman of Kidney Care Partners and a practicing nephrologist. "By supporting care coordination, greater patient choice, coordinated research programs, and economic stability, this bill would improve the delivery of care for millions of Americans living with kidney disease. The kidney care community endorses this bill and applauds the leadership of Senator Crapo on this important health care issue."
Specifically, The Chronic Kidney Disease Improvement in Research and Treatment Act would:
(1) Improve the coordination of care: The legislation would expand care options for patients by, among other things, allowing individuals diagnosed with kidney failure to enroll in the Medicare Advantage program. Under current law, individuals who develop kidney failure are not permitted to enroll in Medicare Advantage (MA) plans despite the Medicare Payment Advisory Commission's recommendation to eliminate the restriction in order to provide ESRD beneficiaries with the same freedom of choice and access to improved coordinated services as other Medicare-enrolled individuals. Therefore, the legislation would allow ESRD patients to enroll in – and reap the benefits of – MA plans. The bill also would reauthorize on a permanent basis the Special Needs Plan (SNP) for patients with kidney failure who need additional care attention, as well as extend the length of time beneficiaries may choose to maintain their existing insurance coverage. Importantly, the legislation looks to the future by establishing a voluntary coordinated care program. The coordinated care program would allow physicians and dialysis facilities to work together to improve the coordination of care and reduce costly hospitalizations and rehospitalizations.
(2) Promote patient access and choice: The legislation would expand patient access to kidney disease education programs and home dialysis treatment options through telemedicine, as well as create incentives for nephrologists and other dialysis health care professionals to work in underserved rural or urban areas. The bills also would establish renal dialysis facilities as a cost-effective alternative to hospital outpatient departments for individuals diagnosed with acute kidney injury.
(3) Expand research and enhance coordination: The legislation would identify gaps in research and improve the coordination of federal research efforts. Specifically, the bills would require the GAO to assess the adequacy of federal funding for CKD research relative to the expenditures for CKD care and identify gaps in research. Additionally, the bills would require improved coordination among the various federal agencies conducting CKD research by requiring the development of a strategic plan. Third, the bills would require the Secretary to conduct a study to better understand the progression of kidney disease and treatment of kidney failure in minority populations. [Read more]
Gift of Life
From The Courier Mail, Queensland, Australia, by HALL, PETER, NEWS LIMITED
Sara Western, 32, has given her 43-year-old husband Brenden the gift of life.
She donated one of her kidneys when his were fast deteriorating because of an incurable disorder.
The Sunshine Coast couple, parents to Kane, 3, and Layla, eight months, had operations almost a month ago and are recovering well.
They have always been an inspiring team, having won three Australian championships and a world title as part of the same Mooloolaba surfboat crew, Sara as a rower and Brenden as sweep and coach.
Sara said her decision to donate was simple.
“To be honest, it was a no-brainer for me. If you love someone that’s just what you do,’’ she said.
“The alternative was having a husband and father of two young children who had no quality of life while on dialysis, possibly for years, while he waited on the transplant list.’’
Brenden said he owed his life to his wife and could not be more grateful. “You just feel so appreciative. I would do anything for Sara … she is amazing,’’ he said.
“She was sitting with me at a specialist appointment when she surprised both of us by asking ‘what’s the procedure if I give a kidney?’ We almost fell off our chairs.’’
Brenden said the couple underwent tests to ensure their compatibility. The process took about 12 months and was completed just in time for him as his condition was worsening. [Read more]
PKD Research
From The New England Journal of Medicine
To the Editor:
In the Halt Progression of Polycystic Kidney Disease (HALT-PKD) study, Schrier et al. (Dec. 11 issue)1 randomly assigned patients with early-stage autosomal dominant polycystic kidney disease (ADPKD) to either a standard blood-pressure target or a low blood-pressure target. The low blood-pressure target was associated with a slower increase in total kidney volume, but not with an overall change in the estimated glomerular filtration rate (GFR). The latter finding might be viewed as being disappointing. However, we would caution that the institution of strict blood-pressure control will result in an acute, hemodynamic, but reversible decrease in the estimated GFR.
A recent scientific workshop sponsored by the U.S. National Kidney Foundation and the Food and Drug Administration concluded that this acute effect is not indicative of irreversible loss of nephrons.2 Accordingly, rather than using the baseline estimated GFR, we would suggest comparing only on-treatment slopes of the estimated GFR. Such an analysis does show a benefit associated with the low blood-pressure target (P=0.05).1 Since a decrease in the estimated GFR is a late phenomenon in many patients with ADPKD,3 a subgroup analysis of on-treatment estimated GFR slopes with the use of forest plots would be of interest, since it might hint at whether certain subgroups of patients with early-stage disease might benefit from a low blood-pressure target.
A. Lianne Messchendorp, M.D.
Ron T. Gansevoort, M.D., Ph.D.
University Medical Center Groningen, Groningen, the Netherlands
r.t.gansevoort@umcg.nl
No potential conflict of interest relevant to this letter was reported.
3 References
To the Editor:
Schrier and colleagues evaluated aggressive blood-pressure control with the use of dual blockade of the renin–angiotensin system in patients with ADPKD. The study also has relevance for physicians who initiated the use of such dual blockade in patients after it was reported that such treatment was more effective than single blockade of the renin–angiotensin–aldosterone system in reducing proteinuria.1
Subsequent studies such as the Ongoing Telmisartan Alone and in Combination with Ramipril Global Endpoint Trial (ONTARGET) and the Aliskiren Trial in Type 2 Diabetes Using Cardiorenal Endpoints (ALTITUDE) showed that dual blockade was not effective in reducing mortality or morbidity from cardiovascular disease but rather was associated with more severe adverse events such as hyperkalemia, hypotension, and acute kidney injury.2,3 A recent meta-analysis therefore concluded that dual therapy should not be used.4
However, those studies included patients with a high preexisting risk of vascular events and death because of diabetes, cardiovascular disease, heart failure, or old age. In contrast, the study by Schrier et al. clearly showed that in a relatively young population (15 to 49 years of age) without vascular disease, dual blockade was used relatively safely and that lowering blood pressure to values of 110/75 mm Hg did not result in adverse effects. Thus, physicians who have successfully used dual blockade to reduce proteinuria in their patients may consider the continued use of such therapy in these patients.
Jack F. Wetzels, M.D., Ph.D.
Radboud University Medical Center, Nijmegen, the Netherlands
jack.wetzels@radboudumc.nl
No potential conflict of interest relevant to this letter was reported.
4 References
Schrier and colleagues evaluated aggressive blood-pressure control with the use of dual blockade of the renin–angiotensin system in patients with ADPKD. The study also has relevance for physicians who initiated the use of such dual blockade in patients after it was reported that such treatment was more effective than single blockade of the renin–angiotensin–aldosterone system in reducing proteinuria.1
Subsequent studies such as the Ongoing Telmisartan Alone and in Combination with Ramipril Global Endpoint Trial (ONTARGET) and the Aliskiren Trial in Type 2 Diabetes Using Cardiorenal Endpoints (ALTITUDE) showed that dual blockade was not effective in reducing mortality or morbidity from cardiovascular disease but rather was associated with more severe adverse events such as hyperkalemia, hypotension, and acute kidney injury.2,3 A recent meta-analysis therefore concluded that dual therapy should not be used.4
However, those studies included patients with a high preexisting risk of vascular events and death because of diabetes, cardiovascular disease, heart failure, or old age. In contrast, the study by Schrier et al. clearly showed that in a relatively young population (15 to 49 years of age) without vascular disease, dual blockade was used relatively safely and that lowering blood pressure to values of 110/75 mm Hg did not result in adverse effects. Thus, physicians who have successfully used dual blockade to reduce proteinuria in their patients may consider the continued use of such therapy in these patients.
Jack F. Wetzels, M.D., Ph.D.
Radboud University Medical Center, Nijmegen, the Netherlands
jack.wetzels@radboudumc.nl
No potential conflict of interest relevant to this letter was reported.
4 References
To the Editor:
The HALT-PKD trial showed that in the low-blood-pressure group, as compared with the standard-blood-pressure group, the annual increase in total kidney volume was significantly less (5.6% vs. 6.6% increase per year). However, there was no benefit with regard to preservation of renal function. Blood-pressure goals were ambitious (95/60 to 110/75 mm Hg in the low-blood-pressure-group and 120/70 to 130/80 mm Hg in the standard-blood-pressure group)1 in these young patients with ADPKD (mean age, 36.6 to 48.7 years). Current treatment guidelines of the European and U.S. Eighth Joint National Committee on Prevention, Detection, Evaluation, and Treatment of High Blood Pressure do not support the use of such low blood-pressure targets in patients with nonproteinuric or even proteinuric chronic kidney disease, but instead they suggest blood-pressure targets lower than 140/90 mm Hg in all patients. The European Society of Cardiology guidelines support blood-pressure targets lower than 130 mm Hg in patients with chronic kidney disease and overt proteinuria.2,3 Blood-pressure goals that are as low as those defined by the HALT-PKD trial investigators may be dangerous in elderly patients with chronic kidney disease, presumably because of the high burden of prevalent cardiovascular disease in these patients. Similarly, in a very large cohort study involving 651,749 U.S. veterans with chronic kidney disease,4 the optimal blood-pressure range was reported to be 130 to 149 mm Hg systolic pressure and 70 to 89 mm Hg diastolic pressure, and mortality increased markedly with blood-pressure levels lower than 120/80 mm Hg.
Urs Benck, M.D.
Bernd Krüger, M.D.
Wilhelm H. Schmitt, M.D.
University Medical Center Mannheim, Mannheim, Germany
urs.benck@umm.de
No potential conflict of interest relevant to this letter was reported.
4 References
The author replies: Messchendorp and Gansevoort raise the issue of using only on-treatment estimated GFR slopes to evaluate the benefit of therapy. In our study, patients in the low-blood-pressure group, as compared with patients in the standard-blood-pressure group, had a slower increase in total kidney volume (P=0.006), a greater reduction in the left-ventricular-mass index (P<0.001), and reduced urinary albumin excretion (P<0.001). We also agree that the on-treatment slope of the estimated GFR shows a benefit for the low blood-pressure group (P=0.05).
In patients with chronic kidney disease such as ADPKD, the degree of proteinuria is a risk factor for cardiovascular complications and a decrease in kidney function. Thus, Wetzels's point is valid in that dual renin–angiotensin blockade may be indicated if it is shown to lower urinary protein excretion significantly more than monotherapy and is safe. Such may be the case in younger patients with ADPKD.
We agree with Benck et al. that aiming for blood pressure of less than 120/80 mm Hg may not be advisable in patients with chronic kidney disease, particularly in elderly patients. However, among patients with chronic kidney disease who have type 2 diabetes, those with blood pressure lower than 130/80 mm Hg have fewer complications and longer survival than those with blood pressure lower than 140/90 mm Hg.1
Robert W. Schrier, M.D.
University of Colorado School of Medicine, Aurora, CO
robert.schrier@ucdenver.edu
Since publication of his article, the author reports no further potential conflict of interest.
1 Reference
From CBC: Chicago Biomedical Consortium, Northwestern University Department of Pharmacology
Polycystic Kidney Disease (PKD) Ion Channels of Primary Cilia
DATE: March 2, 2015
TIME: 4:00 PM - 5:00 PM
SPEAKER: Paul G. DeCaen, Ph.D., HHMI/Harvard Medical School
LOCATION: Ward Building, Ward Conference Room 5-230, 303 E. Chicago Avenue, Chicago, IL 60611, Northwestern University (Chicago Campus)
Description
The Department of Pharmacology eagerly invites you to attend an upcoming seminar, to be presented by Paul G. DeCaen, Ph.D. Dr. DeCaen is presently a Postdoctoral Fellow from the Howard Hughes Medical Institute, Department of Cardiology, Boston Children's Hospital, as well as Department of Neurobiology, Harvard Medical School.
The following, is an overview of this seminar, as described by Dr. DeCaen:
“Polycystic kidney disease proteins (PKDs) are members of the Transient receptor potential (TRP) family of Ca2+-permeant ion channels that populate the membrane of primary cilia. A primary cilium is a solitary, non-motile protuberance from apical side of polarized cells. To advance our understanding of cilia PKD channels, we made cilia-specific fluorophores which enabled electrophysiological recordings of ion channels in the cilia membrane and which detect calcium changes in the ciliary compartment. These results (published last year) genetically identified a heteromeric PKD channel (PKD1-L1 and PKD2-L1) from the cilia of several non-renal tissues and established a high resting calcium [700 nM] concentration within this organelle. We are now poised to demine the why PKD2 mutations can cause Autosomal Dominant Polycystic Kidney Disease and their function in the collecting duct."
CONTACT: Kristina Lynn Ballard 312-503-4892
kristina.ballard@northwestern.edu
The HALT-PKD trial showed that in the low-blood-pressure group, as compared with the standard-blood-pressure group, the annual increase in total kidney volume was significantly less (5.6% vs. 6.6% increase per year). However, there was no benefit with regard to preservation of renal function. Blood-pressure goals were ambitious (95/60 to 110/75 mm Hg in the low-blood-pressure-group and 120/70 to 130/80 mm Hg in the standard-blood-pressure group)1 in these young patients with ADPKD (mean age, 36.6 to 48.7 years). Current treatment guidelines of the European and U.S. Eighth Joint National Committee on Prevention, Detection, Evaluation, and Treatment of High Blood Pressure do not support the use of such low blood-pressure targets in patients with nonproteinuric or even proteinuric chronic kidney disease, but instead they suggest blood-pressure targets lower than 140/90 mm Hg in all patients. The European Society of Cardiology guidelines support blood-pressure targets lower than 130 mm Hg in patients with chronic kidney disease and overt proteinuria.2,3 Blood-pressure goals that are as low as those defined by the HALT-PKD trial investigators may be dangerous in elderly patients with chronic kidney disease, presumably because of the high burden of prevalent cardiovascular disease in these patients. Similarly, in a very large cohort study involving 651,749 U.S. veterans with chronic kidney disease,4 the optimal blood-pressure range was reported to be 130 to 149 mm Hg systolic pressure and 70 to 89 mm Hg diastolic pressure, and mortality increased markedly with blood-pressure levels lower than 120/80 mm Hg.
Urs Benck, M.D.
Bernd Krüger, M.D.
Wilhelm H. Schmitt, M.D.
University Medical Center Mannheim, Mannheim, Germany
urs.benck@umm.de
No potential conflict of interest relevant to this letter was reported.
4 References
The author replies: Messchendorp and Gansevoort raise the issue of using only on-treatment estimated GFR slopes to evaluate the benefit of therapy. In our study, patients in the low-blood-pressure group, as compared with patients in the standard-blood-pressure group, had a slower increase in total kidney volume (P=0.006), a greater reduction in the left-ventricular-mass index (P<0.001), and reduced urinary albumin excretion (P<0.001). We also agree that the on-treatment slope of the estimated GFR shows a benefit for the low blood-pressure group (P=0.05).
In patients with chronic kidney disease such as ADPKD, the degree of proteinuria is a risk factor for cardiovascular complications and a decrease in kidney function. Thus, Wetzels's point is valid in that dual renin–angiotensin blockade may be indicated if it is shown to lower urinary protein excretion significantly more than monotherapy and is safe. Such may be the case in younger patients with ADPKD.
We agree with Benck et al. that aiming for blood pressure of less than 120/80 mm Hg may not be advisable in patients with chronic kidney disease, particularly in elderly patients. However, among patients with chronic kidney disease who have type 2 diabetes, those with blood pressure lower than 130/80 mm Hg have fewer complications and longer survival than those with blood pressure lower than 140/90 mm Hg.1
Robert W. Schrier, M.D.
University of Colorado School of Medicine, Aurora, CO
robert.schrier@ucdenver.edu
Since publication of his article, the author reports no further potential conflict of interest.
1 Reference
TIME: 4:00 PM - 5:00 PM
SPEAKER: Paul G. DeCaen, Ph.D., HHMI/Harvard Medical School
LOCATION: Ward Building, Ward Conference Room 5-230, 303 E. Chicago Avenue, Chicago, IL 60611, Northwestern University (Chicago Campus)
Description
The Department of Pharmacology eagerly invites you to attend an upcoming seminar, to be presented by Paul G. DeCaen, Ph.D. Dr. DeCaen is presently a Postdoctoral Fellow from the Howard Hughes Medical Institute, Department of Cardiology, Boston Children's Hospital, as well as Department of Neurobiology, Harvard Medical School.
The following, is an overview of this seminar, as described by Dr. DeCaen:
“Polycystic kidney disease proteins (PKDs) are members of the Transient receptor potential (TRP) family of Ca2+-permeant ion channels that populate the membrane of primary cilia. A primary cilium is a solitary, non-motile protuberance from apical side of polarized cells. To advance our understanding of cilia PKD channels, we made cilia-specific fluorophores which enabled electrophysiological recordings of ion channels in the cilia membrane and which detect calcium changes in the ciliary compartment. These results (published last year) genetically identified a heteromeric PKD channel (PKD1-L1 and PKD2-L1) from the cilia of several non-renal tissues and established a high resting calcium [700 nM] concentration within this organelle. We are now poised to demine the why PKD2 mutations can cause Autosomal Dominant Polycystic Kidney Disease and their function in the collecting duct."
CONTACT: Kristina Lynn Ballard 312-503-4892
kristina.ballard@northwestern.edu
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