UNEXPLAINED breathlessness is not OK, say a group of doctors who have banded together to tell people about a deadly lung disease.
The disease is pulmonary hypertension, which affects thousands of people and can kill quicker than untreated lung cancer if not caught in time.
Part of the problem is that doctors have been taught it is "very rare" and often mistake it for asthma or other illnesses, says Dr Geoff Strange, CEO of the Pulmonary Hypertension Society.
But the disease is far more common than thought, affecting more than 85,000 people in Australia and New Zealand.
One form of the disease, pulmonary arterial hypertension, is of particular concern.
Although it can affect anyone at any age, most patients are girls and young women and most die or need a lung transplant within four years if not treated.
However, if diagnosed and treated in time, many respond well to a simple oral medicine.
"A young person who is diagnosed and treated early has a reasonable chance of living 20 years," says the organisation's president, Professor Trevor Williams.
Time lost because of delayed treatment cannot be regained. Even 12 weeks is significant, he says.
Patients must go back to their doctor after a month if the treatment they have received for breathlessness is not working, he says.
"Go back until you find an answer," he said.
"Something is not ok if you can't keep up with others walking on the flat or up a gentle slope due to breathlessness."
One of the problems with the disease is that it affects the lungs and the heart, and has fallen between the cracks between different medical specialities, says Prof Williams, who is head of respiratory medicine at the Alfred Hospital in Melbourne.
"Doctors are struggling with this," says Dr Strange.
"A huge change is needed across the medical community and the general community so people understand the importance of breathlessness."
He says the average person with the disease has five GP visits before being referred to a specialist. They then see three different specialists before they are referred to a centre that understands the condition.
"It is taking people too long to have critical tests," says founding president Professor Anne Keogh, senior heart transplant cardiologist at St Vincent's Hospital in Sydney.
"Breathlessness can be just as life threatening as chest pain. Doctors must take it seriously."
She says people who are getting increasingly breathless should seek help.
"Don't put it down to being overweight or unfit."
Stem Cells for Lung Disease - Hope or Hype?
A/Prof Daniel Chambers, Queensland Lung Transplant Service
The Prince Charles Hospital, Brisbane
Stem cell therapy is often seen as the next miraculous breakthrough treatment delivered by medical science. But where does the truth lie? Cells - the building blocks of life: Large organisms like us consist of billions of cells of many different types or 'lineages'. These 'lineage committed' cells are the ones which allow our organs to function normally, but the trade off to becoming a specialised, lineage committed cell is that the cell's ability to readily reproduce is lost. This is where stem cells come in - they can both produce more stem cells (a process called 'self renewal') and a large number of lineage committed cells. While self-renewal is a must for a stem-cell, the scope for lineage differentiation is what defines the stem cell embryonic stem cells can become any type of lineage committed cell, while so-called 'adult' stem cells are more restricted.
Given this background one can immediately sense that stem cells are going to be important not only during development, but also in the ageing process. This is true - it is likely that many of the features of normal aging (e.g. grey hair) are a result of exhaustion of adult stem cell pools, as are many of the 'diseases of aging'. It is also why the field of stem cell therapy is often referred to as a regenerative medicine. You may ask "Why do we age?' and "Is aging necessary?' both fascinating questions, but beyond this article's reach, I urge you to read Thomas Kirkwood's work.
Hype - So what about stem cells and the lung? The very real ethical issues associated with using embryonic stem cells for research and eventually to treat disease have abated now that similar cells can be manufactured from adult cells (so called 'induced pluripotent stem cells' or iPS cells). However, both iPS and embryonic stem cells are a long way from being able to be used to treat human disease because they have an inherent risk of becoming cancerous. It is much more likely that the real breakthroughs are going to come from discovering how the 'adult' stem cells, which we all have, ensure continuous organ regeneration throughout life, and how we can manipulate them to maintain health.
Hope - Adult stem cells are the source of the new cells which keep our organs functioning throughout a lifetime filled with potential threats. For example, a nasty bout of influenza or exposure to toxins will kill some or many of the epithelial cells which line the lung, but local stem cells will repair the damage. But what if these stem cells get exhausted after too many such episodes? Recent evidence suggests that depletion of these cells or loss of their ability to self renew after a lifetime of injury causes chronic lung diseases like idiopathic pulmonary fibrosis (IPF). IPF is a fatal disease whose incidence is increasing by 5% per year as our population ages. IPF now kills over 1000 Australians annually; more than double the number who die from asthma.
But how will studying adult stem cells help people with IPF and other chronic lung diseases? Groundbreaking work done in mice by Dr Jonathan Macqualter and Prof Ivan Bertoncello at the University of Melbourne has identified the master cells which control lung epithelial repair. Together with them we are now working out which cells do the same job in humans. Incredibly, it may be that another adult stem cell found in the lung called a mesenchymal stem cell (MSC) is able to literally 'recharge' the flat batteries in the epithelial stem cell pool by donating some of its own 'batteries' its mitochondria. These observations have challenged much of what we thought we knew about lung repair and have led my team to conduct a first-in-man trial of MSC therapy in IPF to determine whether this kind of treatment is feasible.
Lung, heal thyself - So we don't need to completely reinvent the wheel. Mother Nature already has amazing mechanisms in place to ensure that organs can heal themselves. All we need to do is develop a thorough understanding of this healing process and coax it along in the right direction. In future, developing a thorough understanding of how the body heals itself will lead to the development of new treatments, some of them probably cellular ('stem cell therapies'), some drug based. So, sure there's a lot of hype, but there truly is hope as well.
In late September 2012, the Robyn Barst Paediatric Pulmonary Hypertension Research and Mentoring Fund reached its stage-1 fundraising goal of $1,000,000 to begin awarding grants. This is the world's first research and mentoring fund established to support paediatric research in PH.
Named for Dr. Robyn Barst, a pioneer in the field of PH who provided the gifts to launch this research fund, the Robyn Barst Paediatric PH Research and Mentoring Fund will provide grant opportunities that include both clinical research and patient care components.
The fund is intended to provide young physicians with mentoring to develop clinical research skills as well as appropriate role models for patient care. Young physicians will be mentored by established paediatric PH physicians in PH centres.
Participating physicians will be trained on how to take care of children, and they will also be trained in clinical drug development intended to improve survival as well as quality of life.
"It takes a lot of exposure," says Dr. Barst, since treatments and available drugs can change and vary rapidly over time. Clinicians and researchers must spend considerable time studying and treating the disease firsthand to reach conclusive findings regarding diagnosis and management.
Dr. Barst has seen, and often been the catalyst of, advances in the field of PH. PH treatment options have increased since the beginning of her career when the sole option was heart-lung transplantation. Now patients can be managed on one of nine FDA-approved therapies.
Despite this, however, the management of paediatric PH remain limited with no FDA-approved treatments for children.
"Children are not small adults," Dr. Barst explains, and should not be treated in the same way as adults. "Their response to drugs can be somewhat different. Dosing is different. Side effects are different." The common process of prescribing lower doses of adult medications may not necessarily be the best treatment method for children.
When establishing the fund's goals, Dr. Barst insisted that she did not intend to raise money that would be completely dispersed and depleted within a year. She wanted to create a long lasting and hopefully life changing endowment-like fund to improve overall quality of life and improve outcomes.
"I made it clear that this was to be an endowment-like fund. We want to be able to train physicians and, at the same time, do research and maintain a body of funds so we can do this for many years to come," states Dr. Barst. She hopes that such extensive first-hand involvement will help expand the number of PH paediatric experts.
When the Robyn Barst Paediatric Pulmonary Hypertension Research and Mentoring Fund recently reached $1,000,000, it was activated. Its next stage will be to move to $3,000,000 when it will be able to offer more robust grants. The fund will maintain a $1,000,000 minimum balance. It is also receiving global recognition and recently received generous gifts from PHA's sister organisations, PHA Europe and PHA Korea.
PHA is proud to launch this fund to better understand paediatric PH. Awarding will begin in 2013. More information about Dr. Barst and the Robyn Barst Paediatric Pulmonary Hypertension Research and Mentoring Fund can be found at www.PHAssociation.org/BarstFund.
View a video about Dr. Barst's commitment to advancing care for paediatric PH patients, the development of the first PH treatment and the advancement of the understanding of the disease at www.PHAOnlineUniv.org/PHTrailBlazer-RobynBarstMD.
Read our "Meet the Doctor" interview with Dr. Barst in the next edition of "Phan Mail".
Article courtesy of Pathlight - PHA USA
Denise Bradley’s heart was stopped and every drop of blood was drained from her body to ensure she lived.
During the radical surgery at Royal Perth Hospital her body was cooled to 15°C.
While on a heart and lung machine, her blood circulation was stopped for about 20 minutes as surgeons worked for more than eight hours to save her life.
Hers was one of only a few of the highly risky operations known as pulmonary thrombo endarterectomies performed at RPH each year.
Last Sunday two of the men who made Mrs Bradley’s operation possible were there to watch the
45-year-old play her first game of cricket since the surgery.
The remarkable comeback was capped off when the medium pace bowler took two wickets and scored a few runs with the bat.
In October last year Mrs Bradley, of Wembley, was told that blood clots had begun to form on her pulmonary arteries.
The clots were causing a hardening of thew arteries, reducing blood flow from the heart to the lungs.
Doctors told her she had just two years to live.
The first sign that something was wrong came when the normally highly active mother of three began feeling short of breath.
"I just felt ill all the time," she said "At one stage I had friends coming over to hang up my washing because I was too sick to even do that.
"I struggled to even walk up the road only about 200m to pick the kids up from school. "I had good days and bad days and the bad days I spent all day in bed."
She was told that she needed to undergo the rare surgery as soon as possible.
It has been described as surgery where the patient is literally "brought back from the dead".
It can cause minor brain damage. But it was a fate better than death, according to Mrs Bradley.
"I wasnt scared at all, I was just really excited about getting better," she said. "But I don’t think I understood the enormity of it all at the time."
"When I woke up from the operation I felt like I’d been hit by a truck."
Four months after the operation, Mrs Bradley is now making a good recovery.
"My voice has changed. It’s gone very monotone and I have no life left in it,"she said, "I’ve got little bits and pieces here and there that aren’t quite right but I’m very happy to be alive."
Despite this, Mrs Bradley’s active lifestyle is back on track. She is playing basketball and cricket again.
Her cardio-thoracic surgeon, Mark Edwards, said it was amazing that Mrs Bradley was alive, let alone playing sport again.
"To see her out on the cricket field after this operation is really an absolutely amazing story. As far as I know that has never been done before," he said, "I’m not aware of anybody else going back and playing cricket after this operation."
"I suppose a good way to describe the surgery is the patient is almost in a state of suspended animation and this allows us to open up the ateries to the lungs and clean out the hardening of the material."
Mrs Bradley said it was wonderful that a WA hospital offered the rare surgery.
"I’m happy that I got the right man (RPH respiratiory physician Eli Gabbay), who got the right surgeon and got me into the right hospital to get me fixed." she said.
Cricket has always been Mrs Bradley’s passion. She has played since she was 13.
"I love it, It’s great to be back," she said.
LIFE ON HOLD AS MUM WAITS FOR LUNG DONOR
Ballajura mother-of-two Linda Franklin’s bags are packed but she can’t go on holidays with her family, not even to Rottnest.
Mrs Franklin, 39, has pulmonary hypertension and needs a double lung transplant to save her life.
She has been on the organ donation list for two years and once got the call to attend hospital but the lungs were not compatible. Now she needs oxygen 24 hours a day.
Mrs Franklin said her illness has affected the whole family, especially children Toby 3, and Riley, 7. "We have to live within an hour’s radius of the hospital, we can’t go on a holiday … we have to live on standby," she said.
While Mrs Franklin is lucky to have made the transplant list, she still may not get the lungs she needs.
According to Transplant Australia, 1700 people are on the list but 80 will die waiting for organs each year.
State Cabinet is to consider a proposal for an opt-out organ donation system, under which West Australians will be assumed to be organ donors unless they register otherwise.
Transplant Australia CEO Chris Thomas said an opt-out system would lift WA’s lagging donation rate and save thousands of lives.
This week is Donate Life week, with Australians encouraged to think seriously about organ donation and talk about their wishes with their families.
Congratulations to Linda Franklin who has temprarily been downgraded on the lung transplant list. Linda has been working very hard and it is paying off. Well done Linda!
Article from the West Australian newspaper which was published 21st February 2011.
Pulmonary Hypertension patient Linda Franklin is a real PH star. This is one of several media stories that have been written about Linda.