Immune Deficiency Foundation Applauds Legislation to Improve Patient and Provider Access to Immunoglobulin Products

June 29, 2007

Immune Deficiency Foundation Applauds Legislation to Improve Patient and Provider Access to Immunoglobulin Products

Fri Jun 29, 2:24 PM ET


Contact: Christine Belser, of Immune Deficiency Foundation, Phone: +1- 410-321-6647, Cell: +1-443-824-2280,

WASHINGTON, June 29 /PRNewswire-USNewswire/ — The Immune Deficiency Foundation (IDF) applauds Congressman Kevin Brady (R, TX) who introduced H.R. 2914, known as the Medicare IVIG Access Act of 2007. With bipartisan support, the bill seeks adequate reimbursement for Intravenous Immunoglobulin (IVIG) products and accompanying care in the management of patients with primary immunodeficiency diseases (PIDD) and other serious conditions that require IVIG therapy.

“For most patients living with primary immunodeficiency diseases, IVIG is their only treatment option,” said Marcia Boyle, IDF President and Founder. “The delay or denial of treatment which is occurring under current Medicare reimbursement standards is putting patients at tremendous risk for serious, life threatening complications.”

Because of medical advances like IVIG, many individuals affected by primary immunodeficiency diseases can live normal lives. Without treatment, everyday illnesses like the common cold can put them at risk for infection and more serious complications. Negative health consequences reported by these patients include more infections generally, pneumonia, bronchitis, and increased use of antibiotics.

The current IVIG access and care issue began in January 2005 as a result of the Medicare Modernization Act under Part B, which changed the way physicians and hospital outpatient departments were paid under Medicare Part B. The law reduced IVIG reimbursement rates such that most physicians in outpatient settings could no longer afford to treat Medicare patients requiring IVIG, and either delaying regular treatment or sending many patients to a hospital clinical setting for therapy. Patient access was further complicated in 2006 when similar payment changes went into effect for hospital outpatient departments.

Specifically, the bill seeks to improve health outcomes and quality of life for PIDD patients by:
— Improving reimbursement for IVIG products as well as ancillary services — Allowing for home infusion provisions to cover administrative services — Maintaining pre-administration fees that allow providers to obtain IVIG products

A comprehensive patient survey conducted by IDF that supports today’s announcement found that Medicare patients, when compared to their private pay insurance counterparts, share a disproportionate burden of negative consequences as a result of the changes in Medicare IVIG reimbursement policies. Since January 2005, more than 40 percent of Medicare PIDD patients reported that their treatments had been postponed, with 26 percent reporting serious health consequences as a result of these delays. Additionally, 32 percent of the Medicare patients reported they have been forced to change their preferred IVIG treatment location. Many of these patients now must receive their IVIG in hospitals, which is not the ideal location for patients who are immunodeficient.

An IDF physician survey also found that 51 percent of physicians treating PIDD patients said they have had patients change their site of IVIG therapy because of Medicare reimbursement, and nearly half said they believe that current Medicare IVIG reimbursement policies pose a serious risk to the health of their patients.

“For patients without a full immune system, hospitals — with the possibility of spreading infections — can be dangerous places,” says Rep. Brady. “We want to make sure they can get treated at home, where it is safer. It is important Medicare provides them the access they need to this lifesaving therapy. That’s just common sense.”

Multiple patient, professional and manufacturer organizations contributed to today’s announcement, including IDF, the Clinical Immunology Society, Jeffrey Modell Foundation, GBS/CIDP Foundation International, the Plasma Protein Therapeutics Association, Platelet Disorder Support Association, ASD Healthcare and Accredo Health.

About Primary Immunodeficiency Diseases

Primary immunodeficiency diseases are disorders in which part of the body’s immune system is missing or does not function properly. In contrast to secondary immunodeficiency disease in which the immune system is compromised by factors outside the immune system, such as viruses or chemotherapy, the primary immunodeficiency diseases are caused by intrinsic or genetic defects in the immune system.

Approximately 250,000 people in the United States are diagnosed with primary immunodeficiency diseases. There are over 130 different primary immunodeficiency diseases, including X-linked Agammaglobulinemia (Bruton’s Disease), Common Variable Immune Deficiency, Selective IgA Deficiency, and Severe Combined Immune Deficiency (boy-in-the-bubble disease). Some disorders, such as Selective IgA Deficiency can be quite common, occurring as often as 1/500 to 1/1000 individuals. Others, such as Severe Combined Immune Deficiency, may be as rare as one individual affected per million. Untreated primary immunodeficiencies may be characterized by frequent life-threatening infections and debilitating illnesses.

Because of advances in our medical understanding and treatment of primary immunodeficiency diseases, individuals who in the past would not have survived childhood are now able to live normal lives. Many individuals affected by primary immunodeficiency diseases require life long therapies including intravenous gamma globulin infusions, antibiotic therapies, or bone marrow transplantation. The Immune Deficiency Foundation is dedicated to improving the diagnosis and treatment of primary immunodeficiency diseases through research, education and advocacy.

About The Immune Deficiency Foundation

The Immune Deficiency Foundation (IDF), founded in 1980, is the national non-profit patient organization dedicated to improving the diagnosis and treatment of patients with primary immunodeficiency diseases through research, education, and advocacy.

People with primary immunodeficiency diseases often find it difficult to receive specialized health care, and proper diagnosis and treatment. Individuals affected by primary immunodeficiency diseases also experience difficulties financing their health care, finding educational materials on the disease, and locating others with whom to share their experiences.

The goal of IDF is to help individuals overcome these difficulties and live healthy and productive lives. To learn more about IDF visit
Contact: Christine Belser Immune Deficiency Foundation Phone: 410-321-6647 Cell: 443-824-2280 E-mail:

SOURCE The Immune Deficiency Foundation

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BioRx Creates FULL CIRCLE™ Program in Support of The Immune Deficiency Foundation

June 27, 2007

Biorx Pledges Long-Term Financial Commitment to Patients with Primary Immunodeficiency Diseases and the Immune Deficiency Foundation

BioRx Creates FULL CIRCLE™ Program in Support of The Immune Deficiency Foundation

CINCINNATI — BioRx, one of the nation’s fastest growing providers of specialty pharmaceuticals, announces the creation of a new program to help patients with primary immunodeficiency diseases (PIDD). Their program, called Full Circle™, has been established to provide financial support to the Immune Deficiency Foundation (IDF), the national patient organization with the mission to improve the diagnosis and treatment of patients with primary immune deficiency diseases through research, education and advocacy.

As the first “Sponsor” of IDF’s new Home Healthcare and Specialty Pharmacy Sponsorship program, BioRx has committed to making a base level contribution to the IDF annually. Additionally, BioRx will contribute $1 for every gram of IVIG (intravenous immunoglobulin) or SCIG (subcutaneous immunoglobulin) that the company dispenses to any patient, who is being treated for a primary immunodeficiency.

“We believe in the work that the IDF is doing and feel it is our obligation and privilege to support the PIDD community through their organization,” says Eric Hill, Vice President and co-founder of BioRx. “We hope that this commitment will help enhance the early diagnosis of primary immunodeficiency diseases, improve access to state of the art medical care, and expand scientific and medical research on such diseases.”

According to Marcia Boyle, President and Founder of IDF, “As a patient focused organization, IDF applauds the commitment that BioRx is making to our mission on behalf of patients with primary immunodeficiency diseases. We are excited about this new initiative and thankful for their support and recognition of the importance of our work. As the first company to participate in our Home Healthcare and Specialty Pharmacy Sponsorship Program and their ongoing pledge to support IDF, BioRx has shown a corporate commitment to the PIDD community.”

More information about BioRx’s Full Circle™ program is available by calling 866.44.BIORX or via email at Information may also be requested through the company’s website at

About The Immune Deficiency Foundation
The Immune Deficiency Foundation (IDF), founded in 1980, is the national non-profit patient organization dedicated to improving the diagnosis and treatment of patients with primary immunodeficiency diseases through research, education, and advocacy.

There are over 130 different primary immunodeficiency diseases and they affect approximately 250,000 people in the United States. These individuals live throughout the country and often find it difficult to receive specialized health care, and proper diagnosis and treatment. Individuals affected by primary immunodeficiency diseases also experience difficulties financing their health care, finding educational materials on the disease, and locating others with whom to share their experiences. The goal of IDF is to help individuals overcome these difficulties and live a healthy and productive life. To learn more about IDF visit

About BioRx
Based in Cincinnati, BioRx is a national pharmacy specializing in highly customized care for the primary immunodeficiency diseases (PIDD) community. As one of the nation’s fastest growing providers of IgG and other specialty pharmaceuticals the company’s clinical staff reaches patients and physicians in 48 states. To learn more about BioRx visit

June 27, 2007

Toddler with no immune system dies of organ failure waiting for bone marrow transplant

Evan was always laughing

June 27, 2007
Joanna Frketich
The Hamilton Spectator

Evan Glen spent his short life laughing despite living in a virtual bubble.

The east-Hamilton toddler died suddenly yesterday morning, mere weeks from getting a bone-marrow transplant that could have cured the rare disease that left him with no immune system from birth.

He had what is known as boy in the bubble disease. Evan was a prisoner in his own home or in hospital for his 33 months. He would have been three in September.

“He was still happy,” said his mom Donna Glen. “He was always laughing.”

Just Monday, Evan laughed his way through one of his regular twice-weekly visits to Toronto’s Hospital for Sick Children, giving no signs his organs were about to fail.

He was dead in bed when his mother went to wake him up at 8:30 a.m. Paramedics and staff at McMaster Children’s Hospital tried in vain to revive him.

“He went to bed and he was fine,” said his mom. “None of us thought it would end this way. He never gave up.”

His mom and dad, Jim, had hoped to find a cure for Evan’s severe combined immunodeficiency when they made a public plea in April to find a bone marrow donor.

He’d already had one bone marrow transplant on March 30, 2006, but the match wasn’t good and he developed what’s known as graft-versus-host disease. His donor’s immune cells were literally attacking his body.

Spectator stories were picked up by other Canadian newspapers and prompted an outpouring of support, but a new donor was never found.

“I was hoping the article would bring a happy ending, but I’ve learned there are no happy endings,” said Donna.

Doctors were planning on going ahead this summer with a second transplant using the same donor.

Evan couldn’t walk in his last days because the graft-versus-host disease had so badly damaged his joints. But he spent that time doing what he loved most: being with his family, going for walks in the stroller and watching Cars, his favourite movie.

“Maybe it was too big of a battle,” said his mom. “We have a lot of unanswered questions, but sometimes you don’t know why.”

His parents and big sister Olivia, who is four, hope people will join the bone marrow registry in his memory.

“He was a good boy,” said Donna. “I don’t know what he was here for, but we do appreciate the time we had with him.

“But I’m greedy and I want more.”

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‘Bubble boy’ cuts his first birthday cake … and his last

June 26, 2007

‘Bubble boy’ cuts his first birthday cake … and his last
By Chong Shin Yen
For the first time since he was born, little Chong Khai Aik got to celebrate his birthday.

Sitting on a hospital bed, he blew out the nine candles and cut his birthday cake.

Three weeks later, on 23 Apr, he slipped into unconsciousness.

On 11 May, he died.

Khai Aik had been suffering from severe combined immunodeficiency (SCID) – a rare genetic disorder better known as the ‘Bubble Boy’ disease.

He was born without an immune system and his body could not fight infections and diseases.

Khai Aik’s mother, Madam See Bee Choon, 39, said that her son was admitted to KK Women’s and Children’s Hospital in March for a fever that did not subside.

Madam See, a clerk, said: ‘With his birthday approaching, he asked me one day, ‘Mummy, what is it like to celebrate a birthday?’.

No birthdays

‘His question brought tears to my eyes and it dawned on me that we had never celebrated his birthday before.’

She said that the family did not celebrate birthdays, but didn’t say why.

She and her husband have been estranged for more than a year. They have another son and a daughter, who are healthy.

Madam See said: ‘In my heart, I knew we would never get the chance to let Khai Aik celebrate it if we didn’t do it this time.’

The New Paper first reported on the brave little boy last November.

He relied on an oxygen machine to help him breathe, and he hardly left home for fear of infections.

He had never gone to school.

That was why his birthday party at the hospital on 31 Mar was so special.

Those at the party included his elder siblings and his nanny’s family.

His nanny, Madam Loke Wai Heng, 55, had been looking after him since he was born.

Missing from the party that day was Khai Aik’s mother, who said she was busy and had arrived at the hospital only after the celebrations.

Khai Aik could not contain his excitement and told her all about it when she stepped into his room.

Madam See recalled: ‘Although he was weak, he had this happy glow on his face when he described how they sang the birthday song for him.’

Her daughter later told Madam See that Khai Aik had cried while cutting his birthday cake.

It was tears of joy.

Madam See said that the chocolate birthday cake – decorated with characters from the children’s television show Barney – was chosen by Khai Aik from a catalogue his nanny’s daughter had taken from a cake shop.

Even during the celebrations, the little boy did not forget the hospital’s staff members. ‘He told me he wanted to give a big piece of cake for his doctor and some for the nurses,’ Madam See said.

Along with the cake, Khai Aik had received many birthday presents, which included Lego sets, an MP4 player, soft toys and clothes.

‘He was beaming with joy while opening his presents one by one,’ Madam See said.

Khai Aik was diagnosed with the immune system disorder when he was two, and had been using a machine to breathe since he was three.

Watched cartoons

The only places he had been to were his nanny’s flat or the hospital.

He spent most of his days watching cartoons at his nanny’s home. His elder siblings, a 13-year-old sister and 11-year-old brother, would go to the nanny’s flat after school.

After work, Madam See would take her children home. Her husband does not live with them.

Khai Aik’s nanny still cannot accept his death.

Madam Loke said that Khai Aik was so close to her that he also called her ‘Mummy’.

She added: ‘I knew that with his condition, he would leave us at a young age. But my heart ached whenever I saw that he couldn’t do what other kids do.’

She recalled the day Khai Aik mentioned that he had never gone out for a meal.

‘He told me one day, ‘Mummy, I’ve never been to a restaurant to eat. I wonder what it’s like’.’

That was close to Chinese New Year and the restaurants were packed.

Rare treat

Madam Loke said: ‘It was a big risk to take him out, but we wanted to fulfil his wish. So we took him – and a 5kg cylinder of oxygen – to a Chinese restaurant near Liang Court.’

About a month after his rare treat, Khai Aik was hospitalised.

A week before he slipped into unconsciousness, he rang up everyone close to him from his hospital bed, asking them to visit him.

Madam See said: ‘He called his nanny’s husband, daughter and son, who dote on him very much, and his aunts too.

‘He seemed to know that he would be going soon. He had fought bravely till the end, never complaining.’

Recalling some of her son’s last words, Madam See said: ‘I would sleep in the hospital with him every night and he would tell me, ‘Mummy don’t sleep, you must accompany me. You must sayang (love in Malay) me’.’

But overcome by tiredness, she would drift off after he fell asleep.

When she awoke on the morning of 11 May, Khai Aik had slipped away.

Copyright © 2005 Singapore Press Holdings Ltd.

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Stem-cell transplant from cord blood the only hope

June 26, 2007

Stem-cell transplant from cord blood the only hope

SUFFERERS of severe combined immunodeficiency (SCID) are prone to infections and diseases.

Their bone marrow stem cells are defective and cannot produce white blood cells to fight infections.

David Vetter, a SCID sufferer, lived his entire life inside a sealed plastic bubble. He died when he was 12.

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In 1976, his story was made into a movie, The Boy In The Plastic Bubble, which starred John Travolta.

The disease then came to be known as ‘Bubble Boy’ disease.

The disease is so rare that between 1990 and 2000, doctors here have diagnosed only two cases.

Both were infants and died within a year, a report in the Singapore Medical Journal (2003) stated.

SCID sufferers’ only hope would be a stem-cell transplant from cord blood.

Blood found in the umbilical cord is a rich source of immature cells that can develop into a wide range of different blood cells to replace diseased ones.

Last year, doctors used cord blood to save a baby who suffered from SCID.

Hoh Sin Jun was five months old when he was diagnosed with it, The Straits Times reported.

After taking four months to find a match, doctors from KK Women’s and Children’s Hospital (KKH) performed the transplant last May.

Doctors had considered Sin Jun’s case a significant milestone in dealing with the disease.

But they said that full immune-system recovery can take up to two years and Sin Jun had to be monitored closely.

When contacted, a KKH spokesman said that the boy, now two, is ‘doing well’.

He goes for regular blood tests and checkups.

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ViaCord and Children’s Hospital Oakland Research Institute Announce 100th Cord Blood Transplant

June 13, 2007

ViaCord and Children’s Hospital Oakland Research Institute Announce 100th Cord Blood Transplant

June 13, 2007 07:30 AM Eastern Daylight Time

Represents Most Related Cord Blood Transplant Experience

CAMBRIDGE, Mass. & OAKLAND, Calif.–(BUSINESS WIRE)–ViaCord®, the umbilical cord blood preservation division of ViaCell, Inc. (Nasdaq: VIAC), and Children’s Hospital Oakland Research Institute (CHORI) today announced that they have achieved their 100th umbilical cord blood transplant. The 100th transplant was a sibling transplant to treat Severe Combined Immunodeficiency (SCID), commonly known as “bubble boy” disease. This historic milestone includes transplants through cord blood collected, preserved and stored with ViaCord and transplants using cord blood stored through CHORI’s Sibling Donor Cord Blood Program……

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Cutaneous manifestations of disseminated BCG in a child with severe combined immunodeficiency

June 6, 2007

Oral 7, Cutaneous manifestations of disseminated BCG in a child with severe combined immunodeficiency
Authors: Brodie, T.; Goodyear, H.; Hackett, S.

Source: British Journal of Dermatology, Volume 156, Number 6, June 2007 , pp. 1405-1405(1)

Publisher: Blackwell Publishing


An 8-month-old girl of consanguineous parents presented with multiple subcutaneous nodules on her limbs and trunk. Over the preceding 2 weeks, her bacillus Calmette-Guérin (BCG) wound, which had been weeping since it was administered at birth, had become significantly worse. Both BCG and skin nodules had progressed to deep ulcers surrounded by areas of erythema with scaling. Previous contacts with health services included concerns regarding delayed development and a prolonged admission for a chest infection at 3 months of age (positive for respiratory syncytial virus with Campylobacter in her stool), associated with significant growth faltering requiring nasogastric feeding. This clinical picture raised the suspicion of severe combined immunodeficiency (SCID). Skin biopsy and Ziehl-Neelsen staining demonstrated numerous acid alcohol-fast bacilli within granulomatous lesions. Analysis of lymphocyte subsets, absent lymphocyte proliferations and molecular genetics confirmed the diagnosis of SCID with maternofetal engraftment complicated by disseminated BCG. The patient has recently received a matched unrelated donor human stem cell transplantation.Severe complications following BCG vaccination are rare and almost exclusively confined to patients with immunological impairment, in particular, SCID, chronic granulomatous disease, AIDS and complete DiGeorge syndrome.1 Individuals may present firstly to a dermatologist with subcutaneous nodules or a rash.2 The case described is used to highlight the importance of recognizing the cutaneous manifestations of disseminated BCG and to discuss the pathogenesis of the disease. References 1 Casanova JL, Jouanguy E, Lamhamedi S et al. Immunological conditions of children with BCG disseminated infection. Lancet 1995; 346:581. 2 Al-Bhal LA. Pathological findings for bacilli Calmette-Guérin infections in immunocompetent and immunocompromised patients. Am J Clin Pathol 2000; 113:703-8.

Document Type: Abstract

DOI: 10.1111/j.1365-2133.2007.07876_7.x

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