BMT Centre To Come Up In City

November 23, 2010

Courtesy: fullhyderabad.com

Patients suffering from thallassemia will soon be able to obtain the services of a Centre for Bone Marrow Transplant in the city itself.

A team of doctors from Italy are currently in the city, to find the best possible hospital to set up the BMT unit.

Doctor Pietro Sodani told media persons that he and his team will, under the aegis of ‘Cure Thallassemina India’, a non-profit, non-political and secular organisation providing support to health professionals in developing countries, will finalise the hospital after going through the various corporate hospitals in the city.

Dr. Pietro revealed that very few centres for BMT exist in India, and that the city will soon join other cities like Vellore, Bangalore and Delhi after one is set up here.

He also revealed that the BMT Centre will provide relief to about 1,400 known thallassemia patients in the State.

Thalassemia Major is a fatal blood disorder which not only destroys red blood cells but also produces defective red blood cells. It requires life-long blood transfusion, every 3 to 4 weeks, and medicines, failing which 90% of children afflicted by it would die in childhood. BMT is the only known cure.

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New Therapies Break Sickle Cell’s Painful Grip

April 1, 2009

Courtesy by: miller-mccune.com

A brace of therapies ranging from improved bone marrow transplantation to stem cell research may finally put sickle cell anemia on the run.

Sickle cell anemia, the first genetic disorder recognized by medicine, is still not well understood.

Identified in 1910 by James B. Harrick in Chicago, the disease affects close to 100,000 people in the United States, mostly African American, and millions worldwide. The consequences for patients are devastating.

According to Sophie Lanzkron, director of the Sickle Cell Center for Adults at Johns Hopkins University, 30 percent of sickle cell patients experience pain 90 percent of days. “This is a tough disease; they have intermittent episodes of excruciating pain — they can’t go to college or have careers. Patients spend their lives just trying to manage the pain.”

With no objective measure of the disease, Lanzkron said patients are frequently stigmatized when they seek medical attention — being mistaken for addicts in search of narcotics — and treated with disdain by emergency-room personnel unfamiliar with the disorder.

The physiological consequences of sickle cell anemia include severe damage to organ systems, particularly to the kidneys and lungs, and can reduce life expectancy for those with sickle cell to the mid-40s. Complications include life-threatening infections and stroke — even in young children.

Children who suffer intermittent attacks miss school with predictable consequences for their lives and for their futures. Meanwhile, their caregivers must miss work to tend to them. Added to this are the health-care costs. Sickle cell patients, Lanzkron said, are often “understandably depressed.”

“There are so many unmet needs in this patient population,” says Lanzkron, “and I see people come in with horrible complications.”

But researchers are targeting the disease with new multidisciplinary approaches with promising results. Combining a novel chemotherapy protocol with a proven bone marrow transplant technique, Robert Brodsky, director of the hematology division at Johns Hopkins University Medicine, announced the cure [see correction below] of an adult sickle cell anemia patient — Pamela Newton of Capitol Heights, Md. And researchers with the National Institutes of Health have developed innovative techniques spanning the spectrum from chemotherapy to irradiation to move step-by-step towards a cure for sickle cell and related disorders.

More than meets the eye

Normal red blood cells are disk-shaped and flexible, explained Lanzkron, allowing them to squeeze through tiny capillaries to bring oxygen and nutrients into every part of the body.

In those with sickle cell, their red blood cells contain unusual polymers that intermittently cause the cells to lose their elasticity and lock into a sickle shape, massing and tangling to block blood vessels and capillaries. That’s where the pain starts. “But we know it’s more complicated than that,” she said. The sickle cells also have a reduced oxygen capacity and shortened life expectancy compared to normal blood cells, and they seem to have a negative impact on the blood vessels themselves, “It affects the whole environment.”

“Scientists believe (the) sickle cell trait developed in malarial regions over time through natural selection,” said Lanetta B. Jordan, chief medical officer with the Sickle Cell Disease Association of America. It persists, she says, “because of the survival benefit it confers against malaria.”

Lanzkron elaborated: “People carrying the trait can still get malaria, but they are less likely to die from it.” But, she said, “There is no harm in having the trait,” which is carried by a recessive gene.

However, when two parents have the trait, their offspring each have a one-in-four chance of having sickle-cell disease.  Although mostly associated with blacks in the United States, the disorder is also seen among Latinos, Asians and others with Mediterranean or African ancestry.

Aside from the not-always-effective pain medications, only two approved treatments exist for sickle cell, Jordan said — the chemotherapy drug hydroxyurea to ameliorate crises and bone marrow transplant.

An Elusive and Risky Cure

Bone marrow transplants have been recognized as a cure for sickle cell for more than 20 years, but they have always required a perfect tissue match between the donor and the recipient. Only siblings can meet that requirement, and even then, the odds are one in four of a perfect match. However, because sickle cell is hereditary, closely matched siblings also have a high probability of sharing the disorder.

Lanzkron describes a lifesaving operation: “We give the patient agents that knock out the bone marrow; then it’s like getting a transfusion. We harvest a bit of marrow from the donor and infuse it into the patient.

“The bone marrow cells do the rest. They know where to go.” It sounds deceptively simple, but Lanzkron says that a bone marrow transplant has between a 10 percent to 20 percent mortality rate. Although the procedure has been used to cure the disease in about 200 patients — all of them children — bone marrow transplant is so problematic in adults that it is rarely attempted except in life-threatening circumstances.

“It is very difficult for adults to embark on a transplant course because, by that time, they have experienced so many transfusions that finding a match in itself is a challenge,” Jordan explained. Furthermore, in adulthood, mounting complications and decades of organ damage may render patients too fragile to withstand the chemotherapy required to make it work.

Over the past year, important advances have been made both in widening the pool of potential marrow donors and in lowering the risks of bone marrow transplant.

The intensive chemotherapy regimen, employed up until the 1990s to make way for bone marrow transplants, destroys the patient’s native bone marrow so that a graft can take root. But if the graft fails, the patient, with his own marrow obliterated, can no longer produce new blood cells and must be re-transplanted immediately in order to survive.

But this scenario is turning around dramatically thanks to Brodsky’s innovative technique developed at Johns Hopkins. Brodsky and his team, who reported the first adult cure in May of last year, took a new approach by utilizing a less toxic chemotherapy regime than customarily used for bone marrow transplants.

It employs just enough chemotherapy to prevent the patient’s immune system from rejecting the donated stem cells while preserving as much of the patient’s bone marrow as possible.

Three days after the bone marrow transplant, doctors dose the patient with the drug cyclophosphamide to kill off the donor’s lymphocytes (white blood cells that attack foreign cells) before they can begin an attack upon the body of their new host. This combination therapy gives the recipient patient’s new stem cells a chance to establish themselves in the bone marrow and begin producing healthy blood cells while a compatible — and home-grown — immune system develops.

Most significantly, Brodsky said the procedure allows for transplants not only between fully matched siblings, but also between half-matched pairs, expanding the potential donor pool to parents and other relatives.

Immunosupressive Advances for Sickle Cell and Related Disorders
Jonathan Powell, a National Institutes of Health research fellow, has been collaborating with Dr. John Tisdale at NIH on a different approach, which shows promise not only for sickle cell, but also for thalassemia, Diamond-Blackfan anemia and other severe congenital blood disorders.

Following Tisdale and Powell’s protocol, the donor receives specialized chemotherapy for five days with injections of G-CSF, an agent that stimulates white blood cell formation, to prepare the marrow for transplantation. The cells, once harvested, are frozen for five months before being infused into the patient. Patients undergo their own course of immune suppressing chemotherapy, and are given a single dose of full-body radiation to retard the immune system’s response to the donated stem cells.

According to Powell, 10 of the 11 people with sickle cell anemia who have undergone this procedure have taken well to the grafts. And in both studies, failure is not necessarily of dire consequence.

Javier Bolaños-Meade, assistant professor of oncology at Johns Hopkins, says with the reduced chemotherapy approach used by both NIH and Hopkins, “the worst case scenario is that the transplant doesn’t take — then we’re back to square one and the person ends up still having sickle cell,” but their bone marrow recovers and they can survive to try an alternative therapy.

Powell said NIH’s results have been promising “not in numbers, but in the positive response the patients have had. It shows that we’re on the right track.

“There are no major technical hurdles left. Bone marrow transplant is a way to cure sickle cell — unequivocally.”

In January 2009, NIH’s bone marrow transplant study was expanded to include not only fully matched adults, but also children and half-matched donor/recipient pairs, and recruitment for these new participants is currently under way; likewise for an ongoing study at Hopkins.

Powell says he is exploring collaboration between the NIH study and Brodsky’s research at Hopkins that would draw upon the best of both worlds. “We may end up combining ideas.”

Bolaños-Meade, who led the Hopkins research team, is also confident and says sickle cell shows some of the best results for transplant therapy for any blood disorder. “Given that this is a common condition, if there’s a way to correct the problem, we believe we can have a profound impact.”

*Correction: In an earlier version, we said that Johns Hopkins researcher Robert Brodsky and his colleagues developed “the first-ever cure of an adult sickle cell anemia patient.” Dr. Brodsky says that Pamela Newton was cured, but her case is not the first ever adult cure for sickle cell — other adult patients have successfully received bone marrow transplants and been cured. The approach being developed at Johns Hopkins potentially allows for broader application of bone marrow transplants to cure sickle cell. Brodsky notes that this new technique, used for the first time on Ms. Newton, was probably the first successful haploidentical bone marrow transplant for sickle cell — he’s unaware of others, but he’s not 100 percent certain that there aren’t more out there.


Bone Marrow Transplant Unit opens at PIMS

March 19, 2009

Courtesy by: thenews.com.pk

A two-bed Bone Marrow Transplant (BMT) Unit was inaugurated Tuesday at the Pakistan Institute of Medical Sciences (PIMS), making it the country’s first public sector hospital offering such a facility.

The unit, which has been made possible by virtue of a public-private partnership between PIMS and the Care 2 Children (C2C) Foundation of Italy, has been established in memory of an Italian child Simone Montomoli, who died of cancer, and whose parents contributed 15,000 euros for the civil works of the project in Pakistan.

Minister for Health Mir Aijaz Khan Jhakrani inaugurated the centre in the presence of Italian Ambassador Vincenzo Prati, executive director PIMS Dr Abdul Majeed Rajput, managing director Pakistan Baitual Maal Zamarrud Khan, and Italian paediatric onco-hematologist Dr Lawrence Faulkner, who undertook several visits to Pakistan for materialisation of the project.

The unit will initially function with the assistance of C2C Foundation, which has not only committed to finance the cost of the first six transplants but also of the salaries of the nine staff members appointed for the unit.

“The cost of one transplant in Pakistan is approximately Rs1.2 million. As such, C2C Foundation will contribute a total of Rs7.5 million in treatment costs. It will also bear the cost of staff salaries for a period of two years or till such time that the Rs500 million PC-1 of the project is approved by the government,” Dr Rajput told the audience, simultaneously urging Jhakrani for expeditious approval of the PC-1 so that the facility can be expanded to a 16-bed unit.

Addressing the ceremony, Jhakrani thanked the government of Italy, the C2C Foundation, and the parents of the deceased child for supporting the project, and assured that the PC-1 would be accorded priority in processing and approval as the current facility is grossly insufficient to cater to the needs of all patients requiring bone marrow transplantation.

The unit will carry out bone marrow transplants for thalassemia, aplastic anaemia and childhood leukemias. Thalassemia major is the most common life-threatening genetic disease. Even though long-term supportive care with regular red cell transfusion and intensive chelation therapy may prolong life expectancy to the fourth decade of life, bone marrow transplantation remains the only curative option. Pakistan has around 50,000 registered children suffering from Thalassemia, and about 5,000 new cases are adding up every year.

The six initial transplants will be performed under the supervision of Italian bone marrow transplant doctors nurses, who will also train local professionals for the sake of long-term sustainability of the facility.

Dr Rajput informed that PIMS will be providing infrastructural facilities and support services including diagnostic facilities, equipment and free drugs to patients, whereas the C2C Foundation will provide bone marrow transplant specific tests, which will initially be outsourced and eventually help PIMS to establish these facilities. He said, PIMS will soon be in a position to offer training to other aspiring hospitals and professionals; a commitment to this effect has already been made with the Lahore Children’s Hospital.

The managing director of Baitul Maal paid rich tributes to Dr Sadaf and Dr Khalid for their dedication to the project. He suggested that a certain chunk of the funds available to senators and MNAs should be channelled to the health sector. Zamurrad Khan announced a donation of Rs300,000 in support of treatment of BMT patients. He also shared details of the five new projects being initiated by Baitul Mal. These envisage provision of wheelchairs, hearing aids, and white canes, as well as the establishment of five hepatitis treatment centres in Quetta, Karachi, Lahore, Islamabad and Peshawar.

Zamurrad Khan said, Baitul Mal would provide Rs25,000 to each family with a disabled person. He said, 1,500 wheelchairs have been provided to disabled persons ever since August 15. “Please refer to us, any person requiring a wheelchair, and we will do the needful,” he assured.

Earlier, Italian ambassador Vincenzo Prati expressed his country’s desire to expand cooperation with Pakistan in the domain of health. He invited Jhakrani to visit Italy for exploration of joint avenues. He suggested that a seminar on health cooperation should be planned in Lahore in November.

Dr Lawrence Faulkner shared how impressed he was with the professionalism of the Pakistani team that worked on the initiative. “More help will, however, be needed to build on this initial success and make it sustainable,” he commented, hoping that the unit would provide a scenario for training of health professionals while also motivating Pakistani doctors abroad to return and serve in Pakistan.


And Tomorrow it Could Be Your Child…or Mine!

March 16, 2009

Courtesy by: indiajournal.com

Who would not wish to ex tend a lease on the life of one’s son, daughter or a grandchild? And who would want to lose such a precious gift after having been blessed with one? These painful thoughts often make my nights sleepless when I come to know about a child of the Indian subcontinent pedigree losing the game of life simply because no one came forward to offer a small amount of bone marrow that he or she direly needed to survive. Many such children suffering from acute leukemia or other fatal blood illnesses wither away without seeing the light of future days in the absence of available matching donors. This kind of apathy among our people causes ripples in my heart and turns my stomach upside down. I ask myself how it is that we, the people from the Indian subcontinent, who otherwise are willing to donate any amount of money to build and sustain more temples, gurdwaras, mosques and churches, fail miserably when it comes to donating parts of bodies, parts that will not impair our ability to function perfectly well without. The answer is painfully obvious.

We donate to the places of worship, in part, because of a genuine need, but more often than not, we do so to satisfy our egos and out of a belief that by building more religious places, we will be assured a seat in heaven. For some obscure reasons, a gift that can save the life of a helpless child without causing much discomfort to our bodies and pockets is usually not on our agenda. Rarely do we consider that helping a dying child with such a gift of life might assure us of a rather better seat in heaven, perhaps even in the first row, if there were such a thing? Moreover, the spoken and unspoken words of gratitude of the life thus saved will often go a long way in showering lifelong blessings on the donor.

Most people in the West whom we casually write off as materialistic, stand ready to silently help in finding solutions to ease the pain and suffering of their fellow human beings. They support research by all the available means at their disposal to find new treatments to control illnesses that if left alone could cause havoc. Many rich individuals in Western countries establish or support privately sponsored charitable foundations. Organizations like the American Cancer Society, the American Heart Association, the Parkinson Disease Foundation, the National Multiple Sclerosis Foundation, the Alzheimer’s foundation, the American Epilepsy Society and many more are doing yeoman’s work. To some extent, we, the people of India and other Asian countries try to follow suit, but our pace is no faster than that of a snail. In part, this is due to our ignorance about various disease processes.

For this very reason, I wish to say a few words about bone marrow transplants and how they work. I have a vision that eligible people of the Indian subcontinent ethnicity after learning a bit more about bone marrow donation may gain some insight and understanding about the necessity of such a noble act and offer this priceless gift to those in desperate need of it.

Bone marrow is a spongy red substance present in the center of our bones including the pelvis (hips), the vertebrae (spines), the sternum (breast plate), the ribs, and the skull. This spongy red mesh contains stem cells, also known as grandfather or grandmother cells. They give rise to generations of other mature bone marrow cells which in turn create all the other present in the bloodstream, cells that deliver oxygen and other vital nutrients to different parts of the body. Normal bone marrow is like a factory, churning out as many cells as our body needs. In leukemia and other related disorders, this factory goes haywire. It produces cells that may be aggressive in competition than healthy cells, but are unable to perform their assigned functions in spite of their larger numbers. It is something like having a huge army that is totally unable to fight.

A bone marrow transplant is often the last and only treatment for curing illnesses of the blood such as leukemia (blood cancer), lymphoma and some inherited disorders such as thalassemia major.

Transplants can be autologous or allogeneic. With autologous transplants, one receives back one’s own stem cells. The stem cells first are removed and parked outside of the patient’s body prior to subjecting that patient to a total-body radiation and to chemotherapy, both of which destroy the patient’s bone marrow. Once the marrow is destroyed the parked stem-cells are re-infused back. However, this kind of transplant is not ideal for leukemia.

With allogeneic transplants, the patient receives stem cells from the bone marrow of another person. The ideal donor is an identical twin. If this is not possible, a brother or a sister from the same parents is preferred. Unfortunately, the chance of a match from a non-identical sibling is around 25%. It implies that many patients are unable to get any match at all from their siblings. Therefore, having a donor pool becomes very important in any ethnic community, as common ethnicity increases the chances of finding a good marrow match among people who otherwise are unrelated to the patient.

The use of the word ‘transplant’ here might be a misnomer as in that a bone marrow transplant is not a major procedure like a kidney, heart, or liver transplant. The potential donor donates a few drops of blood to determine if his or her tissue type will match with that of the patient in question. This testing is neither terribly expensive nor difficult. Only when the tissue of a potential donor matches that of a recipient is the donor contacted.

The main procedure of a marrow transplant or transfer requires a donor to have a few small punctures on the back of the pelvis. From these punctures, bone marrow from inside the pelvic bones (hips) is sucked out with a syringe. Such a procedure will need either general or regional (wide-area rather than local) anesthesia. As with any other surgery, some amount of post-operative soreness is often felt. This procedure only takes about one to one-and-a-half hours. Within a few weeks, the body replaces the marrow that was removed and one feels as if nothing has been taken away.

Stem cells can also be obtained from the peripheral blood. In this kind of donation process, one of the veins in the arm is used to remove the stem cells. This procedure requires the prior use of a particular medicine by the donor for four to five days in order to increase the yield of the stem cells when they are harvested. Minor temporary side effects such as bone discomfort or bone pain, muscle pain, fatigue and nausea can result from the procedure and from the medicine. Stem cells from peripheral blood are transplanted to restore diseased stem cells that have been intentionally destroyed by high-dose chemotherapy and radiation in patients suffering from the disorders mentioned above. Once the transplant is finished, the healthy cells from the donor travel to the bone marrow of the patient and begin to produce new blood cells. This restores the health of a good percentage of patients, provided no complications ensue.

Only healthy individuals between the ages of eighteen and sixty are chosen as donors for this gift of life and there is a reason for such discrimination. In donors younger than eighteen years, the issue of consent stands in the way as the procedure is considered a surgical intervention. The donor’s guardian will have to be involved, both because of the law and because of ethical considerations. The limit of 60 years on the higher end is needed to make sure that the donor is relatively healthy and also to make sure that the material obtained from the donor has a good chance of survival in the recipient.

In the U.S., the National Marrow Donor Program (NMDP) provides a donor registry and communicates through a cooperative network of medical facilities present all over the country. Likewise there are similar kinds of organizations in other Western countries too. Although there are over four-and-a-half million adult volunteer donors registered in the NMDP program of the U.S., the donors from many ethnic communities, including people of the Indian sub-continent origin are difficult to find. Moreover, Asians are more polymorphic than Europeans. This means that that Asians are more likely to have more than one HLA or tissue type, thus causing more difficulty in finding a compatible donor. With a larger pool of donors, the chances for locating a compatible ethnic donor becomes somewhat easier.

Through these lines of quasi-medical information, it is hoped that some members of the Indian diaspora might become motivated to help expand the donor bank and thus help the children of ethnic-Indian origin who otherwise could be waiting desperately and painfully to receive such a life- saving gift. They may not have enough time left before the flame of life simply goes dim and then extinguishes forever. By donating such a gift to a helpless child, one could simply snatch his or her life away from the jaws of death and deliver it back to the child to experience it once again in all of its glory. In my opinion there is no better gift than saving the life of a helpless child waiting at the door of death, counting days. Such a gift not only will serve its intended purpose in saving the life of a desperate recipient but it will also bring the comfort and peace to the donor. In fact, it might well also open the doors to heaven for such a donor at the end of his or her life, especially for the one who believes in and hopes for this kind of outcome.

Dr. Jaswant Singh
Sachdev, MD
Phoenix, AZ

(Adapted from Author’s recently published book,   “SQUARE PEGS, ROUND HOLES”   The book is available by contacting the author at 602 741 8021 or emailing at jaswantsachdev@cox.net)


Major legion effort for boy

March 16, 2009

Courtesy by: georginaadvocate.com

Much like our troops, the Royal Canadian Legion is sending aid overseas.

Despite recent obstacles, branch 414 in Woodbridge accepted the challenge of helping a Romanian child in dire need of a bone marrow transplant.

“In a time we might think our doors will close, we’re putting this child ahead of us,” said Andy Straisfeld, branch 414 ways and means officer.

“We have to help the child first and we’re hoping the community will help us later,” Mr. Straisfeld said.

But George Bliderisanu, the nine-month-old child diagnosed with beta major thalassemia, or cooley anemia, isn’t waiting for some small donation.

The legion is determined to raise the 150,000 Euros ($232,000 Cdn.) needed for George’s surgery.

George’s cooley anemia is an inherited blood disorder that reduces the production of hemoglobin. Hemoglobin is found in red blood cells, those same cells that carry oxygen to other cells throughout the body.

The disorder, which progressively worsens with time, greatly affects a child’s growth process.

A bone marrow transplant is the only cure for cooley anemia and will eliminate George’s dependence on monthly blood transfusions.

But the transplant needs to happen before George shows any sign of organ problems.

So the legion is hosting several events to meet their generous goal.

In October, the organization hosted two events including a Johnny Cash concert that raised $2,000.

On Nov. 21, the legion will welcome the Three Elvis’s from Collingwood.

“We got a lot of donations from local restaurants, breweries and wineries, so whatever we can sell we can make for George,” Mr. Straisfeld said.

But it doesn’t end with Elvis either. The organization is already making plans for the spring and summer months.

“We have a big field next to the legion hall that’s perfect for a big fair, and we’re hoping to ask the Rotary and Lions clubs to help out,” Mr. Straisfeld said.

Though a lot of people have expressed an interest in donating a helping hand, Mr. Straisfeld said he has yet to secure any formal commitments.

“Being a son of Romanian parents, I made a passionate plea and the membership felt this was the right thing to do,” he said.

Though legion members willingly took on the challenge, Mr. Straisfeld was initially apprehensive about the proposal.

“I thought it would be a tough sell to the members because we have our own things to worry about,” he said. “But at the end of the day, as long as our doors are open, we can’t say no to the people we’re supposed to serve.”

One of Mr. Straisfeld’s roles is to break the stigma the legion has in the community. He said the legion will always be there for Canadian veterans, but they support other people as well.

“By helping this child, you’re helping the legion, and by helping the legion you’re helping other people,” he said.


Marrow transplants promising as cure for sickle cell

March 16, 2009

Courtesy by: Post-gazette

When Austin Jones would get a fever, it almost always meant a trip to UPMC Children’s Hospital in Pittsburgh.

But today, thanks to a life-altering bone marrow transplant he got from his older brother five years ago, a fever just means the 10-year-old has to “take some Tylenol, eat some chicken soup and go lie down,” said his father, Anthony Jones Sr. of Indiana, Pa.

Austin Jones was born with sickle cell anemia, a hereditary blood disorder that disproportionately affects African-Americans, can cause pain, organ damage, high blood pressure and strokes, and shortens life spans by 25 to 30 years.

He is one of seven patients cited in a new study by doctors at Children’s and three other institutions that could pave the way for wider use of bone marrow transplants to cure sickle cell anemia.

Doctors have known for years that bone marrow transplants are the only way to cure sickle cell for now, but the key to the new study is that it uses a much less intensive regimen of chemotherapy and radiation to prepare patients for the transplant.

In conventional marrow transplants, doctors use heavy doses of chemo and radiation to completely wipe out the patient’s blood-producing marrow, in hopes the transplanted cells will take over that function.

The risk, though, is that some patients will die from infections or an attack on their bodies by the transplanted cells known as graft-vs.-host disease.

For cancer patients, such risks are usually an acceptable trade-off if the only other option is dying from the malignancy, said Dr. Lakshmanan Krishnamurti, a Children’s cancer specialist and lead author of the study appearing in the November issue of Biology of Blood and Marrow Transplantation.

Sickle cell patients don’t face the same risk of dying from their disease, Dr. Krishnamurti said, but when they are told a typical marrow transplant will keep them in the hospital for weeks and make them very sick, many decline to go through with it.

By using less chemo and radiation, the approach taken by Dr. Krishnamurti and his colleagues stops short of wiping out the patient’s own bone marrow, though, and he believes “if you can make the transplant less toxic, people are more likely to go forward with it.”

Six of the seven patients described in the study no longer have sickle cell, which gets its name from the shape the red blood cells assume, which causes them to jam up in people’s capillaries, triggering intense pain and the other complications.

Instead, they now produce healthy, ovoid red blood cells that come from the bone marrow donated by one of their siblings.

Because their own marrow wasn’t obliterated, though, their white blood cells are a mixture of their own and their siblings’, which could even give them an advantage in fighting off infections, Dr. Krishnamurti said.

Besides being less toxic, the new procedure costs less and means less time in the hospital, he said, all of which could be especially persuasive for African-American patients, who sometimes have less health insurance and more distrust of the health care system.

For the Jones family, the less-toxic treatment protocol wasn’t a major factor, partly because Austin’s symptoms had been so severe, said his father.

Austin had his first fever and trip to Children’s when he was 9 months old, Mr. Jones said, and over the next five years, he was back in the hospital repeatedly, including for “silent strokes” — bleeding in the brain that showed no outward symptoms.

So when Dr. Krishnamurti suggested that Austin get a bone marrow transplant from his older brother Anthony Jr., the Joneses didn’t hesitate.

“When we found out Austin was diagnosed with this” after he was born, Mr. Jones said, “our prayer was ‘Lord, not only heal Austin, but Lord, eradicate this disease from the earth.’ So that prayer went forth when he was an infant, and now, when he was 5, we were hearing about transplantation, and it was an answer.”

Austin went into the hospital in the summer of 2003 and had 45 days of chemo, radiation and other preparatory treatment.

“We watched his hair fall out, watched him go down to nothing [in weight], but he was fine with it, because he had gone through a lot of procedures already in his life.

“Needles were not an issue for him. Taking pills was not an issue for him. You know, you never miss something you never had, so he was like, ‘OK, this is the way it is.’ ”

The day of the transplant, doctors took marrow from brother Anthony’s pelvis and infused it into Austin over about four hours from an IV bag.

Life was actually more challenging for the Joneses after they left the hospital, Mr. Jones said. Austin was put on immunosuppressant drugs so his body wouldn’t reject the transplant, “so you’re talking about a child who doesn’t have an immune system, and you’re trying to make your home dust-free and germ-free and you still have an 11-year-old and 9-year-old at home.”

Eventually, though, he was weaned off the immunosuppressants, just as the other patients in the study were, and his new, hybrid immune system kicked into gear.

The only patient whose transplant didn’t work, Dr. Krishnamurti said, stopped taking her immunosuppressants too soon, and even though she recovered, her sickle cell anemia returned.

An outside expert who was not part of the study agreed that the less-intense procedure might pave the way for more bone marrow transplants in sickle cell anemia.

Mary Fabry, a sickle cell researcher at Albert Einstein College of Medicine in New York, said that in Europe, where another red blood cell disorder called beta thalassemia is more prevalent, transplants are more common, because most of those patients are otherwise fated to die in their 20s or 30s.

New treatments have allowed many sickle cell patients in America to live into their 50s and longer, she said, so there has been less urgency to get marrow transplants. “The really big problem with transplantation in sickle cell disease is that the outcome of the disease has been so unpredictable.”

Seeing how it has changed his son’s life, though, Anthony Jones Sr. is a believer.

“Without reservation I would encourage anyone to pursue this,” he said. If sickle cell families were to ask his advice, “I would say, ‘You cannot not do it.’ “


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