Medicare Announces Intention to Cover Intestinal and Multivisceral Transplantation
Early in October, the Health Care Financing Administration (HCFA) announced it’s intention to cover small bowel and multivisceral transplants for Medicare patients. Medicare expects to implement the policy in the spring or summer of 2001. For more information on HCFA’s policy, you may want to check HCFA’s web site at http://www.hcfa.gov/coverage/default.htm. For more information on intestinal transplants, try the Intestinal Transplant Registry web site at http://www.lhsc.on.ca/itr/ or the United Network for Organ Sharing web site at http://www.patients.unos.org/.
Most patients with short bowel syndrome and other severe gastrointestinal disorders which prevent them from maintaining a healthy nutritional status orally are maintained on home parenteral (IV) or enteral (tube-fed) nutrition (homePEN). Survival rates for homePEN patients are good, as is their prognosis for rehabilitation. (see "Survival Rates: HPN vs. SBT"). With dietary counseling and adaptation of their bowel, some of these patients are able to reduce or eliminate their need for homePEN. It is estimated that 10 to 20 percent of all patients started on homePEN will remain dependent on homePEN indefinitely.
A limited number of patients may be candidates for non-transplant surgical options. These procedures that are collectively referred to as Autologous Gastro-Intestinal Reconstruction (AGIR) can be effective in allowing freedom from HPN and its complications (see "More on AGIR").
When these options are exhausted, and a patient has ‘failed HPN,’ (usually because of liver failure or lack of IV access) then they may be a candidate for small bowel transplantation (SBT). SBT can be performed in isolation, in combination with a liver transplantation, or in combination with multiple organs (multivisceral transplantation or MVT). An isolated small bowel graft is typically recommended when a patient develop has lost venous access. Combined small bowel/liver transplants are recommended to patients with irreversible liver failure due to HPN, or intestinal failure associated with a hypercoagulable state that can be corrected by a simultaneous liver graft. Multivisceral transplants are for patients with locally aggressive tumors that can only be removed by a massive evisceration of the abdominal organs.
Intestinal transplantation in humans proved clinically feasible in the late 1980’s. Research shows the procedure is effective, but has considerable morbidity and mortality. Rejection episodes are relatively frequent, and patients develop lymphoproliferative disease and serious infections such as chronic cytomegalovirus or Epstein Barr virus. About half of the patients receiving intestinal transplants survive five years or more. This prognosis is somewhat better in young people ages 2 to 17.
Analyzing the Data
In deciding whether to cover SBT, HCFA considered several studies and two technology assessments.One of the articles, "Intestinal Transplantation: 1997 Report on the International Registry," by David Grant, et al., reported on cumulative intestinal transplantation. This article included data on 272 transplants in 269 patients from 33 intestinal transplant programs. Two-thirds of the recipients were children. Forty-one percent of the procedures were for small bowel transplants alone, 48 percent for small bowel and liver, and 11 percent for multivisceral grafts. One-year patient survival for transplants performed after February 1995 was 69 percent for small bowel alone, 66 percent for small bowel and liver transplants, and 63 percent for multivisceral.
Transplants since 1995 and programs that had performed at least 10 transplants had significantly higher patient and graft survival rates. Three quarters (77 percent) of the current survivors had stopped total parenteral nutrition and resumed oral nutrition. There was no association between type of donor, donor pretreatment or diagnosis, and graft or patient survival. Although most intestinal transplants arise from cadaveric donors, nine patients received grafts from living donors with comparable results to cadaveric transplants. According to David Grant, because most patients function well on HPN, the risks of intestinal transplantation are only warranted when standard therapies have failed.
Current patient and graft survival rates are as follows:
Two-Year SBT Survival Rates*
| Isolated Small Bowel | ||
| Liver/Samll Bowel |
* data from University of Nebraska at Omaha
Oley’s North American Home Parenteral and Enteral Nutrition Patient Registry reports survival for patients on long-term HPN to be 87 to 96 percent at one year and 70 to 90 percent at three years. Five year data are not available.
After a literature review of 211 journal articles, The Center for Practice and Technology Assessment at the Agency for Healthcare Research and Quality (AHRQ) concluded that small bowel and related transplantation appear to be potentially life-saving options for patients who have failed HPN and would therefore otherwise face certain death. In addition, AHRQ found that the data are not sufficient to determine whether the risks and benefits of small bowel transplant and related procedures might yield a net benefit to patients who can continue HPN, but are considered at high risk to fail HPN sometime in the future.
Medicare’s Decision
Medicare takes note of the following facts: there are variations in outcomes and patients require lifelong immunosuppressive therapy; the overall 1-year survival for all intestinal transplantation is approximately 70 percent, but there is a 50 percent or less chance of long-term (five years) survival. Complications following surgery are common, including rejection, cytomeglovirus disease, lymphoproliferative disease and infection (see "Complications from SBT" chart). Among intestinal transplant recipients, hospital readmission occurred once every 1 to 2 years for infection, surgical procedures and/or rejection (this is similar to the HPN hospital readmission rate). Additionally, there is limited data on quality of life following intestinal transplants, and even less comparing transplantation with home parenteral nutrition.
Compliations from SBT
| acute graft rejection | |||
| chronic graft rejection | |||
| cytomegalovirus disease | |||
| lymphoprolifertive disease |
HCFA found the evidence on which to base a determination on Medicare coverage regarding intestinal transplantation was sparse. They agreed that the various forms of intestinal transplantation (i.e., SBT, SB/LT, and MVT) may offer a life-saving therapy for patients with irreversible intestinal failure; however, because the procedure involves high risk, HCFA (and most transplant programs) believe intestinal transplantation should be reserved for patients with life-threatening complications from HPN who are expected to die without transplantation. Kishore Iyer, FRCS, from the University of Omaha Organ Transplantation Program explains, "while the results of intestinal transplantation are steadily improving, they are still inferior to that achieved with HPN and non-transplant surgical options for the patient with intestinal failure....[While] intuitively it would appear that if intestinal transplants were performed on patients before they were desperately ill, the results would be better, ...the overall outcome figures and risks following transplantation have to improve considerably before transplantation can be applied as therapy of first choice for patients with intestinal failure."
Iyer believes it is vitally important for patients who may be candidates for SBT to be referred early to intestinal transplant centers. This allows for early evaluation, with continued and coordinated care between the patient’s local medical providers and intestinal transplant physicians who can ensure that the patient does not lose an appropriate and limited window of opportunity for intestinal transplant. Early referral is especially important for children with less than 30 cm of proximal small bowel who show signs of early cirrhosis; they are at high risk for transplantation
HCFA’s Definition of Failed HPN
The clinical indications for intestinal transplantation supported by the literature are impending liver failure due to HPN, thrombosis of major central venous channels, frequent line infection and sepsis, and severe dehydration. Thus, Medicare will cover intestinal transplantation only in the following clinical situations:
* Impending or overt liver failure due to HPN induced liver injury. The clinical manifestations include elevated serum bilirubin and/or liver enzymes, splenomegaly, thrombocytopenia, gastroesophageal varices, coagulopathy, stomal bleeding or hepatic fibrosis/cirrhosis.
* Thrombosis of the major central venous channels; jugular, subclavian, and femoral veins. Thrombosis of two or more of these vessels is considered a life threatening complication and failure of HPN therapy. Central venous thrombosis may result in a lack of access for HPN infusion, recurrent sepsis due to an infected thrombi, pulmonary embolism, superior vena cava syndrome, or chronic venous insufficiency.
* Frequent line infection and sepsis. The development of two or more episodes of systemic sepsis secondary to line infection per year that requires hospitalization indicates failure of HPN therapy. A single episode of line related fungemia, septic shock and/or Acute Respiratory Distress Syndrome are considered indicators of HPN failure. [Editor’s note: The average sepsis rate for HPN adults is once every two years, and for HPN children once every year. However, many patients have periods of no infection or more frequent infection , so the decision to consider SBT should not focus too strictly on Medicare’s advised guidelines.]
* Frequent episodes of severe dehydration despite intravenous fluid supplement in addition to HPN. Under certain medical conditions such as secretory diarrhea and non-reconstructable gastrointestinal tract, the loss of the gastrointestinal and pancreatobiliary secretions exceeds the maximum intravenous infusion rates that can be tolerated by the cardiopulmonary system. Frequent episodes of dehydration injure all body organs particularly kidneys and the central nervous system with the development of multiple kidney stones, renal failure, and permanent brain damage. [Editor’s note: This is an extremely rare circumstance]
Significant bone disease, metabolic disorders, slowed development, and significant limitations on social and personal activities are not considered indications of therapy failure.
Facility Criteria
As with other organ transplants, Medicare will limit SBT coverage to centers that meet specific medical, experience and administrative criteria, such as patient selection policies, patient management protocols, and volume and outcome measures. Specifically, HCFA will limit Medicare coverage of intestinal transplantation to centers that perform 10 or more transplants per year. At this time, the University of Pittsburgh, University of Omaha and University of Miami are doing the most SBTs, followed by UCLA Medical Center and Mount Sinai in New York City.
In summary, Medicare will cover intestinal transplantation for the purpose of restoring intestinal function in patients with irreversible intestinal failure only when performed on patients who have failed HPN and only when performed in centers that meet approval criteria. Survival rates for SBT are improving but the procedure is still too risky to consider if the patient is doing well on HPN or could be a candidate for non-transplant surgery. As Kishore Iyer put it, "It would be a grave error to view any aspect of treatment in isolation, but rather as different arms of a treatment plan for the challenging patient with intestinal failure. Thus, a dedicated multi-disciplinary approach to the problem of intestinal failure with careful management of diet, HPN, treatment of associated problems such as bacterial overgrowth, judicious use of AGIR and, finally intestinal transplantation will ensure the best possible results for all patients."
More on AGIR
Kishore Iyer, FRCS & Alan Langnas, D.O
Organ Transplantation Program
University of Omaha
One aspect of treatment that has received limited attention is the group of non-transplant surgical options that are referred to as Autologous Gastro-Intestinal Reconstruction (AGIR). The best known of these procedures is the intestinal lengthening procedure of Bianchi. The Bianchi procedure takes advantage of the split blood supply to the small bowel and divides a 10 to 15 cm segment of dilated bowel in half; from one tube, it creates two, and then sews them end to end (see illustration). This both lengthens a short bowel and restores contractability to a dilated or flabby segment. The Bianchi procedure, and others such as tapering, creation of valves, reversed segments, etc. can reduce or eliminate dependency on HPN in select patients with short bowel.
Unfortunately, there are few surgeons trained to perform these procedures and these surgeons have only a small series of patients. Bianchi (who originally developed the lengthening procedure) reported (Journal of the Royal Society of Medicine, 1997) that nine of 20 children who underwent lengthening over a 15 year period, survived long term (mean follow-up of 6.4 years). Seven of the nine were completely off parenteral nutrition and two were on partial parenteral nutrition. The experience at Omaha reported (Annals of Surgery, 1995) as part of a large series of patients with short bowel has been more encouraging: of 14 patients who underwent lengthening, 12 improved, one underwent transplantation and one died. Seven patients were able to come off HPN and in five PN was decreased. Among 160 patients with short bowel syndrome, there were 32 non-transplant surgical procedures. Overall, 84 percent of these procedures led to clinical improvement — after surgery 69 percent of patients were maintained on enteral nutrition and 25 percent required less PN.