Transfusion and Apheresis Science 48 (2013) 119–121

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Transfusion and Apheresis Science

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Hemophilia comprehensive care model waiting to be duplicatedfor other rare chronic conditions

At a time when Ministers of Health are desperatelysearching for new ideas that will lead to quality care, re-duce wait times and manage costs, an old idea is waitingto be duplicated.

The model for delivery of comprehensive care forhemophilia and other inherited bleeding disorders is wellproven. First conceived in the 1970s, it quickly spreadacross Canada and the developed countries and, in the lastdecade, throughout the world. Nation-wide today, thereare 25 comprehensive care clinics each staffed by a hema-tologist, nurse coordinator, physiotherapist, psychologistor social worker, a data manager and other specialists asneeded.12

‘‘The comprehensive care model for hemophilia,’’ saysDr. Bruce Ritchie, Associate Professor of Medicine at theUniversity of Alberta and Medical Director of the Dr. JohnAkabutu Comprehensive Centre for Bleeding Disorders inEdmonton, ‘‘is a fantastic example of quality health carefor a serious, rare, chronic condition delivered rationallyand cost-effectively.’’

The model was put to an extreme test in the 1980swhen 30% of the 2500 patients with hemophilia wereinfected with HIV and 70% with hepatitis C throughplasma-derived blood products. Overnight, the focusshifted from control of bleeding episodes to a life-and-death battle against AIDS and hepatitis.

In the 1990s, with factor concentrates now safe, controland prevention of bleeding, mainly into joints and muscles,regained its role as the primary mission of the clinic teams.Regular prophylactic infusions of clotting factor concen-trates, notably factors VIII and IX, delivered in the homesetting by the patients themselves or their caregivers be-came the standard of care. Since then, 80% of the factorconcentrates distributed in Canada have been infused out-side hospital walls by non-professionals. Today, manylarge hemophilia treatment centres receive fewer than 10visits a week from the hundreds of patients registered.

http://dx.doi.org/10.1016/j.transci.2013.02.036

1 Pritchard AM, Page D. Integrated specialty service readiness in healthreform: connections in haemophilia comprehensive care. Haemophilia2008;14(3):436–43.

2 Srivastava A et al. Guidelines for the management of hemophilia.Haemophilia 2012:1–47. doi:10.1111/j.1365-2516.2012.02909.x.

Treatment in emergency departments has become a rarity.Patients visit the hemophilia treatment centre in persononly once or twice a year for routine assessments, but stayin regular communication by telephone, e-mail, electronicinfusion reporting systems and even tele-health. Musculo-skeletal health has improved dramatically. So has qualityof life.3

So successful is the model, many hospital administra-tors forget about hemophilia. Out of sight, out of mind.Critical services such as physiotherapy are sometimes cutback, saving on hospital budgets but costing the bloodbudget dearly, due to decreased education and prevention,and increased need for rehabilitation combined with high-er consumption of factor concentrates.

Nevertheless, despite some staffing shortages, thehemophilia treatment centre model for care delivery re-mains an example to be duplicated. Patient satisfaction isvery high. Outcomes are comparable with world standards.Many other people with serious, rare, chronic conditionswould benefit tremendously from similar services.

In 2004, a group of patient associations serving peoplewith rare hematological and immunological disorders,many of them treated with blood and blood products,banded together to create a coalition called the Networkof Rare Blood Disorder Organizations (NRBDO). It includedgroups representing patients with bleeding disorders, pri-mary immune deficiency, hereditary angioedema, thalas-semia, sickle cell disease, thrombotic thrombocytopenicpurpura (TTP) and a handful of others. Many of the condi-tions require exactly the same kind of care as hemophilia:a dedicated multi-disciplinary team, education of the pa-tient and family, home infusion of plasma protein productsand provincial recognition of programs so as to providespecialized care. At the first meeting, representatives ofthe Canadian Hemophilia Society were dismayed to learnthat none of the groups enjoyed the level of care that peo-ple with hemophilia take for granted. Very quickly, the

3 Noone et al. A survey of the outcome of prophylaxis, on-demandtreatment or combined treatment in 18–35-year old men with severehaemophilia in six countries. Haemophilia 2012:1–7. doi:10.1111/j.1365-2516.2012.02934.x.

Table 1

Inherited bleedingdisorders

Primary immunedeficiency

Hereditary angioedema Thalassemia Sickle celldisease

ThromboticThrombocytopenicPurpura (TTP)

Provincial designation BC, Alberta, Saskatchewan,Manitoba, Quebec

No No No No No

National patient registries Yes Under construction No No No Underconstruction

Self/family administration of blood products Yes BC, Alberta, Ontario,Quebec

Alberta, Manitoba, Ontario,Quebec, NB, PEI, NS,Newfoundland and Labrador

NA NA NA

Formalized patient, family and association involvement incare

Yes No No No No No

Education of patients, families and health care providers Yes No No No Yes NoStandards of care and portability Yes No No No No Locally, in

developmentDecentralization through outreach Yes, but limited No No No No NoMulti-disciplinary care Yes, with some staff shortages Alberta, Montreal,

Quebec CityAlberta, Montreal, Quebec City Toronto,

MontrealNo Needed outside of

crisesDefined core services delivered by a comprehensive care

teamYes, with some staff shortages Alberta, Montreal,

Quebec CityAlberta, Montreal, Quebec City Toronto,

MontrealToronto,Montreal

NA

Programme evaluation and accreditation Self-evaluation No No No No NoNational collaboration among health care professionals

and patient organizationsYes Limited Limited Limited Limited Limited

Post-marketing surveillance including blood-bornepathogen surveillance

Yes No No No No No

Collaborative national research Yes No No No No LimitedFlexibility in organization Yes NA NA NA NA NA

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goal of comprehensive care for all became the primaryobjective of the NRBDO.

‘‘We understood that there was a better way,’’ saidLanre Tunji-Ajayi, current chair of the NRBDO and Execu-tive Director of the Sickle Cell Awareness Group of Ontario.‘‘Dedicated clinics with inter-disciplinary teams couldmake all the difference in the world for people withchronic blood disorders.’’

Major conferences bringing together patients, healthcare providers, researchers and representatives of Cana-dian Blood Services, Héma-Québec, and the provincesand territories, were held in 2006 and 2009. A genericmodel for delivery of comprehensive care was developedand adopted by all.4

Yet progress has been agonizingly slow. Table 1 lists the14 characteristics of a comprehensive care program as de-fined by the NRBDO in 2006 and where six of the diseasegroups stand with regard to implementation.

The consequences for the under-served disease groupsare obvious: sub-optimal care, increased morbidity andeven mortality, and reduced quality of life. On the otherhand, the benefits of duplicating the hemophilia compre-hensive care model go beyond better health and improvedquality of life. All of these conditions, optimally treated, al-low people to lead long, productive lives. Dedicated nursecoordinators can take on much of the work that now fallsto physicians, many of whom, because they meet peoplewith these conditions only very occasionally, lack the re-quired training. Patients currently treated in over-crowdedemergency departments can be cared for in dedicated clin-ics and, in many cases, in the home setting. The compre-hensive care model facilitates better tracking ofexpensive blood products and can lead to more rationalutilization and cost savings. Specialists, trained in the careof these rare conditions, can avoid inappropriatetreatment.

Plasma protein products and their recombinant alterna-tives, the core treatments for inherited bleeding disorders,

4 2006 Conference proceedings (http://www.hemophilia.ca/en/about-the-chs/collaboration/network-of-rare-blood-disorder-organizations/).

primary immune deficiency and hereditary angioedema,account for close to 50% of the budget of Canadian BloodServices and Héma-Québec. In 2010–2011, these productscost the Canadian health care system 600 million dollars.Avoiding wastage and inappropriate prescription throughspecialized care and better utilization management, possi-ble only in a context of comprehensive care, could savemillions, more than enough to ensure the resourcesneeded for comprehensive care.

Certainly, no one solution fits all. A very large city likeToronto may be able to create comprehensive care clinicsfor each condition. A sparsely populated province likeNewfoundland and Labrador may need to create a singlemulti-disciplinary team that can treat all of these disorderswith the ability to do outreach across the province. Leavingthe care of these complex conditions under the responsi-bility of regional health authorities is not a viable option;the conditions are too rare. Specialized resources must bebrought together, if only virtually. Provincial designationand systematic care delivery are required.

Anyone unconvinced by this case need only spend fivehours in an emergency department with a person whosethroat is swelling and closing due to hereditary angioe-dema, or experience the level of care provided to a youngman in pain from sickle cell disease.

‘‘We have the knowledge, expertise and treatmentproducts in this country,’’ says Dr. Bruce Ritchie. ‘‘We havethe model of care. All that’s needed is the will to try a newway, one that is not so new after all.’’

David PageNational Executive Director,

Canadian Hemophilia Society, Canada