Cancer Treatment Today » Anemia

Ferriprox for sickle cell – pro

M Levin, MD — Wed, 17 Jul 2013 18:25:17 +0000

FERRIPROX® (deferiprone) is an iron chelator indicated for the treatment of patients with transfusional iron overload due to thalassemia syndromes when current chelation therapy is inadequate. The approval in second line is reasonable both because the studies for approval were done in second line and because Ferroprox may be inferior to Exjade in first line (Cemak et al). This is not an innocuous drug; the most serious side effect seen in about two percent of patients treated with Ferriprox was the development of agranulocytosis, a serious and potentially life-threatening reduction in the number of granulocytes (a type of white blood cell that fights infection). The therapy is being approved under the FDA’s accelerated approval program, designed to provide patients with earlier access to promising new drugs followed by further studies to confirm the drug’s clinical benefit. The accelerated approval program allows the agency to approve a drug to treat a serious disease based on clinical data showing that the drug has an effect on an endpoint that is reasonably likely to predict a clinical benefit to patients, or on an effect on a clinical endpoint other than survival or irreversible morbidity (illness).

ApoPharma has agreed to several post-marketing requirement and commitments. One commitment includes further study of the use of Ferriprox in patients with sickle cell disease who have transfusional iron overload. One such study is: Absorption, Metabolism, and Excretion of a Single Dose of Ferriprox® in Patients With Sickle Cell Disease, NCT01835496.

For Lay version see here

At this time, there is not sufficient literature support for the use of Ferriptox in sickle cell disease.

Cermak, Jaroslav,2011, http://www.eventure-online.com/eventure/publicAbstractView.do?id=161926&congressId=4634

Ferriprox, Prescribing Information, 2013

Cyclosporine for aplastic anemia – pro

M Levin, MD — Tue, 23 Apr 2013 23:32:54 +0000

Aplastic anemia in younger patients is generally immune mediated and is treated with immunosupression. When there is no sibling donor, cyclosporine(CSA) is recommended, usually in combination with other drugs but it can also be used alone(1). First-line treatment approaches include immunosuppressive treatment using the combination of antithymocyte globulin and cyclosporine A for patients without a sibling donor and HLA identical sibling transplant for patients younger than age 40 with a donor(2). Despite a theoretical rationale for its use, sirolimus did not improve the response rate in patients with severe aplastic anemia when compared to standard h-ATG/CsA(3). CsA is usually dosed on day 1 to a target trough level between 200 and 400 ng/mL, starting at a dose of 10 mg/kg per day (in children, 15 mg/kg per day).

1.http://aphcon.org/aplastic-anemia-treatment-guidelines.html, 2013

2. Jakob R. Passweg et al, Aplastic Anemia: First-line Treatment by Immunosuppression and Sibling Marrow Transplantation ASH Education Book December 4, 2010 vol. 2010 no. 1 36-42

3.Scheinberg P, Wu CO, Nunez O, et al.(2009) Treatment of severe aplastic anemia with a combination of horse antithymocyte globulin and cyclosporine, with or without sirolimus: a prospective randomized study. Haematologica 94:348354.

4.Phillip Scheinberg1and Neal S. Young, .How I treat acquired aplastic anemia Blood August 9, 2012 vol. 120 no. 6 1185-1196

For Lay version see here

Promacta for Aplastic Anemia

Red cell growth factors for Aplastic Anemia

Erythropoietin therapy for cardiomyopathy – pro

M Levin, MD — Sun, 28 Oct 2012 03:36:52 +0000

Raising Hemoglobin levels is not always an unmitigated belssing. Studies in dialysis patients showed an increased risk of complications when Hb is raised above HB of 10-22. Meta-analysis of reported randomized clincal trials(RCT)s conclude that normalization of hemoglobin with erythropoietin is harmful.

The connection between heart failure(HF) and Hb levels appears to exist but it is not certain that low Hb worsens heart failure. Three studies have looked at raising Hb in HF, one with transfusion and two with erythropietin and produced inconsistent results, with only one of them, by Mancini et al, a randomized study, but it was a small study.

Erythropoietin receptors are present in a variety of tissues, including the heart, and erythropoietin may have anti-inflammatory and antiapoptotic properties. Recently published studies have demonstrated that treatment with rHuEPO favorably attenuated ischemia-reperfusion injury in a mouse model. Whether these findings will have physiologic relevance in humans remains unknown, but they do suggest an additional potential mechanism for beneficial effects of erythropoietin treatment in human HF. On the other hand, higher Hb levels may increase the physiologic stress on the heart and increase hypertension and stroke. Several studies are ongoing . Clearly, routine adoption of erythropoietic agents to treat HF and cardiomyopathy is premature.

Robert N. Foley, Bryan M. Curtis, and Patrick S. ParfreyErythropoietin Therapy, Hemoglobin Targets, and Quality of Life in Healthy Hemodialysis Patients: A Randomized Trial Clin J Am Soc Nephrol. 2009 April; 4(4): 726–733

Mancini D.M., Katz S.D., Lamanca J., Lamanca J., Hudaihed A., Androne A.S.; Effect of erythropoietin on exercise capacity in patients with moderate to severe chronic heart failure, Circulation 107 2003 294-299

Bohlius J, et al. Recombinant human erythropoesis stimulating agents and moratlity in patients

with cancer. A meta analysis of randomized trials. The Lancet,2009;373:1532.

Tonelli M, et al. Benefits and Harms of erythropoesis stimulating agents for anemia related to cancer: a metaanalysis. CMAJ,2009;180(11):e62-71.

Kotecha D, et al. Erythropoetin as a treatment of anemia in heart failure: systemic review of randomized trials. Am Heart J,2011;161(5):822.

Pfeffer MA, et al. A trial of darbepoetin alfa in type 2 diabetis and chronic kidney disease. N Engl J Med,2009;361:2019.

Skali H, et al. Stroke in patients with type 2 diabetes CKD, and anemia treated with darboepotin alpha. Circulation,2011;124:2903.

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Gastric bypass – pro

M Levin, MD — Fri, 31 Aug 2012 13:13:53 +0000

As weight loss begins to slow down after gastric bypass, the risk of nutritional problems increases. This is due to dysfunctional or bypassed small bowel. B12 and iron deficiency are two of the most common problems and often do not respond to typical multivitamin supplementation.

Iron deficiency after gastric bypass is usually only seen in menstruating women or in patients who are actively and chronically bleeding. Ferritin or iron levels and erythrocyte counts need to be monitored after a bypass, as iron deficiency can develop early after surgery or years later; one study found that iron stores continuously declined up to 7 years after bypass surgery. Due to bypass of the lower stomach, in which iron is absorbed, it is very difficult for iron-deficient patients to absorb sufficient oral iron. Many cannot tolerate read meat. Intramuscular iron can be impractical over the long run. Usually, intravenous iron dextran or iron sucrose is used regularly; many patients require intravenous iron several times a year. This is done as an outpatient procedure and is well tolerated by patients.

Brolin RE et al. Prophylactic iron supplementation after Roux-en Y gastric bypass: a prospective, double blind, randomized study. Arch Surg. 1998;133(7):740-744.

Dimitrios V Avgerinos, Omar H Llaguna, Matthew Seigerman, Amanda J Lefkowitz, and I Michael Leitman
Incidence and risk factors for the development of anemia following gastric bypass surgery, World J Gastroenterol. 2010 April 21; 16(15): 1867–1870.

Love AL, Billett HH. Obesity, bariatric surgery, and iron deficiency: true, true, true and related.Am J Hematol. 2008 May;83(5):403-9.

Ferraheme, Ferrlecit or Venofer for iron deficency anemia – pro

M Levin, MD — Fri, 31 Aug 2012 13:11:34 +0000

As IV iron preparation have become safer, they are increasingly being used. However, the oral route is still best, when possible. Ferraheme is the latest IV iron entrant. Feraheme™ (ferumoxytol) Injection is indicated for the treatment of iron deficiency anemia in adult patients with chronic kidney disease (CKD). There are four commercially available i.v. iron products, iron dextran, iron sucrose, and iron ferric gluconate, or Ferrlecit and Ferraheme. The use of iron dextran has decreased because of the risk of anaphylaxis. Iron sucrose recently received Food and Drug Administration (FDA)-approved labeling for the treatment of iron deficiency anemia in NDDCKD patients, making it the first of the non-dextran iron supplements to receive such approval. Before this approval, both iron sucrose and iron ferric gluconate were indicated only for the treatment of iron deficiency in dialysis patients. Ferrlecit is FDA approved for treating iron deficiency anemia in patients undergoing hemodialysis who are also receiving epoetin therapy.

Anemia is a common complication of kidney disease. Although erythropoietin deficiency is the most important cause of anemia in patients with kidney disease, iron deficiency is common and can complicate treatment by causing a relative resistance to epoetin alfa therapy. I.V. iron is widely used in hemodialysis patients but less so in patients with non-dialysis-dependent chronic kidney disease (NDDCKD). Although oral iron can be used in the latter patients, its use is limited by adverse effects, poor compliance, and the long time period required to replete iron stores.

Indications for the use of intravenous iron include chronic uncorrectable bleeding, intestinal malabsorption, intolerance to oral iron, nonadherence, or a hemoglobin level less than 6 g per dL (60 g per L) with signs of poor perfusion in patients who would otherwise receive transfusion (e.g., those who have religious objections). Until recently, iron dextran (Dexferrum) has been the only parenteral iron preparation available in the United States. Unlike ther IV iron preparations it is approved for anemia of iron deficiency. The advantage of iron dextran over Ferrlecit is the ability to administer large doses (200 to 500 mg) at one time. One major drawback of iron dextran is the risk of anaphylactic reactions that can be fatal. There also is a delayed reaction, which consists of myalgias, headache, and arthralgias, that can occur 24 to 48 hours after infusion. Nonsteroidal anti-inflammatory drugs will usually relieve these symptoms, but they may be prolonged in patients with chronic inflammatory joint disease.

In conclusion, Venofer and Ferrlecit is not FDA approved for iron deficiency but for dialysis only. This is because the dialysis group is the largest iron deficency group in the USA and is the group in which FDA required studies had beeen conducted. Off-label use of Venofer or Ferrlecit is appropriate when oral iron was not tolerated. Iron extran is FDA approved for iron deficiency in general.

When oral iron did not work, IV iron in the dose and schedule proposed is medically appropriate. However, Feraheme™ (ferumoxytol) Injection is indicated for the treatment of iron deficiency anemia in adult patients with chronic kidney disease (CKD). The recommended dose of Feraheme is an initial 510 mg intravenous injection followed by a second 510 mg intravenous injection 3 to 8 days later. SINCe it is being given for an indiviudla without kidney disease, it is off-label. Ia gree with the denila of Ferroheme, but other IV prearations that are specifically indicated for iron deficiency alone, are acceptable. A number of IV iron formulations are available, including ferric carboxymaltose (FCM), ferric gluconate (FG), ferumoxytol, iron sucrose (IS), iron isomaltoside (termed ferric derisomaltose in the United States and Australia), and low molecular weight iron dextran (LMW ID). Monoferic and iron dexstran are FDA approved in adults patients who have an intolerance or had unsatisfactory response to oral iron or or who have non-hemodialysis dependent chronic kidney disease (CKD).

Scott B. Silverstein, Intravenous Iron Therapy: A Summary of Treatment Options and Review of Guidelines, Journal of Pharmacy Practice December 2008 vol. Camaschella C. Iron-deficiency anemia. N Engl J Med 2015; 372:1832.

Lu M, Cohen MH, Rieves D, Pazdur R.
FDA report: Ferumoxytol for intravenous iron therapy in adult patients with chronic kidney disease.
Am J Hematol. 2010 May;85(5):315-9.

Ferreiro-Iglesias R, Barreiro-de-Acosta M, Seijo-Ríos S, Lorenzo A, Domínguez-Muñoz JE.
Efficacy of intravenous iron in treating iron deficiency anaemia in patients with inflammatory bowel disease. Are there predictors of response? [Article in English, Spanish]Rev Esp Enferm Dig. 2011 May;103(5):245-9.

Breymann C, Milman N, Mezzacasa A, et al. Ferric carboxymaltose vs. oral iron in the treatment of pregnant women with iron deficiency anemia: an international, open-label, randomized controlled trial (FER-ASAP). J Perinat Med 2016.

Abdulrehman J, Tang GH, Auerbach M, et al. The safety and efficacy of ferumoxytol in the treatment of iron deficiency: a systematic review and meta-analysis. Transfusion 2019; 59:3646.Breymann C, Milman N, Mezzacasa A, et al. Ferric carboxymaltose vs. oral iron in the treatment of pregnant women with iron deficiency anemia: an international, open-label, randomized controlled trial (FER-ASAP). J Perinat Med 2016.

Abdulrehman J, Tang GH, Auerbach M, et al. The safety and efficacy of ferumoxytol in the treatment of iron deficiency: a systematic review and meta-analysis. Transfusion 2019; 59:3646.

Macdougall IC, Strauss WE, McLaughlin J, Li Z, Dellanna F, Hertel J. A randomized comparison of ferumoxytol and iron sucrose for treating iron deficiency anemia in patients with CKD. Clin J Am Soc Nephrol. 2014;9(4):705–712. Onken JE, Bregman DB, Harrington RA, et al. Ferric carboxymaltose in patients with iron-deficiency anemia and impaired renal function: the REPAIR-IDA trial. Nephrol Dial Transplant. 2014;29(4):833-842.

Onken JE, Bregman DB, Harrington RA, et al. A multicenter, randomized, active-controlled study to investigate the efficacy and safety of intravenous ferric carboxymaltose in patients with iron deficiency anemia. Transfusion. 2014;54(2):306-315.

Screening for hemachromatosis – pro

M Levin, MD — Fri, 31 Aug 2012 13:10:11 +0000

Current understanding of hemachormatosis incorporates the fact that some 30% of patients with familial occurrence of iron overload associated with C282Y homozygosity or C282Y/H63D compound heterozygosity never develop symptoms of the disease and this number is higher in menstruating females. It is also now appreciated that this is a disease with a long horizon before organ damage occurs and that close followup makes immediate diagnosis unnecessary in all comers. Consequently guidelines recommend screening for hemochromatosis only in patients with abnormal iron studies, liver disease or end organ damage.

REFERENCES:

National Institute of Diabetes, Digestive and Kidney Diseases. Hemochromatosis.Last Update: March 2014. Available at: http://digestive.niddk.nih.gov/ddiseases/pubs/hemochromatosis/ Accessed March 24, 2016.

Bruce R Bacon, et al, Diagnosis and Management of Hemochromatosis: 2011 Practice Guideline by the American Association for the Study of Liver Diseases. Hepatology. 2011 Jul; 54(1): 328–343.

Bardou-Jacquet E, Morcet J, Manet G, et al. Decreased cardiovascular and extrahepatic cancer-related mortality in treated patients with mild HFE hemochromatosis. J Hepatol 2015; 62:682.

Plasmapheresis in autoimmune hemolytic anemia – pro

M Levin, MD — Thu, 30 Aug 2012 02:15:50 +0000

Autoimmune hemolytic anemia (AIHA) due to the presence of warm agglutinins is almost always due to IgG antibodies that react with protein antigens on the red blood cell (RBC) surface at body temperature. For this reason, they are called “warm agglutinins” even though they seldom directly agglutinate the RBCs. IV Gammaglobulin blocks this process.

I some cases, AIHA can be characterised by a chronic course and an unsatisfactory control of haemolysis, thus requiring prolonged immunosuppressive therapy. Sometimes when medical measures fail, it may be necessary to surgically remove the spleen (splenectomy). The clinical course of the disease may show either resistance to steroids or dependence on high-dose steroids with subsequent development of severe side effects on growth, bone mineralisation, and the endocrine system. Splenectomy is effective in about 50 to 60 percent of the time in IgG antibody diseases but is not usually effective in IgM antibody haemolysis. Splenectomy is of benefit in these people because the spleen behaves like a sieve and if it is removed, even though the RBCs are coated by antibodies, they are no longer caught and destroyed in the spleen.

IVIG is an accepted treatment for autoimmune hemolytic anemia. Unlike steroids, it does not induce remissions but is a temporizing measure until a definitve treatment can be planned and delivered. IVIG is not as effective in AIHA as it is in ITP. Other treatments can sometimes be used.

Plasma exchange (plasmapheresis), is a procedure in which blood is removed and its components (red blood cells, platelets, and plasma) separated. The plasma is discarded while the blood cells and platelets are transfused into the patient along with a plasma substitute. Plasmapheresis is used in several autoimmune conditions to reduce immunoglobulins. For AIHA, there are several case reports of it being effective in refractory cases but no prospective studies have been performed.

Ucar K. Clinical presentation and management of hemolytic anemias. Oncology [Huntingt] 2002;16(9 suppl 10):163-70.

Schwartz RS, Berkman EM, Silberstein LE. Autoimmune hemolytic anemias. In: Hoffman R, Benz EJ Jr, Shattil SJ, Furie B, Cohen HJ, Silberstein LE, et al., eds. Hematology: basic principles and practice. 3d ed. Philadelphia: Churchill Livingstone, 2000:624.

S;Garelli etal, Plasma exchangefor a hemolytic crisis due to autoimmune hemolytic anemia of the IgG warm type Journal Annals of Hematology
Issue Volume 41, Number 5 / November, 1980 387-391

Fabio Aglieco et al, A Case Report of Refractory Warm Autoimmune Hemolytic Anemia Treated With Plasmapheresis and RituximabTherapeutic Apheresis and Dialysis Therapeutic Apheresis and Dialysis Volume 12 Issue 2, Pages 185 – 189

Procrit for anemia of Hepatitis C treatment – pro

M Levin, MD — Thu, 30 Aug 2012 02:08:51 +0000

Procrit is appropriate in the anemias due to erytrhopoietin underproduction or deficiency. In Hepatitis C it is usually both as well as due to chronic illness.

Several studies have evaluated the use of recombinant human erythropoietin alfa (rHuEPO) (Procrit and Epogen) for the treatment of ribavirin/interferon-induced anemia in HCV-infected patients. In a letter to the editor of Hepatology, researchers in France reported the results of a study of 13 patients with HCV, one of whom was also HIV-infected, who developed anemia on interferon alfa-2b/ribavirin treatment and who were treated with rHuEPO.

In one of the first studies, 9 patients received high dose (6 million units 3 times a week) and 3 patients low-dose (3 million units 3 times a week) interferon alfa-2b and 1 patient received PEG-interferon alfa-2b at 80 µg per week. Ribavirin was given at 1 gram or 1.2 gram per day. When anemia developed, rHuEPO was given at a dose of 4,000 units 2 to 3 times weekly to 11 patients, 8,000 units 3 times weekly to 1 patient, and 10,000 units 3 times weekly to 2 patients.

The baseline median hemoglobin in the 13 patients was 13.3 g/dL and the median hemoglobin nadir on interferon/ribavirin was 10.2 g/dL. On rHuEPO, the hemoglobin increased to a median of 11.5 g/dL and none of the patients on rHuEPO stopped treatment due to anemia. It should be noted, however, that the ribavirin dose was also decreased to 800 mg in 3 patients, to 600 mg in 1 patient, and to 200 mg in 1 patient (who had cirrhosis and HIV). The authors reported that 10 patients had an initial response to interferon/ribavirin treatment, with 4 achieving a sustained response, 5 still under treatment or follow-up, and 1 patient relapsed.

The authors conclude, “in our cohort of patients with chronic hepatitis C treated with interferon/ribavirin combination therapy, rHuEPO was beneficial in the treatment of ribavirin-induced anemia and allowed for the maintenance of a generally efficient ribavirin dosage. “Well-designed clinical trials with larger numbers of patients would be useful to establish the benefit of rHuEPO in this clinical situation as well as the optimal dose and frequency of administration.” This report supports the use of rHuEPO in patients with interferon/ribavirin induced anemia. Further studies are underway to evaluate the questions raised by the authors in their conclusion.

A recent study examined the relationship between ribavirin administration and endogenous erythropoietin production. Adequate endogenous erythropoietin production was demonstrated in patients with compensated liver disease in response to ribavirin-induced hemolytic anemia. The response was maintained, although the anemia was not corrected because of ongoing hemolysis and the continuous intake of ribavirin. The authors challenged the 40,000-U/week subcutaneous dosage administered in clinical trials because it was 3 times higher than the physiologic increase in erythropoietin production in response to ribavirin-induced anemia.

In a study of 46 patients with HCV infection, monotherapy with standard or peginterferon caused hemoglobin levels to decline below baseline values in 7 days, with a compensatory 70-96% increase in endogenous erythropoietin levels.] Reticulocyte production did not increase over baseline values; this finding represented an inadequate response to increased erythropoietin level and contributed to the development of anemia. Therefore, despite increased erythropoietin levels, the compensatory response could not overcome the suppressive effects of interferon alfa on bone marrow.This suggests that erytrhopoietin is not useful in this situation. It remains investigational and more and larger studies remain to be done.

Samit Hirawat, Stuart M. Lichtman, Steven L. Allen : Recombinant human erythropoietin use in hemolytic anemia due to prosthetic heart valves: A promising treatment American Journal of Hematology: 66, 3, 224-226, 2001

A. Gergely and others. Treatment of ribavirin/interferon-induced anemia with erythropoietin in patients with hepatitis C. Hepatology 2002; 35:1281.

Dev A, Patel K, Muir A, McHutchison JG. Erythropoietin for ribavirin-induced anemia in hepatitis C: more answers but many more questions. Am J Gastroenterol 2003;98(11): 2344-7.

Durante Mangoni E, Marrone A, Saviano D, et al. Normal erythropoietin response in chronic hepatitis C patients with ribavirin-induced anaemia. Antivir Ther 2003;8(1):57-63.

Peck-Radosavljevic M, Wichlas M, Homoncik-Kraml M, et al. Rapid suppression of hematopoiesis by standard or pegylated interferon-alpha. Gastroenterology 2002;123(1):141-51.

Ortho Biotech Products, L.P. Procrit (epoetin alfa) full prescribing information. Raritan, NJ; 2004.

Oxandrin and other androgens for Fanconi anemia – pro

M Levin, MD — Thu, 30 Aug 2012 02:01:50 +0000

Androgen steroids are male hormones that can stimulate the production of red blood cells (the cells which carry oxygen in the blood) and platelets (cells that help blood clot) and oxandrin is a more recenty approved androgen. Approximately half of FA patients respond well to androgens (male hormones), which stimulate the production of red blood cells, and often, platelets. Sometimes white cell production is stimulated as well. This treatment may be effective for many years, but most patients eventually fail to respond. It is essential that the use of androgens is considered in the context of an eventual bone marrow transplant, as their use may affect adversely the ultimate success of a transplant.

This drug is in a current study: Oxandrolone for the Treatment of Bone Marrow Aplasia in Fanconi Anemia, NCT00243399. The primary purpose of this study is to evaluate the safety of the drug oxandrolone in patients with Fanconi anemia (FA), and secondarily to determine if this drug can help in the treatment of bone marrow failure in these patients. It is hoped that oxandrolone will have less side effects than oxymetholone, the androgen used most frequently in the short-term treatment of bone marrow failure in FA patients. The study s to prove its superiority but it is not expected to be inferior to any other androgen.

http://jcem.endojournals.org/cgi/reprint/86/11/5108.pdf?ck=nck

Alter BP. Inherited bone marrow failure syndromes. In: Nathan DG, Orkin SH, Ginsburg D, Look T, eds. Hematology of Infancy and Childhood. 6th ed. Philadelphia, Pa: Harcourt Health Sciences; 2003:280-365.

Bagby GC, Alter BP. Fanconi anemia. Semin Hematol. Jul 2006;43(3):147-56.

Stem cell transplantation for Fanconi’s Anemia – pro

M Levin, MD — Thu, 30 Aug 2012 01:51:12 +0000

Fanconi anemia, or FA, is a rare, inherited blood disorder that leads to bone marrow failure. A variety of supportive treatments is available but stem cell transplantation is the only curative option (gene transfer therapies are still experimental). The two year survival rate is around 70%. Alternative conditioning regimens using fludarabine and avoiding irradiation have shown promising results so far. For those patients without matched sibling stem cell source, the prognosis for unrelated donors is around 20–40% survival at two years and there is a higher risk of graft rejection in somatic mosaics.
Stem cell transplantation is generally accepted as medically appropriate.

M D Tischkowitz and S V Hodgson
Fanconi anaemia
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M Ayas et al, Stem cell transplantation for patients with Fanconi anemia with low-dose cyclophosphamide and antithymocyte globulins without the use of radiation therapy
Bone Marrow Transplantation (2005) 35, 463–466.

Rackoff WD. Treatment of bone marrow failure. In: Owen J, ed. Fanconi anemia – standards for clinical care. Fanconi Anemia Research Fund Inc, 1902 Jefferson Street, Suite 2, Eugene, Oregon 97405, 1999:9–20.55.

Guardiola P, Socie G, Pasquini R, Dokal I, Ortega JJ, van Weel-Sipman M, Marsh J, Locatelli F, Souillet G, Cahn JY, Ljungman P, Miniero R, Shaw J, Vermylen C, Archimbaud E, Bekassy AN, Krivan G, Di Bartolomeo P, Bacigalupo AL, Gluckman E. Allogeneic stem cell transplantation for Fanconi anaemia. Severe Aplastic Anaemia Working Party of the EBMT and EUFAR. European Group for Blood and Marrow Transplantation. Bone Marrow Transplant1998;21(suppl 2):S24–7.59.

McCloy M, Almeida A, Daly P, Vulliamy T, Roberts IA, Dokal I. Fludarabine-based stem cell transplantation protocol for Fanconi’s anaemia in myelodysplastic transformation. Br J Haematol2001;112:427–9

MacMillan ML, Auerbach AD, Davies SM, Defor TE, Gillio A, Giller R, Harris R, Cairo M, Dusenbery K, Hirsch B, Ramsay NK, Weisdorf DJ, Wagner JE. Haematopoietic cell transplantation in patients with Fanconi anaemia using alternate donors: results of a total body irradiation dose escalation trial. Br J Haematol2000;109:121–9