myeloablative agonists


Summary: Agents that destroy bone marrow activity. They are used to prepare patients for BONE MARROW TRANSPLANTATION or STEM CELL TRANSPLANTATION.

Top Publications

  1. Chae Y, Sohn S, Kim J, Cho Y, Moon J, Shin H, et al. New myeloablative conditioning regimen with fludarabine and busulfan for allogeneic stem cell transplantation: comparison with BuCy2. Bone Marrow Transplant. 2007;40:541-7 pubmed
    ..168; 0.035-0.807; P=0.026). The replacement of cyclophosphamide with fludarabine for myeloablative conditioning seems to be more effective in terms of short-term NRM, and GVHD compared to BuCy2 regimen in allogeneic transplantation. ..
  2. Dudley M, Yang J, Sherry R, Hughes M, Royal R, Kammula U, et al. Adoptive cell therapy for patients with metastatic melanoma: evaluation of intensive myeloablative chemoradiation preparative regimens. J Clin Oncol. 2008;26:5233-9 pubmed publisher
    ..Host lymphodepletion followed by autologous TIL transfer and IL-2 results in objective response rates of 50% to 70% in patients with metastatic melanoma refractory to standard therapies. ..
  3. Sorror M, Maris M, Storer B, Sandmaier B, Diaconescu R, Flowers C, et al. Comparing morbidity and mortality of HLA-matched unrelated donor hematopoietic cell transplantation after nonmyeloablative and myeloablative conditioning: influence of pretransplantation comorbidities. Blood. 2004;104:961-8 pubmed
    ..After adjustment for pretransplantation differences between the 2 patient groups, the hazard ratio was 3.0 (P=.04). Multivariate analyses showed higher pretransplantation comorbidity scores to result in increased toxicity and mortality. ..
  4. Perez Simon J, Diez Campelo M, Martino R, Brunet S, Urbano A, Caballero M, et al. Influence of the intensity of the conditioning regimen on the characteristics of acute and chronic graft-versus-host disease after allogeneic transplantation. Br J Haematol. 2005;130:394-403 pubmed
  5. Alyea E, Kim H, Ho V, Cutler C, Gribben J, DeAngelo D, et al. Comparative outcome of nonmyeloablative and myeloablative allogeneic hematopoietic cell transplantation for patients older than 50 years of age. Blood. 2005;105:1810-4 pubmed
    ..052). Our experience suggests that, in patients over age 50, NST with fludarabine and low-dose busulfan leads to an overall outcome at least as good as that following myeloablative therapy. ..
  6. Arora M, McGlave P, Burns L, Miller J, Barke J, DeFor T, et al. Results of autologous and allogeneic hematopoietic cell transplant therapy for multiple myeloma. Bone Marrow Transplant. 2005;35:1133-40 pubmed
    ..Allogeneic HCT may be suitable in younger patients, soon after diagnosis, and in those with chemosensitive disease. ..
  7. Kennedy G, Butler J, Morton J, Hill G, Western R, Cummings J, et al. Myeloablative allogeneic stem cell transplantation for advanced stage multiple myeloma: very long-term follow up of a single center experience. Clin Lab Haematol. 2006;28:189-97 pubmed
    ..Efforts should continue on alternative allogeneic SCT approaches to reduce the high early TRM rate associated with myeloablative conditioning. ..
  8. Iravani M, Evazi M, Mousavi S, Shamshiri A, Tavakoli M, Ashouri A, et al. Fludarabine and busulfan as a myeloablative conditioning regimen for allogeneic stem cell transplantation in high- and standard-risk leukemic patients. Bone Marrow Transplant. 2007;40:105-10 pubmed
  9. Busca A, Locatelli F, Barbui A, Ghisetti V, Cirillo D, Serra R, et al. Infectious complications following nonmyeloablative allogeneic hematopoietic stem cell transplantation. Transpl Infect Dis. 2003;5:132-9 pubmed
    ..Whether these findings would translate into an improved overall survival needs to be confirmed in larger prospective studies. ..

More Information


  1. Diaz M, Lassaletta A, Perez A, Sevilla J, Madero L, Gonzalez Vicent M. High-dose busulfan and melphalan as conditioning regimen for autologous peripheral blood progenitor cell transplantation in high-risk ewing sarcoma patients: a long-term follow-up single-center study. Pediatr Hematol Oncol. 2010;27:272-82 pubmed publisher
    ..Patients with localized disease at diagnosis and those with good response to treatment before or after transplant would benefit most. ..
  2. Kim J, Goulston C, Sanders S, Lampas M, Zangari M, Tricot G, et al. Cytomegalovirus reactivation following autologous peripheral blood stem cell transplantation for multiple myeloma in the era of novel chemotherapeutics and tandem transplantation. Biol Blood Marrow Transplant. 2012;18:1753-8 pubmed publisher
    ..CMV surveillance should be considered, especially when tandem transplantation is performed using combination chemotherapy with high-dose melphalan. ..
  3. Burt R, Craig R, Milanetti F, Quigley K, Gozdziak P, Bucha J, et al. Autologous nonmyeloablative hematopoietic stem cell transplantation in patients with severe anti-TNF refractory Crohn disease: long-term follow-up. Blood. 2010;116:6123-32 pubmed publisher
    ..This trial was registered at as NCT0027853. ..
  4. Arber C, Halter J, Stern M, Rovo A, Gratwohl A, Tichelli A. Graft source determines human hematopoietic progenitor distribution pattern within the CD34(+) compartment. Bone Marrow Transplant. 2011;46:650-8 pubmed publisher
  5. Taupin P. Transplantation of two populations of stem cells to improve engraftment: WO2008060932. Expert Opin Ther Pat. 2010;20:1259-63 pubmed publisher
    ..The procedure may be extended to other types of stem cells for treating a broad range of diseases and injuries. ..
  6. Omazic B, Remberger M, Barkholt L, Söderdahl G, Potácová Z, Wersäll P, et al. Long-Term Follow-Up of Allogeneic Hematopoietic Stem Cell Transplantation for Solid Cancer. Biol Blood Marrow Transplant. 2016;22:676-681 pubmed publisher
    ..002), and HLA-identical transplant (HR, 5.00; P = .03). With none of these risk factors, survival at 6 years was 50% (n = 6). Long-term survival can be achieved in some patients with solid cancer after HSCT. ..
  7. Lopes J, Jorge S. Acute kidney injury following HCT: incidence, risk factors and outcome. Bone Marrow Transplant. 2011;46:1399-408 pubmed publisher
    ..Herein, we provide a comprehensive and contemporary discussion of the incidence, risk factors and outcome of AKI in patients undergoing HCT, focusing on the differences between the myeloablative and nonmyeloablative regimens. ..
  8. Devine S, Carter S, Soiffer R, Pasquini M, Hari P, Stein A, et al. Low risk of chronic graft-versus-host disease and relapse associated with T cell-depleted peripheral blood stem cell transplantation for acute myelogenous leukemia in first remission: results of the blood and marrow transplant clinical trials network. Biol Blood Marrow Transplant. 2011;17:1343-51 pubmed publisher
    ..HCT after myeloablative chemoradiotherapy can be performed in a multicenter setting using a uniform method of TCD, resulting in a low risk of extensive chronic GVHD and relapse for patients with AML in CR1. ..
  9. Novitzky N, Thomas V. In the absence of clinically significant graft vs. host disease, myeloablative conditioning may allow an effective graft vs. leukaemia effect. Leuk Res. 2012;36:104-9 pubmed publisher
    ..At a median of 1018 days 65% are alive. Grade >1 GvHD was seen in 11%. GvHD and adverse karyotype were associated with treatment failure. In younger patients preservation of the dose intensity may improve cure rates. ..
  10. Wingard J, Carter S, Walsh T, Kurtzberg J, Small T, Baden L, et al. Randomized, double-blind trial of fluconazole versus voriconazole for prevention of invasive fungal infection after allogeneic hematopoietic cell transplantation. Blood. 2010;116:5111-8 pubmed publisher
    ..This trial was registered at as NCT00075803. ..
  11. Cassaday R, Gopal A. Allogeneic hematopoietic cell transplantation in mantle cell lymphoma. Best Pract Res Clin Haematol. 2012;25:165-74 pubmed publisher
  12. Sala Torra O, Martin P, Storer B, Traina F, Sorror M, Storb R, et al. Serious acute or chronic graft-versus-host disease after hematopoietic cell transplantation: a comparison of myeloablative and nonmyeloablative conditioning regimens. Bone Marrow Transplant. 2008;41:887-93 pubmed publisher
    ..Serious GVHD may be considered as an endpoint in clinical trials with GVHD-related outcomes. ..
  13. Khouri I, McLaughlin P, Saliba R, Hosing C, Korbling M, Lee M, et al. Eight-year experience with allogeneic stem cell transplantation for relapsed follicular lymphoma after nonmyeloablative conditioning with fludarabine, cyclophosphamide, and rituximab. Blood. 2008;111:5530-6 pubmed publisher
    ..Only 5 patients were still undergoing immunosuppressive therapy at the time of last follow-up. We believe that the described results are a step forward toward developing a curative strategy for recurrent follicular lymphoma. ..
  14. Cook G, Smith G, Kirkland K, Lee J, Pearce R, Thomson K, et al. Outcome following Reduced-Intensity Allogeneic Stem Cell Transplantation (RIC AlloSCT) for relapsed and refractory mantle cell lymphoma (MCL): a study of the British Society for Blood and Marrow Transplantation. Biol Blood Marrow Transplant. 2010;16:1419-27 pubmed publisher
    ..For patients relapsing post-AlloSCT, the disease is salvageable with DLI. The timing of RIC-AlloSCT should be explored in prospective studies to establish the optimal role in the management of this aggressive lymphoma. ..
  15. Bayraktar U, Bashir Q, Qazilbash M, Champlin R, Ciurea S. Fifty years of melphalan use in hematopoietic stem cell transplantation. Biol Blood Marrow Transplant. 2013;19:344-56 pubmed publisher
    ..This review summarizes the development and the current use of this remarkable drug in hematopoietic SCT. ..
  16. Martin H, Poe M, Provenzale J, Kurtzberg J, Mendizabal A, Escolar M. Neurodevelopmental outcomes of umbilical cord blood transplantation in metachromatic leukodystrophy. Biol Blood Marrow Transplant. 2013;19:616-24 pubmed publisher
    ..As in other leukodystrophies, early intervention correlated with optimal outcomes. We conclude that UCB transplantation benefits children with presymptomatic late-infantile MLD or minimally symptomatic juvenile MLD. ..
  17. Poon L, Hamdi A, Saliba R, Rondon G, Ledesma C, Kendrick M, et al. Outcomes of adults with acute lymphoblastic leukemia relapsing after allogeneic hematopoietic stem cell transplantation. Biol Blood Marrow Transplant. 2013;19:1059-64 pubmed publisher
    ..8). Our data provide more insight into the disease behavior and treatment outcomes of ALL at relapse after HSCT against which future trials may be compared. ..
  18. Chang J, JUCKETT M, Callander N, Kahl B, Gangnon R, Mitchell T, et al. Thalidomide maintenance following high-dose melphalan with autologous stem cell support in myeloma. Clin Lymphoma Myeloma. 2008;8:153-8 pubmed publisher
    ..8 months, and the median OS was > 60 months. Thalidomide maintenance at a goal dose of 200 mg per day was not feasible in this population, with our data suggesting that 100 mg per day is a more reasonable maintenance dose. ..
  19. Delgado J, Marco A, Moreno E, Piñana J, Valcarcel D, Martino R, et al. Reduced-intensity conditioning allogeneic hematopoietic cell transplantation using oral fludarabine as part of the conditioning regimen. Cytotherapy. 2009;11:356-61 pubmed publisher
    ..In addition, oral fludarabine can be more convenient for patients and caregivers, facilitating its implementation. ..
  20. Delgado J, Milligan D, Dreger P. Allogeneic hematopoietic cell transplantation for chronic lymphocytic leukemia: ready for prime time?. Blood. 2009;114:2581-8 pubmed publisher
    ..Finally, we have addressed the most relevant factors when deciding what patients should be considered for allo-HCT and the timing of the procedure. ..
  21. Mori T, Tanaka M, Kobayashi T, Ohashi K, Fujisawa S, Yokota A, et al. Prospective multicenter study of single-unit cord blood transplantation with myeloablative conditioning for adult patients with high-risk hematologic malignancies. Biol Blood Marrow Transplant. 2013;19:486-91 pubmed publisher
    ..These results suggest that the IMSUT CBT procedures can safely provide a high disease-free survival rate in patients with high-risk hematologic malignancies. ..
  22. Abidi M, Agarwal R, Tageja N, Ayash L, Deol A, Al Kadhimi Z, et al. A phase I dose-escalation trial of high-dose melphalan with palifermin for cytoprotection followed by autologous stem cell transplantation for patients with multiple myeloma with normal renal function. Biol Blood Marrow Transplant. 2013;19:56-61 pubmed publisher
    ..This higher dose of melphalan has the potential to improve the efficacy and, hopefully, outcomes of patients with MM with a single ASCT. A phase 2 trial is necessary to better delineate the antimyeloma efficacy of this regimen. ..
  23. Shah A. Successful pregnancy after busulfan/cytoxan conditioning regimen for AML. J Pediatr Hematol Oncol. 2011;33:e180-1 pubmed publisher
    ..In this case report we report the case of a 27 year-old female who had a normal full term delivery 19 years following a myeloablative autologous transplant for relapsed acute myelogenous leukemia...
  24. de Lima M, Porter D, Battiwalla M, Bishop M, Giralt S, Hardy N, et al. Proceedings from the National Cancer Institute's Second International Workshop on the Biology, Prevention, and Treatment of Relapse After Hematopoietic Stem Cell Transplantation: part III. Prevention and treatment of relapse after allogeneic transpla. Biol Blood Marrow Transplant. 2014;20:4-13 pubmed publisher
  25. Shi Q, Fahs S, Wilcox D, Kuether E, Morateck P, Mareno N, et al. Syngeneic transplantation of hematopoietic stem cells that are genetically modified to express factor VIII in platelets restores hemostasis to hemophilia A mice with preexisting FVIII immunity. Blood. 2008;112:2713-21 pubmed publisher
  26. Khandelwal P, Mellor Heineke S, Rehman N, Lane A, Smiley K, Villanueva J, et al. Cytokine Profile of Engraftment Syndrome in Pediatric Hematopoietic Stem Cell Transplant Recipients. Biol Blood Marrow Transplant. 2016;22:690-697 pubmed publisher
    ..The observation of elevated IL-1β suggests that engraftment syndrome could be an inflammasome mediated phenomenon. ..
  27. Oran B, Wagner J, DeFor T, Weisdorf D, Brunstein C. Effect of conditioning regimen intensity on acute myeloid leukemia outcomes after umbilical cord blood transplantation. Biol Blood Marrow Transplant. 2011;17:1327-34 pubmed publisher
    ..UCB with RIC extends the use of allogeneic HCT for older and frail patients without excessive TRM with greater benefit for patients in CR1 and CR2 with longer CR1. ..
  28. Dalakas M, Smith S. A "nema" of hope in the treatment of late-onset nemaline myopathy. Neurology. 2008;71:472-3 pubmed publisher
  29. Mori T, Koh H, Onishi Y, Kako S, Onizuka M, Kanamori H, et al. Impact of cyclophosphamide dose of conditioning on the outcome of allogeneic hematopoietic stem cell transplantation for aplastic anemia from human leukocyte antigen-identical sibling. Int J Hematol. 2016;103:461-8 pubmed publisher
    ..067). These results suggest that CY dose can safely be reduced without increasing graft rejection by adding fludarabine in allogeneic HSCT for aplastic anemia from an HLA-identical sibling. ..
  30. Perkins J, Kim J, Anasetti C, Fernandez H, Perez L, Ayala E, et al. Maximally tolerated busulfan systemic exposure in combination with fludarabine as conditioning before allogeneic hematopoietic cell transplantation. Biol Blood Marrow Transplant. 2012;18:1099-107 pubmed publisher
    ..Assessment of the higher busulfan AUC on relapse prevention requires trials in patients with a homogeneous risk of relapse. ..
  31. Kim H, Lee J, Joo Y, Bae S, Hyun M, Lee J, et al. A randomized comparison of cyclophosphamide vs. reduced dose cyclophosphamide plus fludarabine for allogeneic hematopoietic cell transplantation in patients with aplastic anemia and hypoplastic myelodysplastic syndrome. Ann Hematol. 2012;91:1459-69 pubmed publisher
    ..The 4-year survival rate did not differ between the Cy-ATG and Cy-Flu-ATG arms. Preconditioning with Cy-Flu-ATG was superior to that afforded by Cy-ATG in terms of reducing RRT levels without increasing engraftment failure. ..
  32. Ueno N, Rizzo J, Demirer T, Cheng Y, Hegenbart U, Zhang M, et al. Allogeneic hematopoietic cell transplantation for metastatic breast cancer. Bone Marrow Transplant. 2008;41:537-45 pubmed
    ..These findings support the need for preclinical and clinical studies that facilitate targeted adoptive immunotherapy for breast cancer to explore the benefit of a GVT effect in breast cancer. ..
  33. Wood W, Deal A, Abernethy A, Basch E, Battaglini C, Kim Y, et al. Feasibility of frequent patient-reported outcome surveillance in patients undergoing hematopoietic cell transplantation. Biol Blood Marrow Transplant. 2013;19:450-9 pubmed publisher
    ..Future studies in larger populations to explore predictive models using frequent PRO data for outcomes, including long-term HRQOL and survival, are warranted. ..
  34. Artz A, Wickrema A, Dinner S, Godley L, Kocherginsky M, Odenike O, et al. Pretreatment C-reactive protein is a predictor for outcomes after reduced-intensity allogeneic hematopoietic cell transplantation. Biol Blood Marrow Transplant. 2008;14:1209-16 pubmed publisher
    ..These data require confirmation in non-T cell-depleted conditioning regimens. If validated, they suggest that preconditioning CRP holds promise for enhancing estimates of transplantation tolerance. ..
  35. Krishnamurti L, Kharbanda S, Biernacki M, Zhang W, Baker K, Wagner J, et al. Stable long-term donor engraftment following reduced-intensity hematopoietic cell transplantation for sickle cell disease. Biol Blood Marrow Transplant. 2008;14:1270-8 pubmed publisher
    ..These findings demonstrate the curative potential of allogeneic HCT after an RIC regimen in patients with SCD. ..
  36. Comenzo R, Steingart R, Cohen A. High-dose melphalan versus melphalan plus dexamethasone for AL amyloidosis. N Engl J Med. 2008;358:92; author reply 92-3 pubmed
  37. Thirugnanam R, George B, Chendamarai E, Lakshmi K, Balasubramanian P, Viswabandya A, et al. Comparison of clinical outcomes of patients with relapsed acute promyelocytic leukemia induced with arsenic trioxide and consolidated with either an autologous stem cell transplant or an arsenic trioxide-based regimen. Biol Blood Marrow Transplant. 2009;15:1479-84 pubmed publisher
  38. Frossard V, Ketterer N, Rosselet A, Meier P, Cairoli A, Duchosal M, et al. Early intensification and autologous stem cell transplantation in patients with systemic AL amyloidosis: a single-centre experience. Ann Hematol. 2009;88:681-5 pubmed publisher
    ..This suggests that high-dose chemotherapy and ASCT are still valid treatment options in AL amyloidosis and that a significant number of patients with renal involvement might benefit from this approach. ..
  39. Wareham N, Heilmann C, Abrahamsson J, Forestier E, Gustafsson B, Ha S, et al. Outcome of poor response paediatric AML using early SCT. Eur J Haematol. 2013;90:187-94 pubmed publisher
    ..6 years (range 0.4 - 8.1 years) and 3-year probability of survival 70% (95% CI 59-77%). The poor responders in the NOPHO-AML 2004 protocol had a favourable prognosis treated with time-intensive induction followed by SCT. ..
  40. Ramanujam M, Davidson A. BAFF blockade for systemic lupus erythematosus: will the promise be fulfilled?. Immunol Rev. 2008;223:156-74 pubmed publisher
    ..Further work will be needed both in animal models and humans to determine the most appropriate clinical applications for BAFF blockade. ..
  41. Baronciani D, Rambaldi A, Iori A, Di Bartolomeo P, Pilo F, Pettinau M, et al. Treosulfan/fludarabine as an allogeneic hematopoietic stem cell transplant conditioning regimen for high-risk patients. Am J Hematol. 2008;83:717-20 pubmed publisher
    ..Longer follow-up and further prospective studies are necessary to evaluate this regimen. ..
  42. Lin M, Epport K, Azen C, Parkman R, Kohn D, Shah A. Long-term neurocognitive function of pediatric patients with severe combined immune deficiency (SCID): pre- and post-hematopoietic stem cell transplant (HSCT). J Clin Immunol. 2009;29:231-7 pubmed publisher
    ..These restrictions keep patients from acquiring developmentally appropriate cognitive skills as well as gross and fine motor developmental milestones. Longitudinal follow-up will be important to quantify acquisition of skills. ..
  43. Kierdorf K, Katzmarski N, Haas C, Prinz M. Bone marrow cell recruitment to the brain in the absence of irradiation or parabiosis bias. PLoS ONE. 2013;8:e58544 pubmed publisher
  44. Mignot A, Varnous S, Redonnet M, Jaccard A, Epailly E, Vermes E, et al. Heart transplantation in systemic (AL) amyloidosis: a retrospective study of eight French patients. Arch Cardiovasc Dis. 2008;101:523-32 pubmed publisher
    ..Appropriate haematological therapy, including MD, may result in a survival benefit in AL amyloidosis patients with advanced heart failure requiring transplantation. ..
  45. Lazarus H, Kan F, Tarima S, Champlin R, Confer D, Frey N, et al. Rapid transport and infusion of hematopoietic cells is associated with improved outcome after myeloablative therapy and unrelated donor transplant. Biol Blood Marrow Transplant. 2009;15:589-96 pubmed publisher
    ..These data support a general review of current transport procedures, especially for BM grafts requiring longer transport time and every effort made to minimize time from collection to infusion. ..
  46. Bautista G, Regidor C, Gonzalo Daganzo R, Cabrera J. [Umbilical cord blood cell transplantation from an unrelated donor: dual transplantation]. Methods Find Exp Clin Pharmacol. 2010;32 Suppl A:47-51 pubmed
    ..Opportunistic infections have occurred over a long period of time. This procedures makes allogeneic hematopoietic transplantation accessible to almost all patients. ..
  47. Nakamura Y, Usui F, Ono T, Takeda K, Nirasawa K, Kagami H, et al. Germline replacement by transfer of primordial germ cells into partially sterilized embryos in the chicken. Biol Reprod. 2010;83:130-7 pubmed publisher
    ..This novel germline replacement technique provides a powerful tool for studying germline differentiation, for generating transgenic individuals, and for conserving genetic resources in birds. ..
  48. Armand P, Sainvil M, Kim H, Rhodes J, Cutler C, Ho V, et al. Pre-transplantation iron chelation in patients with MDS or acute leukemia and iron overload undergoing myeloablative allo-SCT. Bone Marrow Transplant. 2013;48:146-7 pubmed publisher
  49. Al Hashmi S, Hassan Z, Sadeghi B, Rozell B, Hassan M. Dynamics of early histopathological changes in GVHD after busulphan/cyclophosphamide conditioning regimen. Int J Clin Exp Pathol. 2011;4:596-605 pubmed
    ..This is the first study describing early histopathological changes after conditioning regimen with busulphan/cyclophosphamide and dynamics of GVHD development in several major internal organs...
  50. Mickelson D, Sproat L, Dean R, Sobecks R, Rybicki L, Kalaycio M, et al. Comparison of donor chimerism following myeloablative and nonmyeloablative allogeneic hematopoietic SCT. Bone Marrow Transplant. 2011;46:84-9 pubmed publisher
    ..Donor T-cell chimerism of ? 90% was significantly associated with development of extensive chronic GVHD. The value of routine surveillance of chimerism following any of the preparative regimens used in this study should be reevaluated. ..
  51. Główka F, Romański M, Wachowiak J. High-dose treosulfan in conditioning prior to hematopoietic stem cell transplantation. Expert Opin Investig Drugs. 2010;19:1275-95 pubmed publisher
    ..However, we expect TREO to be registered as a novel conditioning agent in the near future. ..
  52. George B, Mathews V, Viswabandya A, Kavitha M, Srivastava A, Chandy M. Fludarabine based reduced intensity conditioning regimens in children undergoing allogeneic stem cell transplantation for severe aplastic anemia. Pediatr Transplant. 2008;12:14-9 pubmed
    ..36.3%; p = 0.133) and improved event free (85.7 vs. 54.5%; p = 0.177) and overall survival (85.7 vs. 63.6%; p = 0.350). Flu with Cy +/- ATG reduces rejection and improves overall and event free survival in children with aplastic anemia. ..
  53. Duchartre Y, Petit N, Moya C, Lalanne M, Dubus P, Verneuil H, et al. Neonatal bone marrow transplantation prevents liver disease in a murine model of erythropoietic protoporphyria. J Hepatol. 2011;55:162-70 pubmed publisher
    ..We demonstrated for the first time that BMT could be successfully used to prevent liver disease in EPP mice and suggested that BMT would be an attractive therapeutic option to prevent severe liver dysfunction in EPP patients. ..