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610 Chapter 11: Minitransplants 14. Andreani M, Manna M, Lucarelli G, Tonucci P, Agostinelli F, Ripalti M, Rapa S, Talevi N, Galimberti M, Nesci S: Persistence of mixed chimerism in patients transplanted for the treatment of thalassemia. Blood 87:3494-3499, 1996. 15. Walters MC, Patience M, Leisenring W, Eckman JR, Scott JP, Mentzer WC, Davies SC, Ohene-Frempong K, Bernaudin F, Matthews DC, Storb R, Sullivan KM: Bone marrow transplantation for sickle cell disease. N Engl J Med 335:369-376, 1996. 16. Yu C, Ostrander E, Bryant E, Burnett R, Storb R: Use of (CA) n polymorphisms to deter mine the origin of blood cells after allogeneic canine marrow grafting. Transplantation 58:701-706, 1994. 17. Storb R, Raff RF, Graham T, Appelbaum FR, Deeg HJ, Schuening FG, Shulman H, Pepe M: Marrow toxicity of fractionated versus single dose total body irradiation is identical in a canine model. Int J Radiât Oncol Biol Phys 26:275-283, 1993. 18. Storb R, Raff RF, Appelbaum FR, Graham TC, Schuening FG, Sale G, Pepe M: Comparison of fractionated to single-dose total body irradiation in conditioning canine littermates for DLA-identical marrow grafts. Blood 74:1139-1143, 1989. 19. Storb R, Raff RF, Appelbaum FR, Deeg HJ, Graham TC, Schuening FG, Shulman H, Yu C, Bryant E, Burnett R, Seidel K: DLA-identical bone marrow grafts after low-dose total body irradiation: The effect of canine recombinant hematopoietic growth factors. Blood 84:3558-3566, 1994. 20. Yu C, Storb R, Mathey B, Deeg HJ, Schuening FG, Graham TC, Seidel K, Burnett R, Wagner JL, Shulman H, Sandmaier BM: DLA-identical bone marrow grafts after low- dose total body irradiation: Effects of high-dose corticosteroids and cyclosporine on engraftment. Blood 86:4376-4381, 1995. 21. Kernan NA, Bordignon C, Heller G, Cunningham I, Castro-Malaspina H, Shank B, Flomenberg N, Burns J, Yang SY, Black P, Collins NH, O'Reilly RJ: Graft failure after T-cell-depleted human leukocyte antigen identical marrow transplants for leukemia: I. Analysis of risk factors and results of secondary transplants. Blood 74:2227-2236, 1989. 22. Bjerke JW, Lorenz J, Martin PJ, Storb R, Hansen JA, Anasetti C: Treatment of graft fail ure with anti-CD3 antibody BC3, glucocorticoids and infusion of donor hematopoietic cells (Abstract). Blood 86:107a, 1995. 23. Storb R, Yu C, Wagner JL, Deeg HJ, Nash RA, Kiem H-P, Leisenring W, Shulman H: Stable mixed hematopoietic chimerism in DLA-identical littermate dogs given sublethal total body irradiation before and pharmacological immunosuppression after marrow trans plantation. Blood 89:3048-3054, 1997. 24. Yu C, Seidel K, Nash RA, Deeg HJ, Sandmaier BM, Barsoukov A, Santos E, Storb R: Synergism between mycophenolate mofetil and cyclosporine in preventing graft-versus- host disease among lethally irradiated dogs given DLA-nonidentical unrelated marrow grafts. Blood 91:2581-2587, 1998. 25. Storb R, Deeg HJ: Failure of allogeneic canine marrow grafts after total body irradiation: Allogeneic "resistance" vs transfusion induced sensitization. Transplantation 42:571-580, 1986. 26. Sandmaier BM, Storb R, Santos EB, Krizanac-Bengez L, Lian T, McSweeney PA, Yu C, Schuening FG, Deeg HJ, Graham T: Allogeneic transplants of canine peripheral blood stem cells mobilized by recombinant canine hematopoietic growth factors. Blood
87:3508-3513,1996. Sandmaier et al. 27. Tan PHS, Santos EB, Rossbach HC, Sandmaier BM: Enhancement of natural killer activ ity by an antibody to CD44. J Immunol 150:812-820,1993. 28. Tan PH, Liu Y, Santos EB, Sandmaier BM: Mechanisms of enhancement of natural killer activity by an antibody to CD44: Increase in conjugate formation and release of tumor necrosis factor a. Cell Immunol 164:255-264,1995. 29. Raff RF, Sandmaier BM, Graham T, Loughran TP Jr, Pettinger M, Starb R: "Resistance" to DLA-nonidentical canine unrelated marrow grafts is unrestricted by the major histo compatibility complex. Exp Hematol 22:893-897,1994. 30. Raff RF, Deeg HJ, Loughran TP Jr, Graham TC, Aprile JA, Sale GE, Storb R: Characterization of host cells involved in resistance to marrow grafts in dogs transplant ed from unrelated DLA-nonidentical donors. Blood 68:861-868, 1986. 31. Sandmaier BM, Storb R, Liu Y, Santos EB, Bryant E, Schuening FG, Deeg HJ, Seidel K, Graham T: An anti-CD44 antibody does not enhance engraftment of DLA-identical marrow after low-dose total body irradiation. Transplant Immunol 4:271-274,1996. 32. Storb R, Weiden PL, Graham TC, Lerner KG, Nelson N, Thomas ED: Hemopoietic grafts between DLA-identical canine littermates following dimethyl myleran. Evidence for resistance to grafts not associated with DLA and abrogated by antithymocyte serum. Transplantation 24:349-357, 1977. 33. Weiden PL, Storb R, Graham TC, Schroeder ML: Severe hereditary haemolytic anaemia in dogs treated by marrow transplantation. Br J Haematol 33:357-362,1976. 34. Weiden PL, Hackman RC, Deeg HJ, Graham TC, Thomas ED, Storb R: Long-term sur vival and reversal of iron overload after marrow transplantation in dogs with congenital hemolytic anemia. Blood 57:66-70, 1981. 35. Thomas ED, Buckner CD, Sanders JE, Papayannopoulou T, Borgna-Pignatti C, De Stefano P, Sullivan KM, Clift RA, Storb R: Marrow transplantation for thalassaemia. Lancet ii:227-229,1982. 36. Deeg HJ, Sale GE, Storb R, Graham TC, Schuening F, Appelbaum FR, Thomas ED: Engraftment of DLA-nonidentical bone marrow facilitated by recipient treatment with anti-class II monoclonal antibody and methotrexate. Transplantation 44:340-345, 1987. 37. Sandmaier BM, Storb R, Appelbaum FR, Gallatin WM: An antibody that facilitates hematopoietic engraftment recognizes CD44. Blood 76:630-635, 1990. 38. Schuening F, Storb R, Goehle S, Meyer J, Graham TC, Deeg HJ, Appelbaum FR, Sale GE, Graf L, Loughran TP Jr: Facilitation of engraftment of DLA-nonidentical marrow by treatment of recipients with monoclonal antibody directed against marrow cells sur viving radiation. Transplantation 44:607-613,1987. 39. Sandmaier BM, Storb R, Bennett KL, Appelbaum FR, Santos EB: Epitope specificity of CD44 for monoclonal antibody dependent facilitation of marrow engraftment in a canine model. Blood 91:3494-3502,1998. 40. Hope ACA: A simplified Monte Carlo significance test procedure. J Royal Stat Soc Series B, 30:582-598,1968. 611
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AUTOLOGOUS BLOOD AND MARROW TRANSPL
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AUTOLOGOUS BLOOD AND MARROW TRANSPL
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ACKNOWLEDGMENTS We wish to thank Wi
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THE VAN BEKKUM STEM CELL AWARD Prof
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xii Table of Contents CHAPTER 2: AL
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xiv Table of Contents Late Non-Rela
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xvi Table of Contents Pretreatment
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xviii Table of Contents CHAPTER 7:
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XX Table of Contents Long-Term Obse
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xxii Table of Contents CHAPTER 13:
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0-9 LIST OF ABBREVIATIONS 2CDA 2-ch
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List of Abbreviations xxvii EFS eve
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List of Abbreviations MOPP mechlore
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VOD veno-occlusive disease VPC viru
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4HC-Purged Autologous Stem Cell Tra
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1.0-1 + 0.2 Miller et al. 5 Event F
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Miller et al. Adjusted probability
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Who Benefits From Autograft in AML
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Burnett 11 which to set the transpl
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Table 1. Outcome after relapse in M
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Immunotherapy in AML: No Positive E
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Simonsson et al. inhibit IL-2 induc
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Autologous Bone Marrow Transplantat
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Ball et al. 21 CD15) and AML-2-23 (
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Ball et al. 23 The ex vivo treatmen
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Ball et al. 25 Table 2. Factors inv
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Ball et al. Overall Survival for Pa
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Ball et al. Overall Survival for Pa
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Ball et al. 31 Overall Survival for
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Ball et al. Table 4. Actuarial over
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Ball et al. myeloid leukemia. The G
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Ball et al. 1993. 38. Mehta J, Powl
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Can Autologous Stem Cell Transplant
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De Witte et al. 41 novo AML. Auer r
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De Witte et al. hematopoietic engra
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De Witte et al. 23. Fenaux P, Laï
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Stein et al. and fever. No patient
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Stein et al. after doses 1, 3, and
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Stein et al. early after the transp
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autologous stem-cell rescue. / Clin
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Bone Marrow Transplantation for Acu
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Table 2. Autologous BMT in ALL in f
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Rowe 61 or adult patients with acut
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Hoelzer and Gôkbuget minitransplan
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Hoelzer and Gdkbuget 65 blood, earl
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Table 3. Risk factors for the defin
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Hoelzer and Gôkbuget treatment opt
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Hoeker and Gökbuget tical sibling
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Sierra et al. INTRODUCTION Two-thir
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Sierra et al. Table 2. Adverse char
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1,0 0,0 J Sierra et al. 0 20 40 60
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Sierra et al. Months Figure 4 < 30,
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Sierra et al. No adverse factors (n
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1994. Sierra et al. 83 10. Canals C
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Martin, Atta, and Hoelzer 85 adult
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Martin, Atta, and Hoelzer 87 Single
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100. Martin, Atta, and Hoelzer 89 P
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Martin, Atta, and Hoelzer 91 chromo
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Autologous vs. Unrelated Allogeneic
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Boyer and Yeager 95 outcomes after
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Boyer and Y eager 97 Table 2. Hypot
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Boyer and Y eager SUMMARY Most of t
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CHAPTER 3 CML
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Autograft Followed by Allograft Wit
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106 Chapter 3: CML (one low-grade a
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108 Chapter 3: CML DISCUSSION Myelo
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110 Chapter 3: CML Philadelphia-neg
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The Case for Manipulation of CML Au
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Toward a Cure by Drug Treatment? Th
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116 Chapter 3 :CML Table 1. Prolong
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118 Chapter 3: CML Table 3. German
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120 Chapter 3: CML Table 5. Normal
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122 Chapter 3: CML Table 8. Surviva
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124 Chapter 3: CML 88:3118-3122, 19
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126 Chapter 3: CML RK, Klingemann H
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The Tyrphostin AG957 Inhibits the I
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130 Chapter 3: CML mobilization. Al
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132 Chapter 3: CML A B 100 n 0 1 5
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134 Chapter 3: CML The in vitro sel
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136 Chapter 3: CML detecting leukem
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Progressive Hodglcin's Lymphoma Fol
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Reece et al. survival is observed i
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0.2 -I 0.0 0 Reece et al. 143 ii i
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Reece et al. Table 3. Causes of non
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Reece et al. carmustine, etoposide
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CD34 + Selection of Hematopoietic B
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Lazarus et al. 151 significantly de
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Lazarus et al. 153 (Sigma, St Louis
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Lazarus et al. 155 Table 2. Effect
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Lazarus et al. 157 difference. Furt
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Lazarus et al. 159 cells after myel
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The European CUP/UP Trial: A Prelim
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Table 1. Patient characteristics at
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Schouten et al. licular non-Hodgkin
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Sweetenham et al. vs. autologous pe
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Table 1. Patient characteristics, g
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Table 2. Patient characteristics, g
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Table 3. Sites of relapse, group A
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Table 4. Patterns of relapse, group
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A 100 80 %OS 60 40 20 B 100 %OS | S
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Sweetenham et al. new sites, and fi
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Sweetenham et al. Treatment options
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Molecular Remission: A Worthwhile A
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Early or Late Autotransplant in Hig
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Schenkein et al. 187 PATIENTS AND M
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Schenkein et al. 189 Toxicity Toxic
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Schenkein et al. 191 16. Glick JH,
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Santini et al. 193 to evaluate the
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Table 1. Continued Santini et al. 1
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Santini et al. 197 cytosine arabino
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1 0 0 1 90- 80- 70- (0 u. 60' o so-
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Hodgkin's lymphoma. N EnglJ Med 333
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CHAPTER 5 MYELOMA
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206 Chapter 5: Myeloma independent
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208 Chapter 5: Myeloma disease (MRD
Page 244 and 245:
210 Chapter 5: Myeloma Scandinavia,
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212 Chapter 5: Myeloma (0 n o B co
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214 Chapter 5: Myeloma B cq d ? o c
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216 Chapter 5: Myeloma patients wit
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218 Chapter 5: Myeloma PBSC transpl
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220 Chapter 5: Myeloma 25. Seiden M
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Autologous Transplantation in Multi
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224 Chapter 5: Myeloma 0 52 44 U §
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226 Chapter 5: Myeloma BM ^ (CD34±
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228 Chapter 5: Myeloma IFM 94 : OS
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230 Chapter 5: Myeloma two groups a
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232 Chapter 5: Myeloma increase bot
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234 Chapter 5: Myeloma REFERENCES 1
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236 Chapter 5: Myeloma Intensified
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238 Chapter 5: Myeloma of CD34 + ce
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Chapter 5: Myeloma Table 2. Descrip
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242 Chapter 5: Myeloma o 8H 0 2 4 6
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244 Chapter 5: Myeloma ated with sh
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The Presence of Micrometastases in
Page 283 and 284:
Kvalheim et al. 249 Table 1. Immuno
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Kvalheim et al. 251 Figure 1. Sixty
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Kvalheim et al. 253 DISCUSSION In t
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Kvalheim et al. 255 Bastert G: Micr
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Hood et al. 257 In an effort to inc
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#CD34 + cells in blood = Hood et al
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Hood et al. 261 Table 3. Frequency
Page 297 and 298:
Hood et al. 263 REFERENCES 1. Rill
Page 299 and 300:
The Clinical Significance of Breast
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Moss et al. tests, and bilateral po
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Moss et al. 269 0 15 70 143 200 400
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Moss et al. 271 0 3 6 12 18 24 30 3
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Moss et al. 273 8. Passos-Coelho J,
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Autotransplantation for Men With Br
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McCarthy et al. 277 radiation, two
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High Dose Sequential Chemotherapy W
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Viens et al. 281 3 g/m 2 and doxoru
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Table 1. Tumor characteristics Exte
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Table 4. Response Viens et al. 285
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Viens et al. 287 Table 5. Pathologi
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Viens et al. 289 apy for inoperable
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Gliick et al. 291 INTRODUCTION The
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Gluck et al. 293 RESULTS Patient de
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Glück et al. 295 Table 3. Response
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Gluck et al. 297 Overall Survival O
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Gluck et al. 299 Sudbury patients w
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Gluck et al. 301 anthracycline or t
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Repetitive High-Dose Therapy With P
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Prince et al. paramagnetic separati
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Prince et al. Table 1. High-dose re
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Table 2. Patient characteristics (n
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Table 4. Hematopoietic recovery fol
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1.0- M C .S 0.8- a c *fe o o> 0.6-
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M 1.0 I 0.8H s? VI o.6H S 0.2 0.0 P
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a Prince et al. days to platelets >
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Table 5. Results of Isolex 300i CD3
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Prince et al. 321 Figure 5, continu
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Prince et al. 323 months after tran
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Prince et al. 325 excessive to be u
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Prince et al. 327 eight patients ha
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Prince et al. 329 marrow transplant
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Prince et al. intensive chemotherap
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-Q CO .Q O 1.0 0.9 0.8 0.7 0.6 0.5
Page 369 and 370:
.Q CD .Q O 1.0 0.9 0.8 0.7 0.6 0.5
Page 371 and 372:
oba Q. 1.0 0.9 Dicke et al. Stage I
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Vahdat with peripheral blood progen
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Evaluation of Prognostic Factors fo
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Fields et al. 343 PATIENTS AND METH
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Table 1. Chemotherapy regimens. Fie
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Fields et al. Stage II; 4-9 Pos LN
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U U Fields et al.
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Fields et al. 351 between 1 and 2 y
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European Trends and the EBMT Databa
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25% % 22% / 17% \ Rosti et al. 355
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Rosti et al. 357 The new Italian St
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Rosti et al. 6. Haas R, Schmid H, H
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Bewick et al. 363 INTRODUCTION The
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Bewick et al. 365 Blood samples obt
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Bewick et al. 367 HDCT with ABSC su
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Bewick et al. Progression Free Surv
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Bewick et al. pression in MBC, i.e.
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Bewick et al. 14. Kandl HL, Seymour
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CHAPTER 7 OTHER SOLID TUMORS
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378 Chapter 7: Solid Tumors INTRODU
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380 Chapter 7: Solid Tumors Prepara
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382 Chapter 7: Solid Tumors Surviva
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384 Chapter 7: Solid Tumors months
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386 Chapter 7: Solid Tumors have be
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High-Dose Chemotherapy in the Manag
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390 Chapter 7: Solid Tumors followe
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392 Chapter 7: Solid Tumors Table 1
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394 Chapter 7: Solid Tumors (mucosi
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Three-Fold Intensification by Seque
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High-Dose Therapy for Small Cell Lu
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404 Chapter 7: Solid Tumors chemoth
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406 Chapter 7: Solid Tumors SCLC ha
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408 Chapter 7: Solid Tumors relapse
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410 Chapter 7: Solid Tumors 69:585-
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412 Chapter 7: Solid Tumors ogous b
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414 Chapter 7: Solid Tumors 64. Bru
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416 Chapter 7: Solid Tumors Prignot
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418 Chapter 7: Solid Tumors CO > CO
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420 Chapter 7: Solid Tumors TANDEM
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Multiple Courses of Cyclophosphamid
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424 Chapter 7: Solid Tumors and the
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426 Chapter 7: Solid Tumors Thiotep
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430 Chapter 7: Solid Tumors E 100 0
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432 Chapter 7: Solid Tumors course
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434 Chapter 7: Solid Tumors boplati
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436 Chapter 7: Solid Tumors INTRODU
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438 - Chapter 7: Solid Tumors Table
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440 Chapter 7: Solid Tumors seeded
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442 Chapter 7: Solid Tumors DISCUSS
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444 Chapter 7: Solid Tumors 10. Di
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446 Chapter 7: Solid Tumors plasma
Page 483 and 484:
Autologous Stem Cell Transplantatio
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Keystone 451 hypertension, anemia,
Page 487 and 488:
Fassas et al. 453 INTRODUCTION Mult
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Fassas et al. Table 2. Stem cell mo
Page 491 and 492:
Table 3. Toxicity after stem cell i
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Fassas et al. Table 5. Trial 1: cha
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Fassas et al. MS PROGRESSION FREE S
Page 497 and 498:
Fassas et al. ic or autologous bone
Page 499 and 500:
1 2 1 6 EAE. Burt et al. 465 Becaus
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Burt et al. Since cyclophosphamide
Page 503 and 504:
Burt et al. 469 toxicity study, we
Page 505 and 506:
Burt et dl. All 23. Mor F, Cohen IR
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Hasler, Gratwohl, and Tyndall an ev
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Hasler, Gratwohl, and Tyndall 475 B
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Kuis, Wulffraat, and Sanders 477 st
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Kuis, Wulffraat, and Sanders neousl
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CHAPTER 9 LONG-TERM EFFECTS
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484 Chapter 9: Long-Term Effects po
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486 Chapter 9: Long-Term Effects Ta
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488 Chapter 9: Long-Term Effects ml
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490 Chapter 9: Long-Term Effects 4.
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492 Chapter 9: Long-Term Effects us
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494 Chapter 9: Long-Term Effects AB
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Page 532 and 533:
498 Chapter 9: Long-Term Effects En
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500 Chapter 9: Long-Term Effects Rl
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502 Chapter 9: Long-Term Effects RE
Page 538 and 539:
504 Chapter 9: Long-Term Effects A,
Page 540 and 541:
Long-Term Follow-Up of Autologous T
Page 542 and 543:
Page 545:
CHAPTER 10 GRAFT MANIPULATION
Page 548 and 549:
514 Chapter 10: Graft Manipulation
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Page 594 and 595: 560 Chapter 10: Graft Manipulation
Page 606 and 607: Stem Cell Reinfusion Over Two Conse
Page 614 and 615: Final Report of the First Prospecti
Page 635 and 636: Canine Hematopoietic Stem Allograft
Page 637 and 638: Sandmaier et al. 603 The resultant
Page 639 and 640: Sandmaier et al. 605 synthesis path
Page 641 and 642: Sandmaier et al. 607 interactions o
Page 643: Sandmaier et al. 1991. 2. Burchenal
Page 647 and 648: Giralt, Khouri, and Champlin Table
Page 649 and 650: Giralt, Khouri, and Champlin seven
Page 651 and 652: 1.0 3 0.2 Giralt, Khouri, and Champ
Page 653: CHAPTER 12 GENE THERAPY
Page 656 and 657: 622 Chapter 12: Gene Therapy INTROD
Page 658 and 659: 624 Chapter 12: Gene Therapy RESULT
Page 660 and 661: 626 Chapter 12: Gene Therapy envisi
Page 662 and 663: 628 Chapter 12: Gene Therapy cer in
Page 664 and 665: A Vaccine of Melanoma Peptide Loade
Page 667 and 668: B43(anti-CD 19)-Gen i stein Immunot
Page 669 and 670: Targeting Immunotherapy for the Tre
Page 671 and 672: McGuinness andArceci 637 modified m
Page 673 and 674: McGuinness andArceci 639 leukemic c
Page 675 and 676: McGuinness and Arced 641 in G418. T
Page 677 and 678: McGuinness andArceci 643 Figure 2
Page 679 and 680: mRNA for hB7-1 (1491 bp) mRNA for C
Page 681 and 682: McGuinness and Arced CD80PE C080PE
Page 683 and 684: McGuinness andArceci 649 8. Robert
Page 685 and 686: McGuinness andArceci immune respons
Page 687 and 688: McGuinness andArceci 75. Jubinsky F
Page 689 and 690: Slavin et al. 655 up suggests, as p
Page 691 and 692: Slavin et al. 657 was a phase lib c
Page 693 and 694: Slavin et al. presented, appears to
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Antitumor Immunotherapy in Autologo
Page 697 and 698:
Klingemann et al. 663 Table 3. Meth
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CHAPTER 14 RADIOIMMUNOTHERAPY
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668 Chapter 14: Radioimmunotherapy
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Page 712 and 713:
Page 714 and 715:
Selection of Radioisotopes, Chelate
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Radioimmunotherapy of Common Epithe
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CHAPTER 15 NEW AVENUES
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698 Chapter 15: New Avenues Table 1
Page 734 and 735:
700 Chapter 15: New Avenues An alte
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702 Chapter 15: New Avenues who can
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704 Chapter 15: New Avenues myeloid
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706 Chapter 15: New Avenues 48. Gir
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Emerging Viral Infections in Blood
Page 744 and 745:
710 Chapter 15: New Avenues influen
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712 Chapter 15: New Avenues Among a
Page 748 and 749:
714 Chapter 15: New Avenues physica
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716 Chapter 15: New Avenues respira
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Minimal Therapy Models of Transplan
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CHAPTER 16 SUMMARIES
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724 Chapter 16: Summaries although
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726 Chapter 16: Summaries use of po
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728 Chapter 16: Summaries Table 1.
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736 Chapter 16: Summaries IL-15. Th
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738 Chapter 16: Summaries Patterns
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740 List of Participants Thomas L.
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742 List of Participants Sergio A.
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744 List of Participants Hans Marti
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746 List of Participants David P. S
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748 List of Participants AUTHOR IND
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750 Author Index Hurd, D.D. 483 Kui
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752 Author Index Stiff, Patrick J.
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754 Key Word Index Hematologic mali
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ISBN 1-891524-04-6
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