Sporadic late-onset nemaline myopathy with MGUS: long-term follow-up after melphalan and SCT.
OBJECTIVE
Sporadic late-onset nemaline myopathy (SLONM) is a rare, late-onset myopathy that progresses subacutely. If associated with a monoclonal gammopathy of unknown significance (MGUS), the outcome is unfavorable: the majority of these patients die within 1 to 5 years of respiratory failure. This study aims to qualitatively assess the long-term treatment effect of high-dose melphalan (HDM) followed by autologous stem cell transplantation (SCT) in a series of 8 patients with SLONM-MGUS.


METHODS
We performed a retrospective case series study (n = 8) on the long-term (1-8 years) treatment effect of HDM followed by autologous SCT (HDM-SCT) on survival, muscle strength, and functional capacities.


RESULTS
Seven patients showed a lasting moderate-good clinical response, 2 of them after the second HDM-SCT. All of them had a complete, a very good partial, or a partial hematologic response. One patient showed no clinical or hematologic response and died.


CONCLUSIONS
This case series shows the positive effect of HDM-SCT in this rare disorder. Factors that may portend an unfavorable outcome are a long disease course before the hematologic treatment and a poor hematologic response. Age at onset, level and type of M protein (κ vs λ), and severity of muscle weakness were not associated with a specific outcome.


METHODS
This study provides Class IV evidence that for patients with SLONM-MGUS, HDM-SCT increases the probability of survival and functional improvement.
From the Departments of Neurology (N.C.V., B.G.v.E.) and Pathology (M.L.), Radboud University Medical Centre, Nijmegen, the Netherlands; Department of Internal Medicine (O.B.), Hôpital Pitié Salpêtrière, UPMC, U974, DHU I2B, Paris, France; Department of Hematology (M.C.M., H.L.), University Medical Center Utrecht, the Netherlands; Department of Pathology (M.L.), Antwerp University Hospital, University of Antwerp, Edegem; Departments of Internal Medicine (W.M.) and Hematology (M.D.), University Hospital Leuven, Belgium; Neurology Service (T.K., J.N.), Lausanne University Hospital CHUV, Lausanne; Department of Medical Oncology (T.P.), University Hospital Bern, Switzerland; Electromyography and Neuromuscular Pathologies Department Lyon Est (F.B.), Bron Hospitals, France; Departments of Pathology (N.R.) and Neurology (B.E.), Hôpital Pitié Salpêtrière, Myology Institute, Paris; Department of Hematology (V.L., P.v.d.B.), Hôpital Pitié Salpêtrière, UPMC Univ Paris 6 GRC 11 GRECHY, Paris, France; Neuromuscular Reference Centre, Department of Neurology (V.L., P.v.d.B.), and Department of Hematology (M.C.V.), Cliniques Universitaires Saint-Luc, University of Louvain, Brussels, Belgium. nicol.voermans@radboudumc.nl.
Nicol C
NC
Voermans
From the Departments of Neurology (N.C.V., B.G.v.E.) and Pathology (M.L.), Radboud University Medical Centre, Nijmegen, the Netherlands; Department of Internal Medicine (O.B.), Hôpital Pitié Salpêtrière, UPMC, U974, DHU I2B, Paris, France; Department of Hematology (M.C.M., H.L.), University Medical Center Utrecht, the Netherlands; Department of Pathology (M.L.), Antwerp University Hospital, University of Antwerp, Edegem; Departments of Internal Medicine (W.M.) and Hematology (M.D.), University Hospital Leuven, Belgium; Neurology Service (T.K., J.N.), Lausanne University Hospital CHUV, Lausanne; Department of Medical Oncology (T.P.), University Hospital Bern, Switzerland; Electromyography and Neuromuscular Pathologies Department Lyon Est (F.B.), Bron Hospitals, France; Departments of Pathology (N.R.) and Neurology (B.E.), Hôpital Pitié Salpêtrière, Myology Institute, Paris; Department of Hematology (V.L., P.v.d.B.), Hôpital Pitié Salpêtrière, UPMC Univ Paris 6 GRC 11 GRECHY, Paris, France; Neuromuscular Reference Centre, Department of Neurology (V.L., P.v.d.B.), and Department of Hematology (M.C.V.), Cliniques Universitaires Saint-Luc, University of Louvain, Brussels, Belgium.
Olivier
O
Benveniste
From the Departments of Neurology (N.C.V., B.G.v.E.) and Pathology (M.L.), Radboud University Medical Centre, Nijmegen, the Netherlands; Department of Internal Medicine (O.B.), Hôpital Pitié Salpêtrière, UPMC, U974, DHU I2B, Paris, France; Department of Hematology (M.C.M., H.L.), University Medical Center Utrecht, the Netherlands; Department of Pathology (M.L.), Antwerp University Hospital, University of Antwerp, Edegem; Departments of Internal Medicine (W.M.) and Hematology (M.D.), University Hospital Leuven, Belgium; Neurology Service (T.K., J.N.), Lausanne University Hospital CHUV, Lausanne; Department of Medical Oncology (T.P.), University Hospital Bern, Switzerland; Electromyography and Neuromuscular Pathologies Department Lyon Est (F.B.), Bron Hospitals, France; Departments of Pathology (N.R.) and Neurology (B.E.), Hôpital Pitié Salpêtrière, Myology Institute, Paris; Department of Hematology (V.L., P.v.d.B.), Hôpital Pitié Salpêtrière, UPMC Univ Paris 6 GRC 11 GRECHY, Paris, France; Neuromuscular Reference Centre, Department of Neurology (V.L., P.v.d.B.), and Department of Hematology (M.C.V.), Cliniques Universitaires Saint-Luc, University of Louvain, Brussels, Belgium.
Monique C
MC
Minnema
From the Departments of Neurology (N.C.V., B.G.v.E.) and Pathology (M.L.), Radboud University Medical Centre, Nijmegen, the Netherlands; Department of Internal Medicine (O.B.), Hôpital Pitié Salpêtrière, UPMC, U974, DHU I2B, Paris, France; Department of Hematology (M.C.M., H.L.), University Medical Center Utrecht, the Netherlands; Department of Pathology (M.L.), Antwerp University Hospital, University of Antwerp, Edegem; Departments of Internal Medicine (W.M.) and Hematology (M.D.), University Hospital Leuven, Belgium; Neurology Service (T.K., J.N.), Lausanne University Hospital CHUV, Lausanne; Department of Medical Oncology (T.P.), University Hospital Bern, Switzerland; Electromyography and Neuromuscular Pathologies Department Lyon Est (F.B.), Bron Hospitals, France; Departments of Pathology (N.R.) and Neurology (B.E.), Hôpital Pitié Salpêtrière, Myology Institute, Paris; Department of Hematology (V.L., P.v.d.B.), Hôpital Pitié Salpêtrière, UPMC Univ Paris 6 GRC 11 GRECHY, Paris, France; Neuromuscular Reference Centre, Department of Neurology (V.L., P.v.d.B.), and Department of Hematology (M.C.V.), Cliniques Universitaires Saint-Luc, University of Louvain, Brussels, Belgium.
Henk
H
Lokhorst
From the Departments of Neurology (N.C.V., B.G.v.E.) and Pathology (M.L.), Radboud University Medical Centre, Nijmegen, the Netherlands; Department of Internal Medicine (O.B.), Hôpital Pitié Salpêtrière, UPMC, U974, DHU I2B, Paris, France; Department of Hematology (M.C.M., H.L.), University Medical Center Utrecht, the Netherlands; Department of Pathology (M.L.), Antwerp University Hospital, University of Antwerp, Edegem; Departments of Internal Medicine (W.M.) and Hematology (M.D.), University Hospital Leuven, Belgium; Neurology Service (T.K., J.N.), Lausanne University Hospital CHUV, Lausanne; Department of Medical Oncology (T.P.), University Hospital Bern, Switzerland; Electromyography and Neuromuscular Pathologies Department Lyon Est (F.B.), Bron Hospitals, France; Departments of Pathology (N.R.) and Neurology (B.E.), Hôpital Pitié Salpêtrière, Myology Institute, Paris; Department of Hematology (V.L., P.v.d.B.), Hôpital Pitié Salpêtrière, UPMC Univ Paris 6 GRC 11 GRECHY, Paris, France; Neuromuscular Reference Centre, Department of Neurology (V.L., P.v.d.B.), and Department of Hematology (M.C.V.), Cliniques Universitaires Saint-Luc, University of Louvain, Brussels, Belgium.
Martin
M
Lammens
From the Departments of Neurology (N.C.V., B.G.v.E.) and Pathology (M.L.), Radboud University Medical Centre, Nijmegen, the Netherlands; Department of Internal Medicine (O.B.), Hôpital Pitié Salpêtrière, UPMC, U974, DHU I2B, Paris, France; Department of Hematology (M.C.M., H.L.), University Medical Center Utrecht, the Netherlands; Department of Pathology (M.L.), Antwerp University Hospital, University of Antwerp, Edegem; Departments of Internal Medicine (W.M.) and Hematology (M.D.), University Hospital Leuven, Belgium; Neurology Service (T.K., J.N.), Lausanne University Hospital CHUV, Lausanne; Department of Medical Oncology (T.P.), University Hospital Bern, Switzerland; Electromyography and Neuromuscular Pathologies Department Lyon Est (F.B.), Bron Hospitals, France; Departments of Pathology (N.R.) and Neurology (B.E.), Hôpital Pitié Salpêtrière, Myology Institute, Paris; Department of Hematology (V.L., P.v.d.B.), Hôpital Pitié Salpêtrière, UPMC Univ Paris 6 GRC 11 GRECHY, Paris, France; Neuromuscular Reference Centre, Department of Neurology (V.L., P.v.d.B.), and Department of Hematology (M.C.V.), Cliniques Universitaires Saint-Luc, University of Louvain, Brussels, Belgium.
Wouter
W
Meersseman
From the Departments of Neurology (N.C.V., B.G.v.E.) and Pathology (M.L.), Radboud University Medical Centre, Nijmegen, the Netherlands; Department of Internal Medicine (O.B.), Hôpital Pitié Salpêtrière, UPMC, U974, DHU I2B, Paris, France; Department of Hematology (M.C.M., H.L.), University Medical Center Utrecht, the Netherlands; Department of Pathology (M.L.), Antwerp University Hospital, University of Antwerp, Edegem; Departments of Internal Medicine (W.M.) and Hematology (M.D.), University Hospital Leuven, Belgium; Neurology Service (T.K., J.N.), Lausanne University Hospital CHUV, Lausanne; Department of Medical Oncology (T.P.), University Hospital Bern, Switzerland; Electromyography and Neuromuscular Pathologies Department Lyon Est (F.B.), Bron Hospitals, France; Departments of Pathology (N.R.) and Neurology (B.E.), Hôpital Pitié Salpêtrière, Myology Institute, Paris; Department of Hematology (V.L., P.v.d.B.), Hôpital Pitié Salpêtrière, UPMC Univ Paris 6 GRC 11 GRECHY, Paris, France; Neuromuscular Reference Centre, Department of Neurology (V.L., P.v.d.B.), and Department of Hematology (M.C.V.), Cliniques Universitaires Saint-Luc, University of Louvain, Brussels, Belgium.
Michel
M
Delforge
From the Departments of Neurology (N.C.V., B.G.v.E.) and Pathology (M.L.), Radboud University Medical Centre, Nijmegen, the Netherlands; Department of Internal Medicine (O.B.), Hôpital Pitié Salpêtrière, UPMC, U974, DHU I2B, Paris, France; Department of Hematology (M.C.M., H.L.), University Medical Center Utrecht, the Netherlands; Department of Pathology (M.L.), Antwerp University Hospital, University of Antwerp, Edegem; Departments of Internal Medicine (W.M.) and Hematology (M.D.), University Hospital Leuven, Belgium; Neurology Service (T.K., J.N.), Lausanne University Hospital CHUV, Lausanne; Department of Medical Oncology (T.P.), University Hospital Bern, Switzerland; Electromyography and Neuromuscular Pathologies Department Lyon Est (F.B.), Bron Hospitals, France; Departments of Pathology (N.R.) and Neurology (B.E.), Hôpital Pitié Salpêtrière, Myology Institute, Paris; Department of Hematology (V.L., P.v.d.B.), Hôpital Pitié Salpêtrière, UPMC Univ Paris 6 GRC 11 GRECHY, Paris, France; Neuromuscular Reference Centre, Department of Neurology (V.L., P.v.d.B.), and Department of Hematology (M.C.V.), Cliniques Universitaires Saint-Luc, University of Louvain, Brussels, Belgium.
Thierry
T
Kuntzer
From the Departments of Neurology (N.C.V., B.G.v.E.) and Pathology (M.L.), Radboud University Medical Centre, Nijmegen, the Netherlands; Department of Internal Medicine (O.B.), Hôpital Pitié Salpêtrière, UPMC, U974, DHU I2B, Paris, France; Department of Hematology (M.C.M., H.L.), University Medical Center Utrecht, the Netherlands; Department of Pathology (M.L.), Antwerp University Hospital, University of Antwerp, Edegem; Departments of Internal Medicine (W.M.) and Hematology (M.D.), University Hospital Leuven, Belgium; Neurology Service (T.K., J.N.), Lausanne University Hospital CHUV, Lausanne; Department of Medical Oncology (T.P.), University Hospital Bern, Switzerland; Electromyography and Neuromuscular Pathologies Department Lyon Est (F.B.), Bron Hospitals, France; Departments of Pathology (N.R.) and Neurology (B.E.), Hôpital Pitié Salpêtrière, Myology Institute, Paris; Department of Hematology (V.L., P.v.d.B.), Hôpital Pitié Salpêtrière, UPMC Univ Paris 6 GRC 11 GRECHY, Paris, France; Neuromuscular Reference Centre, Department of Neurology (V.L., P.v.d.B.), and Department of Hematology (M.C.V.), Cliniques Universitaires Saint-Luc, University of Louvain, Brussels, Belgium.
Jan
J
Novy
From the Departments of Neurology (N.C.V., B.G.v.E.) and Pathology (M.L.), Radboud University Medical Centre, Nijmegen, the Netherlands; Department of Internal Medicine (O.B.), Hôpital Pitié Salpêtrière, UPMC, U974, DHU I2B, Paris, France; Department of Hematology (M.C.M., H.L.), University Medical Center Utrecht, the Netherlands; Department of Pathology (M.L.), Antwerp University Hospital, University of Antwerp, Edegem; Departments of Internal Medicine (W.M.) and Hematology (M.D.), University Hospital Leuven, Belgium; Neurology Service (T.K., J.N.), Lausanne University Hospital CHUV, Lausanne; Department of Medical Oncology (T.P.), University Hospital Bern, Switzerland; Electromyography and Neuromuscular Pathologies Department Lyon Est (F.B.), Bron Hospitals, France; Departments of Pathology (N.R.) and Neurology (B.E.), Hôpital Pitié Salpêtrière, Myology Institute, Paris; Department of Hematology (V.L., P.v.d.B.), Hôpital Pitié Salpêtrière, UPMC Univ Paris 6 GRC 11 GRECHY, Paris, France; Neuromuscular Reference Centre, Department of Neurology (V.L., P.v.d.B.), and Department of Hematology (M.C.V.), Cliniques Universitaires Saint-Luc, University of Louvain, Brussels, Belgium.
Thomas
T
Pabst
From the Departments of Neurology (N.C.V., B.G.v.E.) and Pathology (M.L.), Radboud University Medical Centre, Nijmegen, the Netherlands; Department of Internal Medicine (O.B.), Hôpital Pitié Salpêtrière, UPMC, U974, DHU I2B, Paris, France; Department of Hematology (M.C.M., H.L.), University Medical Center Utrecht, the Netherlands; Department of Pathology (M.L.), Antwerp University Hospital, University of Antwerp, Edegem; Departments of Internal Medicine (W.M.) and Hematology (M.D.), University Hospital Leuven, Belgium; Neurology Service (T.K., J.N.), Lausanne University Hospital CHUV, Lausanne; Department of Medical Oncology (T.P.), University Hospital Bern, Switzerland; Electromyography and Neuromuscular Pathologies Department Lyon Est (F.B.), Bron Hospitals, France; Departments of Pathology (N.R.) and Neurology (B.E.), Hôpital Pitié Salpêtrière, Myology Institute, Paris; Department of Hematology (V.L., P.v.d.B.), Hôpital Pitié Salpêtrière, UPMC Univ Paris 6 GRC 11 GRECHY, Paris, France; Neuromuscular Reference Centre, Department of Neurology (V.L., P.v.d.B.), and Department of Hematology (M.C.V.), Cliniques Universitaires Saint-Luc, University of Louvain, Brussels, Belgium.
Françoise
F
Bouhour
From the Departments of Neurology (N.C.V., B.G.v.E.) and Pathology (M.L.), Radboud University Medical Centre, Nijmegen, the Netherlands; Department of Internal Medicine (O.B.), Hôpital Pitié Salpêtrière, UPMC, U974, DHU I2B, Paris, France; Department of Hematology (M.C.M., H.L.), University Medical Center Utrecht, the Netherlands; Department of Pathology (M.L.), Antwerp University Hospital, University of Antwerp, Edegem; Departments of Internal Medicine (W.M.) and Hematology (M.D.), University Hospital Leuven, Belgium; Neurology Service (T.K., J.N.), Lausanne University Hospital CHUV, Lausanne; Department of Medical Oncology (T.P.), University Hospital Bern, Switzerland; Electromyography and Neuromuscular Pathologies Department Lyon Est (F.B.), Bron Hospitals, France; Departments of Pathology (N.R.) and Neurology (B.E.), Hôpital Pitié Salpêtrière, Myology Institute, Paris; Department of Hematology (V.L., P.v.d.B.), Hôpital Pitié Salpêtrière, UPMC Univ Paris 6 GRC 11 GRECHY, Paris, France; Neuromuscular Reference Centre, Department of Neurology (V.L., P.v.d.B.), and Department of Hematology (M.C.V.), Cliniques Universitaires Saint-Luc, University of Louvain, Brussels, Belgium.
Norma
N
Romero
From the Departments of Neurology (N.C.V., B.G.v.E.) and Pathology (M.L.), Radboud University Medical Centre, Nijmegen, the Netherlands; Department of Internal Medicine (O.B.), Hôpital Pitié Salpêtrière, UPMC, U974, DHU I2B, Paris, France; Department of Hematology (M.C.M., H.L.), University Medical Center Utrecht, the Netherlands; Department of Pathology (M.L.), Antwerp University Hospital, University of Antwerp, Edegem; Departments of Internal Medicine (W.M.) and Hematology (M.D.), University Hospital Leuven, Belgium; Neurology Service (T.K., J.N.), Lausanne University Hospital CHUV, Lausanne; Department of Medical Oncology (T.P.), University Hospital Bern, Switzerland; Electromyography and Neuromuscular Pathologies Department Lyon Est (F.B.), Bron Hospitals, France; Departments of Pathology (N.R.) and Neurology (B.E.), Hôpital Pitié Salpêtrière, Myology Institute, Paris; Department of Hematology (V.L., P.v.d.B.), Hôpital Pitié Salpêtrière, UPMC Univ Paris 6 GRC 11 GRECHY, Paris, France; Neuromuscular Reference Centre, Department of Neurology (V.L., P.v.d.B.), and Department of Hematology (M.C.V.), Cliniques Universitaires Saint-Luc, University of Louvain, Brussels, Belgium.
Veronique
V
Leblond
From the Departments of Neurology (N.C.V., B.G.v.E.) and Pathology (M.L.), Radboud University Medical Centre, Nijmegen, the Netherlands; Department of Internal Medicine (O.B.), Hôpital Pitié Salpêtrière, UPMC, U974, DHU I2B, Paris, France; Department of Hematology (M.C.M., H.L.), University Medical Center Utrecht, the Netherlands; Department of Pathology (M.L.), Antwerp University Hospital, University of Antwerp, Edegem; Departments of Internal Medicine (W.M.) and Hematology (M.D.), University Hospital Leuven, Belgium; Neurology Service (T.K., J.N.), Lausanne University Hospital CHUV, Lausanne; Department of Medical Oncology (T.P.), University Hospital Bern, Switzerland; Electromyography and Neuromuscular Pathologies Department Lyon Est (F.B.), Bron Hospitals, France; Departments of Pathology (N.R.) and Neurology (B.E.), Hôpital Pitié Salpêtrière, Myology Institute, Paris; Department of Hematology (V.L., P.v.d.B.), Hôpital Pitié Salpêtrière, UPMC Univ Paris 6 GRC 11 GRECHY, Paris, France; Neuromuscular Reference Centre, Department of Neurology (V.L., P.v.d.B.), and Department of Hematology (M.C.V.), Cliniques Universitaires Saint-Luc, University of Louvain, Brussels, Belgium.
Peter van den
Pv
Bergh
From the Departments of Neurology (N.C.V., B.G.v.E.) and Pathology (M.L.), Radboud University Medical Centre, Nijmegen, the Netherlands; Department of Internal Medicine (O.B.), Hôpital Pitié Salpêtrière, UPMC, U974, DHU I2B, Paris, France; Department of Hematology (M.C.M., H.L.), University Medical Center Utrecht, the Netherlands; Department of Pathology (M.L.), Antwerp University Hospital, University of Antwerp, Edegem; Departments of Internal Medicine (W.M.) and Hematology (M.D.), University Hospital Leuven, Belgium; Neurology Service (T.K., J.N.), Lausanne University Hospital CHUV, Lausanne; Department of Medical Oncology (T.P.), University Hospital Bern, Switzerland; Electromyography and Neuromuscular Pathologies Department Lyon Est (F.B.), Bron Hospitals, France; Departments of Pathology (N.R.) and Neurology (B.E.), Hôpital Pitié Salpêtrière, Myology Institute, Paris; Department of Hematology (V.L., P.v.d.B.), Hôpital Pitié Salpêtrière, UPMC Univ Paris 6 GRC 11 GRECHY, Paris, France; Neuromuscular Reference Centre, Department of Neurology (V.L., P.v.d.B.), and Department of Hematology (M.C.V.), Cliniques Universitaires Saint-Luc, University of Louvain, Brussels, Belgium.
Marie-Christiane
MC
Vekemans
From the Departments of Neurology (N.C.V., B.G.v.E.) and Pathology (M.L.), Radboud University Medical Centre, Nijmegen, the Netherlands; Department of Internal Medicine (O.B.), Hôpital Pitié Salpêtrière, UPMC, U974, DHU I2B, Paris, France; Department of Hematology (M.C.M., H.L.), University Medical Center Utrecht, the Netherlands; Department of Pathology (M.L.), Antwerp University Hospital, University of Antwerp, Edegem; Departments of Internal Medicine (W.M.) and Hematology (M.D.), University Hospital Leuven, Belgium; Neurology Service (T.K., J.N.), Lausanne University Hospital CHUV, Lausanne; Department of Medical Oncology (T.P.), University Hospital Bern, Switzerland; Electromyography and Neuromuscular Pathologies Department Lyon Est (F.B.), Bron Hospitals, France; Departments of Pathology (N.R.) and Neurology (B.E.), Hôpital Pitié Salpêtrière, Myology Institute, Paris; Department of Hematology (V.L., P.v.d.B.), Hôpital Pitié Salpêtrière, UPMC Univ Paris 6 GRC 11 GRECHY, Paris, France; Neuromuscular Reference Centre, Department of Neurology (V.L., P.v.d.B.), and Department of Hematology (M.C.V.), Cliniques Universitaires Saint-Luc, University of Louvain, Brussels, Belgium.
Baziel G
BG
van Engelen
From the Departments of Neurology (N.C.V., B.G.v.E.) and Pathology (M.L.), Radboud University Medical Centre, Nijmegen, the Netherlands; Department of Internal Medicine (O.B.), Hôpital Pitié Salpêtrière, UPMC, U974, DHU I2B, Paris, France; Department of Hematology (M.C.M., H.L.), University Medical Center Utrecht, the Netherlands; Department of Pathology (M.L.), Antwerp University Hospital, University of Antwerp, Edegem; Departments of Internal Medicine (W.M.) and Hematology (M.D.), University Hospital Leuven, Belgium; Neurology Service (T.K., J.N.), Lausanne University Hospital CHUV, Lausanne; Department of Medical Oncology (T.P.), University Hospital Bern, Switzerland; Electromyography and Neuromuscular Pathologies Department Lyon Est (F.B.), Bron Hospitals, France; Departments of Pathology (N.R.) and Neurology (B.E.), Hôpital Pitié Salpêtrière, Myology Institute, Paris; Department of Hematology (V.L., P.v.d.B.), Hôpital Pitié Salpêtrière, UPMC Univ Paris 6 GRC 11 GRECHY, Paris, France; Neuromuscular Reference Centre, Department of Neurology (V.L., P.v.d.B.), and Department of Hematology (M.C.V.), Cliniques Universitaires Saint-Luc, University of Louvain, Brussels, Belgium.
Bruno
B
Eymard
D000328:Adult; D017668:Age of Onset; D005260:Female; D005500:Follow-Up Studies; D006801:Humans; D008297:Male; D008558:Melphalan / Q000008:administration & dosage / Q000009:adverse effects* / Q000627:therapeutic use*; D017696:Myopathies, Nemaline / Q000150:complications / Q000628:therapy*; D010265:Paraproteinemias / Q000150:complications / Q000628:therapy*; D012189:Retrospective Studies; D033581:Stem Cell Transplantation; D014182:Transplantation, Autologous / Q000379:methods; D016896:Treatment Outcome
D008558:Melphalan